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A CHES Publication

Spring/Summer 2019 I Volume 13

SAFE ACCESS to

CANNABIS

A Desire for the Masses, Necessity for Some

Advocacy to Protect: Patient Assistance l ATHN7 l Factor Your Way: Empowering You to Achieve Your Goals l Your Identity, Your Child’s Identity l Transitioning from High School to College or Work l How Mindfulness Keeps Us in the Moment l and more!


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LifeLines for HealthSM Disclaimers The views and opinions of our writers are not a reflection of Comprehensive Health Education ServicesTM, Inc. (CHES) or its sponsors. This newsletter is designed to provide a forum for community members to express their views from an open and honest platform. It is meant to provide a sharing of knowledge and experience to help one another. Nothing in this newsletter is meant to replace the advice of your HTC, medical professional team or insurance provider. You are always urged to seek the opinion of a healthcare professional for treatment and your specific insurance provider for information. We take your privacy very seriously. We would never disclose your personal health information without your express written consent. We would never sell nor make available our secure database to anyone. Articles and pictures may not be reproduced, published, and/or placed on websites without the express written permission of CHES. In every publication of LifeLines for HealthSM, we will provide links to other websites that are not owned or controlled by CHES or its sponsors. We cannot be responsible for privacy practices of other website owners, nor can we be responsible for the accuracy of the information provided.

Letter From the Editors Welcome! CHES is celebrating its 10-year anniversary of providing education, support and advocacy for the inhibitor community. Thank you as always for your faith and trust in us as a company whose key principles are accuracy, empathy and integrity. Here at CHES, our hearts and commitment to those managing an active inhibitor has never been stronger. We look forward to seeing you at any one of our five (5) national programs in 2019 with topics to include: ALL current methods of inhibitor management, biopsychosocial aspects of managing a chronic illness, grief, self-infusion, aqua-therapy, financial planning, mindfulness and pain to mention a few. After the Shock, an inhibitor family camp that allows us to invite families younger than age six for the first time will be returning. This is a CHES run program replacing Inhibitor Family Camp at Victory Junction. Our location at Camp Zeke in Pennsylvania offers water front privileges for swimming and boating, a heated outdoor pool for aqua-therapy; all within a beautiful wooded campus. We will continue to offer Inhibitor Family Camp at The Painted Turtle in CA in September 2019. Leverage, the Ultimate Inhibitor Adventure with GutMonkey will take place in September in OR. We are offering the Mom’s Uninhibited Meeting and Momentum, Men’s Retreat in July in Santa Fe, NM. It remains our honor to provide these programs. We remain humbled by your experiences, your resilience and your grace as you meet each challenge head on. Our programs are meant to augment the wonderful resources available and are as always, free of charge to attendees. In Bloodlines, we have published MASAC’s December 6, 2018 recommendations on Hemlibra for your convenience. As individuals managing an inhibitor, we are constantly assessing ways to treat them. Hemlibra has taken this community by storm and for many who have tried tolerizing with both recombinant and plasma-derived products, (with or without Rituxan), it is a true game-changer. In Family Matters, we begin the exploration process into how chronic illness affects your own or child’s identity. How is that identity changing for so many who have lived a life with constant bleeds and now are not? In the Spring of 2016, we published our most popular feature on medicinal cannabis. Since that time, nearly 33 states now have access to cannabis in one form or another, with more coming. Read more about its use, studies and advocacy to treat pain and other complex medical conditions from New England’s own, Mark Zatyrka, CEO of INSA with 3 medical/recreational sales locations in Massachusetts. (Concluded on pg 3)


2019 PROGRAM GUIDE

Event opportunities for the inhibitor community

CHES has been serving the needs of those with rare bleeding conditions since 2009. As long time members of the bleeding disorder community, our mission is to inspire awareness and self-reliance for patients with chronic health conditions, their families, and their communities. Below is a brief overview of the various programs we are offering to the inhibitor community in 2019. More details on each of our programs can be found on our website: www.CHES.education

Friday, June. 21st - Monday, 24th, 2019 Camp Zeke - Lakewood, PA

After the Shock provides parents and caregivers of diagnosed children (ages 0-18) with hemophilia and inhibitors the support they truly need. CHES understands at a personal level the obstacles and challenges that coincide with raising a child with inhibitors. We strive to equip every new parent with the tools to navigate his/her child through everything from hospital stays to home infusion. This weekend program is packed full of education and support. We play, learn, and grow while we build stronger relationships.

LEVERAGE

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Date: Wed, Sept. 4th - Sun, 8th, 2019 YMCA Camp Collins - Gresham, OR

Leverage is a pioneering, national program for young adults from ages 18 and up that have hemophilia with an inhibitor. The program consists of life changing experiences that allow participants to challenge themselves in ways they never thought possible through a series of outdoor adventure, experiential education activities, including fishing, rafting, reflection, and various ropes courses with adaptations in place.

The Ultimate Inhibitor Adventure

Friday, Sept. 27th - Monday, Oct. 30th, 2019 The Painted Turtle - Lake Hughes, CA

Inhibitor Family Camp is specially designed to meet the needs and limitations of children (ages 6-18) with both hemophilia & inhibitors. Immediate family members are invited because we understand that chronic illness affects the entire family. We play, learn, and grow while we build a stronger community.

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Friday, July 19th - Sunday, 21st, 2019 Santa Fe, New Mexico

Momentum is a men’s only, national program for those ages 18 and up that continue to live with both - hemophilia and an inhibitor. The program provides opportunities to not only speak with specialists familiar with inhibitors, but most of all, amongst themselves about issues and challenges important to them. The CHESTM logo and program logos are registered trademarks of Comprehensive Health Education Services, LLC. The use of these marks are restricted in part or their entirety without expressed written consent.


Integrity, Accuracy, Empathy...

FEATURE 8 I Safe Access to Cannabis: A desire for the masses, necessity for some Expert, Mark Zatyrka guides us through an explanatory tour of how cannabis (with its natural properties) reacts in the body and why it can be safely applicable in a multitude of conditions. With case examples, Mark introduces us to families who truly feel cannabis is a necessity along with their sacrificial stories that prove it.

CONTENTS COMMUNITY CHATTER

BLOODLINES

4 I MUM: a Journey of Faith

16 I New Extended Half-life, ESPEROCT® Joins the Market

New inhibitor mom, Amy Gayton entrusted CHES by committing to the first weekend away from her son when she attended MUM Mom’s, Uninhibited Meeting. This is her journey.

6 I Momentum: Brotherhood Open to adults with active inhibitors, Momentum is a promise to participants for improvement of life, a resource for self-care, and undeniably, it’s a brotherhood.

WHAT’S the PLAN? 34 I Transitioning from High School to College or Work You’ve managed to guide your child through adolescence and the teen years, but the journey doesn’t end there. Our expert, Lisa Cosseboom explains what learning accommodations you and your student may seek in the college world, as well as for those seeking vocational rehabilitation, and how to get them.

WHAT’S NEW 36 I ATHN7: Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Hemophilia The American Thrombosis and Hemostasis Network (ATHN) has begun what some might call “The ultimate study of hemophilia A and B treatment options”, matching clotting factors, bypassing products, and non-factor products up against each other.

38 I Advocacy to Protect: Patient Assistance The pendulum of patient assistance has shifted away from the patient’s favor. James Romano of PSI explains the sneaky ways insurance providers are enforcing prohibitions of patient assistance and what’s being done to combat these restrictions. 2

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Novo Nordisk’s portfolio expands further into the FVIIId market with an FDA-stamped approval on ESPEROCT. Trial results and release dates revealed here.

17 I MASAC Recommendations: Hemlibra With new drug, Hemlibra’s rapidly increasing use, confusion and inconsistency exist on just how this product should be administered in a variety of situations. MASAC (NHF’s Medical And Scientific Advisory Council) explains it’s newest set of recommendations for use.

24 I Factor Your Way: Empowering You to Achieve Your Goals As we change over time, so do our goals. New goals can sometimes force new treatment avenues. But to establish an effective plan, all factors should be carefully evaluated - like age, weight, PK levels, and more. Case studies Josh and Chip can explain.

FAMILY MATTERS 28 I Your Identity, Your Child’s Identity Establishing a healthy identity and a strong self-esteem can be a real challenge, especially for anyone living with a chronic medical condition. Dr. Gary McClain gives advice for both you and your child.

MIND BODY CONNECTION 42 I How Mindfulness Keeps Us in the Moment Mindfulness expert, Krystyn Strother reminds us to live in the moment. Easier said than done. But perhaps her tips will help.


Letter From the Editors (conclusion from inside page) We get questions all the time about transitional services from high school to work or college. When should that planning begin? How do you or your child get connected to Vocational Rehabilitation Services in your state and what does your state have to offer? What’s the Plan endeavors to answer some of those questions. What’s New features information about ATHN 7 Study, Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Hemophilia, and the newest extended release product from Novo Nordisk. We are hearing more and more about accumulator adjusters and insurance’s rejection of third party/manufacturer’s programs that assist with deductibles and out of pocket maximiums. PSI provides us an update regarding HR 3742 and HR 3976 Access to Marketplace Insurance Act. Community Chatter highlights our 2018 pilot of the Mom’s Uninhibited Meeting (MUM) for mothers of children with an active inhibitor. There are so many pressures on mothers, who are typically the primary caregiver in their child’s care. We appreciate Amy Gaytan’s perspective regarding what MUM meant to her. CHES neither endorses nor recommends any inhibitor treatment, as we are an education company, not physicians. As always, we encourage you as consumers to do your homework, avail yourself of studies, and know the risks before you make any decision that could produce positive or negative outcomes in the years to come. The decision to change treatment, remain with what works, or try any of the new and upcoming products is a personal decision that should be made between yourself, your physician, trusting family/friends and your child if they are old enough to understand. Do you have an idea, comment or suggestion? We really want to hear from you! - Janet Brewer & Eric Lowe jbrewer@ches.education l elowe@ches.education

“Courage is what it takes to stand up and speak; courage is also what it takes to sit down and listen.” - Winston Churchill

CONTENTS


by Amy Gaytan

a I

n November 2018, I had the opportunity to attend the ‘ Mom’s Uninhibited Meeting (MUM) offered by CHES. It was by chance that I saw the social media post and figured I wouldn’t be able to attend with everything going on in my life, but thankfully, I did! I had no idea what to expect. My weekend started as I boarded the plane to Nashville. It was difficult being away from my family for the first time since my son had been born. Although, after the evening session that day, I immediately felt that I had made the right decision. The first night was special because it set the stage for the rest of the weekend. Even though I had never met any of these lovely women, it was easy to talk with them and share experiences since we all knew we had at least one thing in common. That night, I felt emotionally drained because the words that were shared struck a chord with me. The weight of my feelings was put into words that I could not or dared not articulate, or even think about, but benefited deeply from hearing them spoken. My son was diagnosed with severe Hemophilia A in the early summer of 2018. About a month after that diagnosis, we found out he had developed inhibitors. To say we were overwhelmed is an understatement. The information that we had to take in seemed impossible to digest. Right when we thought we had a handle on it, something new would come up. Having the opportunity to learn and take things in that weekend opened my eyes to how much I still need to learn but also, more importantly, that my family has a

Journey of Fa it h

choice when it comes to my son’s treatment and we can and should ask questions. The educational sessions provided were eye opening. Since every mom had a different story and had been on their journey for different amounts of time, it was even more helpful. Each had different questions and stories that sparked questions for me and helped me learn even more than what was taught during the sessions. These things all changed my perspective on my son’s treatment. MUM 2019 INFO It has enabled me to have a stronger Location: Eldorado Hotel, Santa Fe, voice when speaking New Mexico with his doctors and Date: Friday, July 18th - 19th, 2019 it encouraged me to Registration: Closes July 1st, 2019 continue to champion at CHES.education/MUM for his best interest. Thanks to the help of Kathy Byrne, I was even able to stick my own vein! Having never been a fan of needles, it really was a milestone for me, and it encouraged me that someday I can learn to do the same for my son. I think that every mom with a child who has an inhibitor should attend the MUM program. For the moms that are just beginning on this journey like I was, it was a great opportunity to learn, connect, and share. For moms that are further along in their journey, it was a great opportunity to guide and support those of us who are just getting started. I can’t thank Janet, and the CHES team enough for offering such a wonderful weekend. I am very grateful for the opportunity that I had to experience this MUM weekend. Amy and her husband, Oscar have been married for 4 years and live in Orange County, CA with their 2-year-old son Hudson. She works at the Disneyland Resort as a Food and Beverage manager.

Supported by

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COMMUNITY CHATTER


FACTOR REPLACEMENT REFLECTS THE PROTECTION WITHIN

For people with hemophilia, Factor treatment temporarily replaces what’s missing.1,2 With a long track record of proven results, Factor treatment works with your body’s natural blood clotting process to form a proper clot.2-6 Brought to you by Takeda, dedicated to pursuing advancements in hemophilia for more than 70 years.7

Stay empowered by the possibilities. References: 1. Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016;388:187-197. 2. Canadian Hemophilia Society. Factor replacement therapy. http://www.hemophilia.ca/en/bleeding-disorders/hemophilia-a-and-b/the-treatment-of-hemophilia/ factor-replacement-therapy/. Accessed May 18, 2018. 3. Franchini M, Mannucci PM. The history of hemophilia. Semin Thromb Hemost. 2014;40:571-576. 4. Hvas AM, Sørensen HT, Norengaard L, Christiansen K, Ingerslev J, Sørensen B. Tranexamic acid combined with recombinant factor VIII increases clot resistance to accelerated fibrinolysis in severe hemophilia A. J Thromb Haemost. 2007;5:2408-2414. 5. Antovic A, Mikovic D, Elezovic I, Zabczyk M, Hutenby K, Antovic JP. Improvement of fibrin clot structure after factor VIII injection in haemophilia A patients treated on demand. Thromb Haemost. 2014;111(4):656-661. 6. Berg JM, Tymoczko JL, Stryer L. Many enzymes are activated by specific proteolytic cleavage. In: Biochemistry. 5th ed. New York, NY: WH Freeman; 2002. https://www.ncbi. nlm.nih.gov/books/NBK22589/. Accessed May 18, 2018. 7. Shire. Shire’s 70+ year commitment to the hemophilia community. https://www.shire.com/en/newsroom/2018/january/7sossj. Accessed June 6, 2018. Copyright © 2019 Takeda Pharmaceutical Company Limited. 300 Shire Way, Lexington, MA 02421. 1-800-828-2088. All rights reserved. TAKEDA and the TAKEDA logo are trademarks or registered trademarks of Takeda Pharmaceutical Company Limited. S46132 03/19


-Brotherhoodby Eric Lowe

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etween managing my own health (including a daily ITT regimen), partially running a business, raising two small children who have recently been dually-diagnosed with bleeding disorders, cheer practice, dance practice, and being married to a spouse who also works, “overwhelmed” has become a word of persistency in my life. Momentum (CHES’ men’s inhibitor retreat) was created to improve the quality of life for adults living with active inhibitors. As one of the creators of Momentum, I can humbly say that I benefit just as much as the average Joe. Or in this case, the average Bro. That’s what these meetings become to most of us. It’s not just a place of resources to better our lives, it’s also a brotherhood.

Although I don’t recall the specific methods of the session, I do remember leaving in a calm, relaxed state. As someone who has just surpassed their 11th anniversary of ITT, vein preservation is a big concern. Like almost everyone else in this group, I can jokingly say I could be a phlebotomist tomorrow if I wanted to. But CHES’ acclaimed Kathy Byrne, who has decades of infusion experience under her belt, had tips that even I had not known of. Just changing to a 27 gauge needle has really proven to provide more mileage out of my veins. And marking a hard-tofind vein with a fingernail impression has bumped my “sticking average” up to nearly 100%. But I’m not going to reveal all of her secrets here, as I hope to see each and every (qualifying) person who reads this article at a CHES program. Dr. Jonathan Bernstein of Connecticut Children’s Medical Center in Hartford gave us the updates on the latest and greatest products for inhibitor folks, as well as some that we either overlooked or had forgotten. After all, some products may not be new and shiny, but that doesn’t mean doctors and researchers aren’t reinventing treatment methods around them. Personally, the variety of products and the multitude of concerns and unanswered questions surrounding them are yet another overwhelming factor in my life, so I have chosen the wait-and-see method while maintaining ITT.

As Janet and I were prepping for Momentum last summer (along with the rest of the CHES team), she asked me if I was ready for this. “This” meaning acting as the lead facilitator of the program, as she (who usually takes on this responsibility for most CHES programs) would not be attending. After 10 years of doing this, I still feel the nerves of most people’s biggest fear - public speaking. But to Janet’s question, I replied, “absolutely, these are my people.” When I speak to this group, I’m not hosting guests or friends. I’m strengthening the bonds of my brothers as well as my own. Some of these guys I’ve known for years, and some were new acquaintances for me, but we all have opened arms to each other. There are no judgements, no cliches’, no negativity, hatred, or disrespect - only brotherly love and acceptance. We feel we understand each other better than anyone else can. And although we may not see or speak with each other more than a few times a year, the connection picks right back up where it left off.

To close the programs off, we hopped in the pool as Chad Brown, ex pro-am wake boarder with hemophilia A and professional coach as president/CEO of the Wingmen Foundation, gave us some lessons on low-impact, water aerobics in a playful yet beneficial manner. I look forward to reconnecting with all of our community friends and family each year at our programs, but Momentum is really something extra special to me. As I said, “my people”.

Supported by

Serving my overwhelmed state was the opener to the weekend, which was both happily welcomed and received as Emily Taylor sat us all down in a circle for some mindfulness techniques and theory of pain exercises.

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COMMUNITY CHATTER


You believe your hemophilia shouldn’t hold you back. We agree. We’re studying a monthly, subcutaneous injection of an investigational medication for males with hemophilia. Talk to your doctor about participating in one of the ATLAS clinical trials. See other side for more information

Who Can Participate in ATLAS?

What is the Investigational Medication?

To be eligible for one of the ATLAS trials you or your child must:

Fitusiran, the investigational medication being studied in the ATLAS trials, is a potential new approach to reducing or preventing bleeds in people with severe hemophilia A and B. Fitusiran is not factor replacement. It has been designed:

• Be male and at least 12 years old • Have severe hemophilia A or B • Have had at least 6 bleeds in the past 6 months treated on-demand or a minimum of 2 bleeds in the past 6 months requiring bypassing agent in patients with inhibitory antibodies to FVIII or FIX Additional criteria will be assessed to determine eligibility.

What is the Purpose of the ATLAS trials? The ATLAS trials are studying an investigational medication called fitusiran to evaluate its safety and ability to prevent or reduce the frequency of bleeds in males with severe hemophilia A and B, with and without inhibitors.

• To lower the amount of antithrombin so that the body can create more thrombin to support the natural clotting process • As a monthly injection under the skin. Fitusiran does not require venous access • As a fixed dose injection, meaning that everyone receives the same dose regardless of factor levels or weight • As a synthetic medication, meaning that it is not made from plasma, human or animal cells

Where Can I Learn More? To learn more about the ATLAS trials and see if you or your child may be eligible, please visit https://clinicaltrials.gov

Expressing an interest in the ATLAS trials does not oblige you in any way to participate.


SAFE ACCESS to

CANNABIS

A Desire for the Masses, Necessity for Some By: Mark Zatyrka

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t is reported that over 100 million US adults deal with chronic pain. Statistically 1 out of every two patients report pain as being the cause for inquiry with a medical professional (Zou & Kumar, 2018). With the cost of the prescribed pain medications being above a half a trillion dollars annually, many go into medical bankruptcy trying to find a reasonable quality of life. Most are willing to embrace a naturally occurring compound because it can be digested

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by the body and mind without severe negative side effects, contrary to over the counter drugs and prescriptions which come with an array of side effects and often result in patients needing to take additional medication (sometimes 3-4) to alleviate side effects caused by the first medication. The stream of medications at times can seem endless for those in chronic pain management situations and those with debilitating health issues.


The medical community has discovered that several components of the cannabis plant offer daily relief from pain, nausea, sleep issues, seizures, depression and anxiety. The component that is most widely recognized and utilized currently is known as Cannabidiol, or more commonly “CBD”. CBD is the crucial component within certain cannabis strains, aiding in the relief from a plethora of potential health issues. It is the non-psychoactive

compound that offers a wide array of medicinal benefits without resulting in the feeling which is commonly referred to as being ‘high’. This feeling of being high is a result of the other well-known component of cannabis, THC (Kubala, 2018).

FEATURE


Endocannabinoid System

Your body can naturally receive and process all parts of the cannabis plant without harm or side effects to your body when it is used properly and efficiently. Cannabinoids resemble endocannabinoids and can essentially be absorbed and recognized by your body as though they were internally produced. Cannabinoids bind to receptor sites in your brain and body that allow for the different effects, which are varying by the cannabis strain, to take place. Within your body, what are known as your CB1 and CB2 receptors are largely involved in various aspects of central neural activities and disorders, including appetite, learning and memory, anxiety, depression, multiple sclerosis, neurodegeneration, epilepsy, and even addiction (Mary’s Medicinal’s, 2018).

Family Focus A renowned case of success in the use of cannabis with children is that of Charlotte Figi and her family. Charlotte was age 2 when she had her first seizure. Her family physicians were absolutely convinced it was a onetime occurrence, but this became a prevalent issue in all of their lives. Ultimately, she was diagnosed with a rare form of epilepsy called Dravet

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Illustration by Geed on Adobe Stock

Similar to hemophilia, the endocannabinoid system is rarely discussed in medical schools, however, we are learning it can play an important role in our overall health. Each person is born with an endocannabinoid system which helps regulate different physiological and cognitive processes from fertility to the sensations of pain. This internal processing system in each one of us is the primary reason we as humans can utilize cannabis for a wide range of effects that it can present when used in various ways. Our endocannabinoid system is essentially the system which allows for and develops communication between our cells. The CB1R and the endocannabinoid system are largely involved in various aspects of central neural activities and disorders, including appetite, learning and memory, anxiety, depression, schizophrenia, stroke, multiple sclerosis, neurodegeneration, epilepsy, and addiction (Zou & Kumar, 2018).

Previously, cannabis wouldn’t even enter the conversation until young adulthood, but now more than ever parents are looking to cannabis to solve the severe issues that their child may be dealing with. We are on the precipice of understanding the benefits of cannabis with children, therefore long-term scientific data is not available at this point, yet there are many anecdotal and surface studies that have occurred to open the minds of many.

Syndrome. This is a type of epilepsy that typically does not respond to normal forms of medication or therapy. Her family felt helpless as their baby seemed to be slipping away and they worried about her ongoing development. As they went down every road that western medicine had to offer, they eventually had to accept that it was time to consider an alternative. This is when Charlotte’s

Spring/Summer 2019

family began the conversation with their physician about the use of CBD oil. They had heard anecdotal reports of cannabis helping with the frequency of Grand Mal seizures in adults and were curious if their daughter could benefit from such a regimen as well. Although no doctor wanted to risk their license on approving the use of CBD oil for Charlotte due to her age, the family did not give up. Eventually


they found Dr. Margaret Gedde, who approved the use of ‘pure CBD oil’ for her seizures. The next step was a long process, but they were able to find the Stanley brothers who had been growing medical marijuana for quite some time and were already heavily focused on isolating the genetics that were best suited for cultivating one specific component, CBD. They were able to go through and identify the specific traits within the strain, that they would eventually name Charlotte’s Web, to ensure less than 0.3% THC would be present within each yield along with very high levels of CBD. The Stanley brothers’ pride in themselves on providing access to safe cannabis is their life long goal, which they are achieving daily. From the cannabis grown by the Stanley brothers they were able to create a high end and very potent CBD focused oil extracted from the plant itself. Charlotte went from dealing with 300+ Grand Mal seizures per week to less than 1 per week with the use of the CBD oil produced for her. This case is the cornerstone of how the cannabis industry and CBD industry took the momentum they had gained

via countless decades of initiatives and used it to launch the next phase in the cannabis movement (tiffany@ sweethoneybeehealth.com, 2018). What the Figi family was able to achieve, by finding a responsible vendor and a medical professional who was willing to provide access to highend medicinal cannabis, may easily be identified as a historical turning point for the cannabis industry. As a result of coverage of Charlotte’s story by numerous media outlets including CNN, acceptance and excitement around the potential surrounding CBD rose overnight; over the past 5-6 years, families have become increasingly willing to move across the country in order to gain legal and safe access to this plantbased medication. Hundreds of families have made this journey, uprooting their family from their home state in order to pursue accessible legalized marijuana, forgoing any federal laws in the pursuit of this medical alternative.

Colorado has become the ‘end all, be all’ for parents in pursuit of medicinal marijuana as a treatment for their children. “Hundreds of families have moved to Colorado in hopes of healing their sick children — kids conventional medicine has failed” (Ingold, Amon, & Pierce, 2014). While some are met with helping hands and success stories erupt, others are met

FEATURE


with a harsher reality that they are too late for CBD to help, or they lack accessibility to help in administering cannabis treatments and creating proper regimens for proper dosing, so they are left to their own devices and must administer the medication to their children in their own home without proper guidance. Overall, the amount of research and information regarding children and medicinal use of cannabis is quite minimal,

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therefore we must rely on current research to continue to decipher the reality of cannabis within the medical community. This has done little to hinder the hundreds of registered medical marijuana patients under the age of 18, brought by their parents to Colorado: In 2014 there were 400+ registered patients under the age of 18, many of whom were not residents of the state until after seeing the CNN report on Charlotte Figi and her progress (https://www. colorado.gov/, 2017).


Mark and Maria Botker from Minnesota were willing to separate their own family to find help for Greta, their 7-year-old child. While Maria traveled to Colorado with Greta seeking medical help, Mark remained behind at their farmhouse in Minnesota with their two older daughters (10, 13). In search of any help in mitigating the number of seizures their daughter was incurring daily, the family had gone through multiple rounds of pharmaceutical regiments and went as far as allowing brain surgery to be performed on Greta, but nothing helped. They came to a final resolution that their next step was to investigate cannabis and the potential for CBD (Bello, 2014). After only a few months of living in Colorado they realized their neighbors, Anna and Biagio Burriesci, were dealing with a very similar situation. The Burriesci family moved to Colorado from New York seeking the benefits of medical marijuana for their young daughter Grace. In doing so, they lost their jobs and their financial security, taking over a $200,000 loss on their estate in Queens, New York in order to pursue accessible and legalized medical cannabis. “This condition ultimately meant death for our kid, so we were going to war for her,” “Families are desperate,” Biagio stated. Their daughter went from experiencing 300 seizures a day to less than 5 daily and, thanks to the reduction in her episodes, she was able to learn useful motor skills to aid in coping through these episodes (Bello, 2014).

Breakthrough within the Cannabis Community In June of 2018 the FDA approved the use of concentrated CBD, called Epidiolex, to treat rare forms of epilepsy. This non-synthetic cannabis-derived product is the first plantderived cannabinoid pharmaceutical ever approved by the FDA. The company that created it, known as Greenwich Biosciences, has been at the helm of outstanding research, creating an avenue for specific cannabis products to become mainstream. Epidiolex has been available since November 2018, with sales in the first 2 months rising over a staggering $4.5 million dollars, and over 500 physicians prescribing this medication, the demand and support for this medication is apparent. As we continue to advance with research and development, we are also gaining approval by the government, setting the stage for the future of other cannabis products and overall legalization (Partnership, 2018). Only 3 months after the release of Epidiolex, the U.S. Drug Enforcement Administration removed certain forms of CBD products from the Schedule 1 class and reclassified the cannabis-derived products as Schedule 5. This decision is the first official admission by the government that cannabis has medicinal value. Anything that is on the approved list of items and tests under 0.1% of THC is federally approved for sale and use with a prescription. CBD products derived from cannabis will be available for the masses in our lifetime, guaranteed, but it is not over yet as we all need to help and continue to move the needle in the right direction for overall

progression (Hemp Industry Daily, 2018). On March 12, 2019 a legislative bill, initiated by a Wichita couple with special needs children, will be moving forward in Topeka. “House Bill 2244 was introduced into the Kansas legislature to allow people with life-threatening medical conditions to get treatment with CBD oil with a small amount of THC.” In its title, the bill “authorizes the use of cannabidiol treatment preparation to treat certain medical conditions.” Both daughters of Gwen and Scott Hartley were born with microcephaly, a birth defect that causes the head to be under-developed and therefore smaller. Claire recently passed at the age of 17, and her parents have dedicated their lives to helping their 12-year-old live a full life. Titled “Claire and Lola’s law”, their parents hope to help other families seeking advice on, help with, and most importantly access to, CBD oil. The reality of this law change could be ground breaking, as Claire and Lola’s mother Gwen explained: The law is actually an affirmative defense law which would allow us to go to a legal state, purchase the THC CBD oil that would benefit our child, bring it back into Kansas and not get arrested or lose custody of my child,” Gwen explained last week. If this was approved this could be the light at the end of the tunnel for numerous families and a signal to other states to lift the ban on CBD use for severely debilitating disorders and life-threatening illness (Viviani & Kwch, 2019). Many adults wonder: Is cannabis safe? Why would cannabis be the best option? Will it help? If I were to choose this route will my personality or decision-making change, or be altered in some way? Is it safe enough for my child? Is it proven to help my/my child’s disorder or level of pain? These are all valid questions when first entering the world of cannabis. I am always excited to share with others that it is proven through scientific studies over decades of research, that our own individual endocannabinoid system is intended to receive and utilize all types of cannabinoids.

We are literally built to utilize cannabis for the benefits it offers, pure and simple.

FEATURE


With legalization and decriminalization on the rise, we are finally moving the needle in the right direction for cannabis patients and users across the United States. 33 states have approved medical access for patients and 10 states have approved recreational access for patrons. The level of progress at the state level gives momentum and hope for legalization at the federal level, making this an approved substance across the country and allowing for safe access for the masses (NCSL, 2018).

Potential health issues that can improve with the use of CBD:

It can bring a grown man to his knees to see his only child in danger or worse to see them facing death at a young age. Those that are dealing with the consistent thought and therefore mindset of losing a child to a type of disorder or disease that can be treated in the same country that you are a citizen within seems unjustifiable. Many of us seek a quality of life balance as we get older and realize what is important to us. What if you were never given the chance to feel love, to speak to your parents, to feed yourself your favorite meal or play a board game during a family gathering? Many of us in the bleeding disorders community know how it feels as a parent of a child that could not enjoy their young life. Please ask yourself some hard questions and try to put yourself in another’s shoes to try and feel the level of utter dismay and confusion that they feel day in and day out. The way we resolve this issue is to keep this subject on the forefront of discussions until all laws are altered or changed in a way to allow for safe and responsible access to cannabis and all products derived from this plant. It can change the world if given the chance.

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Addiction

Anxiety

ADHD

Autism

Cancer – varying types

Crohn’s Disease

Depression

Epilepsy

Fibromyalgia

Inflammation

Migraines

Multiple Sclerosis

Nervous System

- varying issues

Neuropathy

OCD

Pain – varying levels

Psoriasis

Skin Issues – varying types

Sleep disorders (Sam, 2019)


References: Bello, M. (2014, February 18). Parents move to Colorado for ‘miracle’ pot for children. Retrieved March 8, 2019, from https://www.usatoday.com/story/news/nation/2014/02/17/ moving-medical-marijuana-epilepsy-children/5255323/ G. (2019). MS Spasticity. Retrieved March 8, 2019, from https://www.gwpharm.com/healthcare-professionals/ sativex/patient-information Hemp Industry Daily. (2018, September 27). DEA moves CBD medicines off Schedule 1, reclassifying as Schedule 5. Retrieved March 8, 2019, from https://mjbizdaily.com/deamoves-cbd-medicines-off-schedule-1-a-limited-expansionof-cannabis-access/ Hill, K. P., Palastro, M. D., Johnson, B., & Ditre, J. W. (2017). Cannabis and Pain: A Clinical Review. Cannabis and Cannabinoid Research, 2(1), 96-104. doi:10.1089/ can.2017.0017 Ingold, J., Amon, J., & Pierce, L. (2014). CBD in Colorado: Seeking a marijuana miracle. Retrieved March 8, 2019, from http://extras.denverpost.com/stateofhope/ Kubala, J. (2018, February 26). 7 Benefits and Uses of CBD Oil (Plus Side Effects). Retrieved March 1, 2019, from https:// www.healthline.com/nutrition/cbd-oil-benefits Colorado.gov. (2017, August). Medical Marijuana Registry Program Statistics [PDF]. State of Colorado. G. (2019, February 26). GW Pharmaceuticals plc Reports Financial Results and Operational Progress for the Quarter Ended December 31, 2018. Retrieved March 8, 2019, from http://ir.gwpharm.com/news-releases/news-release-details/ gw-pharmaceuticals-plc-reports-financial-results-andoperational

Mary’s Medicinals. (Ed.). (2018). SCIENCE OF CANNABINOIDS. Retrieved March 1, 2019, from https:// marysmedicinals.com/science/ NCSL. (Ed.). (2018, December 14). MARIJUANA OVERVIEW. Retrieved March 03, 2019, from http://www.ncsl.org/ research/civil-and-criminal-justice/marijuana-overview.aspx Partnership. (2018, October 03). What Parents Should Know About Kids Using CBD. Retrieved March 8, 2019, from https://drugfree.org/parent-blog/what-parents-shouldknow-about-kids-using-cbd/ Sam. (2019). Home. Retrieved March 8, 2019, from https:// www.theroc.us/ Tiffany@sweethoneybeehealth.com. (2018, August 3). CBD, Charlotte Figi and Fighting for Life. Retrieved March 8, 2019, from http://sweethoneybeehealth.com/cbd/2018/08/03/ cbd-charlotte-figi-and-fighting-for-life/ Viviani, N., & Kwch. (2019, February 28). Bill allowing THC CBD oil for medical treatment moves forward in Topeka. Retrieved March 8, 2019, from https://www.wibw.com/ content/news/Bill-allowing-THC-CBD-oil-for-medicaltreatment-moves-forward-in-Topeka-506519011.html Zou, S., & Kumar, U. (2018). Cannabinoid Receptors and the Endocannabinoid System: Signaling and Function in the Central Nervous System. International Journal of Molecular Sciences, 19(3), 833. doi:10.3390/ijms19030833

Mark Zatyrka lives with severe hemophilia A and many of its complications. Zatyrka was formerly an owner of American Homecare Federation (AHF), a specialty homecare pharmacy for individuals living with bleeding disorders. Currently, Zatyrka is CEO of Insa, a vertically integrated cannabis operator in Massachusetts and Pennsylvania. Insa’s mission is to illuminate this path for others by providing premium, personalized, locally-grown medical and adult-use cannabis products that inspire people to feel and live better. Insa’s uncommon products ignite the benefits of this powerful and wildly versatile plant. Zatyrka’s growers, artisans, chefs, and thinkers combine deep knowledge and experience to cultivate premium cannabis and cannabis products with diverse terpene and cannabinoid profiles. Mark lives in CT with his wife and 6-year-old daughters. He is an active volunteer with the New England Hemophilia Association and the Hole in the Wall Gang Camp.

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Discover more about IXINITY

®

Visit IXINITY.com

Aptevo BioTherapeutics LLC, Berwyn, PA 19312 IXINITY® [coagulation factor IX (recombinant)] and any and all Aptevo BioTherapeutics LLC brand, product, service and feature names, logos, and slogans are trademarks or registered trademarks of Aptevo BioTherapeutics LLC in the United States and/or other countries. © 2018 Aptevo BioTherapeutics LLC.

All rights reserved.

CM-FIX-0258

New Extended Half-life, ESPEROCT® Joins the Market By: Janet Brewer M.Ed

O

n February 19, 2019, Novo Nordisk announced the US FDA approval of ESPEROCT®.

ESPEROCT® (turoctocog alfa pegol, N8-GP) for the treatment of adults and children with hemophilia A has been approved for routine prophylaxis, on-demand treatment, control of bleeding episodes and perioperative management of bleeding. ESPEROCT® is an extended half-life product which according to company reports, provides a 1.6 fold half-life prolongation for adults/adolescents

and 1.9 fold half-life for children compared to standard half-life factor VIII products.

Due to third-party IP agreements, Approval of ESPEROCT® is the result Novo Nordisk will not be able to launch of a five year, prospective, multiESPEROCT® before 2020 in the USA. center clinical trial of 270 (202 adults/ adolescents and 68 Esperoct® joins other extended half-life products children) previously currently in the hemophilia market space, such as: treated patients with severe hemophilia A, Hemophilia A Hemophilia B with no history of an Eloctate (Sanofi) Alprolix (Sanofi) inhibitor. No reported Adynovate (Takeda) Rebinyn (Novo Nordisk) safety concerns were Afstyla (CSL Behring) Idelvion (CSL Behring)

https://www.novonordisk.com/media/news-details.2235689.html

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identified after 5 years of clinical exposure.

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Jivi (Bayer)


HEMLIBRA

MASAC Recommendations:

T

By: Janet Brewer M.Ed

he National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC) released their recommendations on the use of Hemlibra (emicizumab) on December 6, 2018. Hemlibra was approved for use on November 16, 2017 for persons with hemophilia A and inhibitors. In addition, it was approved for persons with hemophilia A with or without an inhibitor on October 4, 2018. For purposes of this article, CHES’ focus remains on the inhibitor community. For full MASAC recommendations https://www.hemophilia.org/Researchers-Healthcare-Providers/Medical-and-Scientific-Advisory-Council-MASAC/MASACRecommendations/Recommendation-on-the-Use-and-Management-of-Emicizumab-kxwh-Hemlibra-for-Hemophilia-Awith-and-without-Inhibitors

Clinical Management Guidance *Full prescribing information is available in the package insert. In addition, the prescribing information contains a boxed warning regarding the risk of thrombotic microangiopathy (TMA) and thromboembolism. Because of the complexity of these adverse events in association with the use of activated prothrombin complex concentrates (aPCC) to treat breakthrough bleeding in patients receiving emicizumab, it should be prescribed and bleeding events should be followed by, or in close coordination, with all the appropriate staff of the patient’s HTC, including physicians/providers and nurse coordinators. The following recommendations are intended to address clinical management issues that are not addressed fully within the package insert.

?

1. Who is appropriate to be considered for treatment with emicizumab? Physicians caring for persons with hemophilia A (PwHA), of any age or severity, with or without inhibitors should discuss this new therapeutic option with them, including an assessment of the risks and benefits of emicizumab compared to their existing therapy. However, based on the clinical trial data, any PwHA with an inhibitor who is having frequent bleeding episodes and is on either episodic therapy or bypassing agent (BPA) prophylaxis will likely derive significant benefit from emicizumab. Those on BPA prophylaxis with few bleeding episodes could either continue on that BPA or could consider switching to emicizumab based on overall cost-effectiveness and reduced burden of administration. In addition, infants

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? BLOODLINES


should be considered for prophylaxis with emicizumab at any time after birth given the increased risk of intracranial hemorrhage prior to initiation of FVIII prophylaxis. Note, there is limited data on the use of emicizumab in infants under 6 months of age and the pharmacokinetic exposure is likely to be lower compared to older infants and children. We strongly encourage prospective data collection concurrent with its use in this age group.

2. Recommendations for initiation of emicizumab a. PwHA and inhibitors Administration of activated prothrombin complex concentrates (aPCCs) should be discontinued at least 24 hours prior to initiation of emicizumab. b. PwHA and no inhibitors... (skipped) c.

Patient education should include:

i. Giving the loading dose(s) under medical supervision, in order to review and observe self-administration technique, and determination of what dose will be used after the 4-week loading period is complete. ii. Discussion of the appropriate clotting factor product (factor VIII concentrate or bypassing agent) and dose to use for a breakthrough bleed, review of the signs and symptoms of a bleed, and stressing the need to contact the Hemophilia Treatment Center (HTC) if a breakthrough bleed requires more than 1-2 treatments and/or is not responsive to treatment. iii. Warning against concomitant use of aPCCs and associated risks, including what a thrombosis is, and the associated symptoms and to seek medical help should it occur iv. Reminding when emicizumab is re-ordered, patients must provide their current weight to assure proper dosing and proper vial combinations to achieve the calculated dose. v. Patients must contact the HTC in the event of surgery, whether elective or emergent, to assure a plan for treatment is developed, including if any clotting factor product will be used, and, if so, at what dose and frequency, and if antifibrinolytic agents are advised.

viii. Loss of efficacy should prompt evaluation for antidrug antibodies (see below). ix. Patients should be counseled they should continue to travel with an emergency dose of factor concentrate to allow prompt management of any significant and serious or life-threatening bleeding

3. Recommendations on Acute Bleed Management – PwHA and Inhibitors Despite the high efficacy in the prevention of bleeding events, clinicians and patients should still expect breakthrough bleeding events in patients on emicizumab prophylaxis, which in PwHA with inhibitors will likely mean the need for concomitant use of alternative hemostatic therapies. Due to the serious adverse events observed in the clinical trial program, we are reiterating below the previous interim guidance on management of breakthrough bleeding, surveillance for thromboembolic and TMA events, and recommendation on appropriate use of laboratory assays. No TMA or serious thrombotic events have been reported to date with adherence to these management principles after implementation within the global clinical trial program or since commercial availability within the US in pediatric and adult patients. a. General approach to breakthrough bleeding: Emicizumab is likely to transform the bleeding phenotype of patients to a milder phenotype. Given improved baseline hemostasis in patients on emicizumab prophylaxis, the current paradigm of treating at the first signs and symptoms of bleeding in some cases should change. Significant and serious or life-threatening bleeding should continue to be treated promptly. However, there should be additional evaluation of muscle and joint complaints prior to treatment with an additional hemostatic agent. For equivocal signs or symptoms of minor bleeds, the patient should contact their provider before initiating bypass therapy treatment.

vi. Patients should be warned that if they stop emicizumab, residual plasma levels may persist for as long as 6 months, and thus, risk mitigation regarding the use of aPCCs should be used during that 6-month period to avoid the risk for thrombotic and TMA complications vii. Regular clinical review of the efficacy for bleed prevention should be conducted once the patient has reached the maintenance dosing and at 3 months, 6 months and 12 months after initiation of therapy, and thereafter at 6 month intervals at the discretion of their physician.

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A

B


b. Caution with dose and duration of bypass therapy: i. Acute bleeding events should be managed with rFVIIa, at a dose of 90-120 mcg/kg as an initial dose. The vast majority of bleeds should be able to be managed with 1-3 doses administered at no more frequency than q2h intervals. ii. Use of aPCC for breakthrough bleed treatment for patients on emicizumab should be avoided if possible, and rFVIIa should be the first option used to treat. If aPCC is used, it should be limited to no more than 50 IU/kg administered as an initial dose and not exceed 100 IU/kg/day. Duration of aPCC therapy should also be minimized, as prolonged use for >24 hours, especially with doses above 100 IU/kg/day, was associated with thrombosis and TMA. iii. Repeated dosing of any BPA beyond the above recommendations should be performed under medical supervision with consideration for verifying severity of bleeds prior to continuing to repeat dosing. iv. For significant bleeding that is not responding to BPA, consider using porcine factor VIII or human FVIII if partially tolerized. Use of these agents would also allow therapeutic monitoring with access to the bovine chromogenic assay. v. Clinical and laboratory monitoring: All patients on emicizumab who have received aPCC for breakthrough bleeding for more than 24 hrs should be evaluated for any clinical symptoms that could be consistent with a thromboembolic event (with a high level of suspicion for atypical sites, e.g. cerebral sinus venous thrombosis), should have laboratory monitoring to look for evidence for TMA (this should include D-dimer, prothrombin fragment F1+2 (if available), platelet count, serum creatinine, LDH and peripheral blood smear analysis to look for schistocytes). Monitoring should continue daily while the patient continues to receive aPCC until 48 hours following the last dose of aPCC. vi. Management of TMA: Use of aPCC should cease immediately. The half-life of emicizumab is 28 days and it will remain in the patient’s system for months after discontinuation of emicizumab. Each of the reported cases of emicizumab+aPCC TMA resolved quickly after discontinuation of aPCC with supportive care alone or in conjunction with plasmapheresis. In the 2 cases treated with plasmapheresis, emicizumab was not completely removed and in spite of discontinuation of emicizumab one of these patients had measurable emicizumab laboratory effects for several months. Emicizumab was restarted in two patients without recurrence of TMA.

4. Recommendations on Acute Bleed Management – PwHA without inhibitors... (skipped) 5. Recommendations on laboratory assays while on emicizumab Although emicizumab mimics the function of activated factor VIII, it is biologically different from activated factor VIII, having much lower affinity for factor IXa and X, not requiring activation, and not deactivated by the protein C/S pathway. These fundamental differences affect our interpretation of clotting assays. a. Laboratory monitoring of emicizumab is not required while on routine prophylactic dosing b. aPTT-based assays, including clot-based FVIII activity assays, should not be performed while on emicizumab, as they will yield misleading results (ie. artifactually shortened aPTT and elevated FVIII activity). c. Chromogenic FVIII activity assays will only provide an assessment of emicizumab activity if the assay includes all human reagents but these are not widely available. A chromogenic FVIII assay that uses bovine reagents may be used to assay the FVIII activity of any additional FVIII concentrate administered or endogenous FVIII. d. The clot-based Bethesda assay cannot be utilized to

assess FVIII inhibitor levels. However, a bovine-reagent chromogenic-based inhibitor assay can report out FVIII inhibitor levels. If samples are submitted to the CDC for central laboratory testing, they must be clearly identified that the patient is on emicizumab. e. Exploratory laboratory assays: i. Thromboelastography/thrombelastometry/ thrombin generation ii. Clot waveform analysis

BLOODLINES


These and other assays under continued investigation are likely to allow for quantitation of emicizumab concentration from plasma. However, due to the intrinsic differences between emicizumab and FVIII, this complicates assignment and interpretation of FVIII-equivalent activity. Caution should remain in extrapolating clinical correlates using ‘FVIIIequivalence’ from such assays until further clinical data is available.

Summary of Assay Principles with Emicizumab Results affected by emicizumab • Activated partial thromboplastin time (aPTT) • Bethesda assays (clotting-based) for FVIII inhibitors • One-stage, aPTT-based single factor assays (eg. FVIII activity) • aPTT-based activated protein C resistance • Activated clotting time (ACT)

1. Anti-drug antibodies: a. The prescribing information for emicizumab includes important safety information on the risk for anti-drug antibodies. Use of any therapeutic protein carries the potential for the development of antidrug antibodies. These were observed within the global clinical trial program with emicizumab and are described within the US and European Union (EU) emicizumab product labels. The immunogenicity of emicizumab was evaluated using an enzyme-linked

Result unaffected by emicizumab • Bethesda assays using bovine chromogenic reagents for FICC inhibitors • Thrombin time (TT) • One-stage, prothrombin time (PT)-based single factor assays (eg. FX activity) • Immuno-based assays (i.e. ELISA, turbidometric methods) - eg. VWF antigen • Genetic tests of coagulation factors (eg. Factor V Leiden, Prothrombin 20210 mutation)

pharmacokinetics). One patient from HAVEN 2, who developed an anti-emicizumab neutralizing antibody, experienced loss of efficacy after 5 weeks of treatment. There was no clinically apparent impact of the presence of anti-emicizumab antibodies on safety. To date, more than 1100 persons with hemophilia, with and without inhibitors to factor VIII have been treated with emicizumab worldwide. b. The development of an anti-drug antibody to emicizumab is distinct from the development of an inhibitor to factor VIII. Notably, anti-drug antibodies directed against emicizumab may affect how it works in the patient but will not affect the person’s underlying hemophilia or inhibitor status, nor the ability to manage bleeding events with their conventional therapies, including bypassing agents. c. Continued diligence in evaluation of clinical efficacy of emicizumab as would be expected for any hemophilia therapeutic. Loss of efficacy of emicizumab may be manifest by an increase in breakthrough bleeding events. Patients concerned about a loss of efficacy should seek prompt evaluation by their provider.

immunosorbent assay or an electrochemiluminescence assay. In the dose-finding trial (n = 18), four patients tested positive for anti-emicizumab antibodies. In the pooled HAVEN clinical trials, 3.5% of patients (14/398) tested positive for anti-emicizumab antibodies and <1% (3/398) developed anti-emicizumab antibodies with neutralizing potential (based on declining

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d. We have provided information on assays that may be utilized for evaluation of the activity of emicizumab, utilizing a chromogenic factor VIII activity assay with human reagents that are recognized by emicizumab. However, the widely available conventional aPTT and clot-based factor VIII activity assays can be used in evaluating a case of loss of efficacy. The aPTT should be within the normal range in all persons with hemophilia when obtained during ongoing treatment with emicizumab; similarly, clot-based factor VIII activity assays will be well above the normal range. In


the course of evaluating a reported loss of efficacy, a prolonged conventional aPTT assay and/or a low factor VIII activity (by clot-based assay or chromogenic assay using human reagents) in a person treated with emicizumab should be a useful initial evaluation for the presence of a neutralizing anti-drug antibody directed against emicizumab. Additional information regarding laboratory assays may be found within the prescribing information. e. There are no commercially available assays in the US for determination of anti-drug antibodies directed against emicizumab. Should a patient and provider have suspicion of an anti-drug antibody outside of the ongoing clinical trial program, they should contact the manufacturer (https://www.gene.com/contact-us/ submit-medical-inquiry) for guidance on subsequent evaluation. f. Recommend that patients and providers continue diligence in reporting any unanticipated adverse events. Available reporting mechanisms can be reviewed here (https://www.fda.gov/Drugs/ GuidanceComplianceRegulatoryInformation/Survei...)

2. Recommendations on surgical management with emicizumab Emicizumab is approved for prophylaxis but how this extends to surgical prophylaxis remains to be fully understood. While it improves hemostasis, it does not normalize hemostasis. This is especially important when planning hemostatic control in the surgical setting. Within the clinical trials, some patients had adequate hemostatic control with emicizumab alone for minor

surgical procedures while others did not. This is similar to what has historically been seen in patients with mild hemophilia. a. Surgeries should be conducted at centers with appropriate experience and access to necessary laboratory monitoring assays for concomitant use of hemostatic agents b. Elective surgeries should be conducted after patients have completed the loading dose phase and are at steady state maintenance dosing. c. Emicizumab alone should not be presumed to be adequate for major procedures where current standards of care are to maintain factor levels within the normal range for a period of days. d. Close monitoring of bleeding control as well as access to appropriate laboratory assays to monitor therapy (eg. chromogenic FVIII assay with FVIII replacement) is very important when determining treatment plans for patients on emicizumab needing surgical procedures. e. For major surgeries and procedures where bleeding could result in serious complications, patients should be provided rFVIIa or FVIII replacement pre- and postoperatively to maintain adequate hemostasis at the discretion of the treating physician. Further research/ experience is needed to form better defined treatment plans for different surgical procedures. f. Providers are cautioned to consider that bleeding complications from surgeries in hemophilia patients still greatly outweigh thrombotic complications in frequency and morbidity/mortality.

Note: For illustrative purposes, only. Any surgical procedure involving Hemlibra should be coordinated with the recommendations in this article and under the care of the individualâ&#x20AC;&#x2122;s medical care team.

BLOODLINES


3. Recommendations on concomitant use of emicizumab during immune tolerance induction. There are no clinical data on the concomitant use of emicizumab prophylaxis during immune tolerance induction (ITI). There has been significant academic debate as to the need for ITI with the availability of emicizumab (and other novel therapeutic agents under continued investigation). There is considerable uncertainty as to the implications of persistent factor VIII inhibitors in PwHA given the degree of efficacy for bleed protection provided by emicizumab. Nevertheless, MASAC recommends that the pros and cons of the various approaches for a PwHA and inhibitors be part of a patient/clinician shared decision-making and ITI should remain an option for their care. We also recommend that long term follow up and interventional trials that include the concomitant use of emicizumab with ITI should be encouraged. If ITI with concomitant emicizumab prophylaxis will be pursued we provide the following recommendations: a. No more than 50 IU/kg per dose be administered unless observation will occur within a clinical trial. This relates to the uncertainty as to any potential incremental risk of an elevated FVIII level, even transiently, in patients on emicizumab who may need treatment with a BPA for breakthrough bleeding during ITI. b. Data on the use of emicizumab prophylaxis with ITI should be conducted under a clinical trial or as part of existing databases collecting data on the natural history of emicizumab use within the US.

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4. Other unresolved issues and potential new avenues of research a. MASAC recognizes the potential utility of emicizumab prophylaxis in patients with severe Type 3 von Willebrand disease with inhibitors to von Willebrand factor b. ATHN7 is a prospective observational study collecting data on the use of emicizumab within the US patient population and we strongly encourage that the availability of this study be discussed with patients who are prescribed emicizumab c. We encourage additional investigations to determine potential safe and efficacious dosing of aPCCs with concurrent emicizumab d. We encourage the continued accumulation of management of surgical experiences in patients prescribed emicizumab e. There is insufficient data on the level or protection provided by emicizumab prophylaxis to patients who participate in high impact activities/organized sports and MASAC encourages additional research to study this.


5. Additional patient education recommendations a. Revised ER and travel letters that include: i. information that the patient is on emicizumab prophylaxis ii. the potential impact on laboratory assays and acute bleed management iii. Contact information for the HTC b. Provision of dosing cards that detail the dose and frequency of FVIII concentrate or BPA for management of breakthrough bleeding c. Instructions on when to call the HTC d. Instructions on Travel i. Revised travel letter as detailed above ii. Need to travel with appropriate dose of FVIII concentrate or BPA agent to manage breakthrough bleeding

References: 1. https://www.gene.com/download/pdf/hemlibra_prescribing.pdf 2. https://www.emicizumabinfo.com 3. Lenting PJ, Denis CV and Christophe OD. Emicizumab, a bispecific antibody recognizing coagulation factors IX and X: how does it actually compare to factor VIII? Blood (2017) 130(23):2463-2468. 4. Young G, Callaghan M, Dunn A, Kruse-Jarres R and Pipe S. Emicizumab for hemophilia A with factor VIII inhibitors. Expert Rev Hematol (2018) 11(11):835-846. 5. Oldenburg J,Mahlangu JN,Kim B,Schmitt C,Callaghan MU,Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med (2017) 377(9):809-818. 6. Mahlangu J, Oldenburg J, Callaghan MU, Shima M, Santagostino E, Moore M, Recht M, Garcia C, Yang R, Lehle M, Macharia H, Asikanius E, Levy GG, Kruse-Jarres R. Bleeding events and safety outcomes in persons with haemophilia A with inhibitors: A prospective, multi-centre, non-interventional study. Haemophilia (2018) 7. Mahlangu J, Oldenburg J, Paz-Priel I, Negrier C, Niggli M, Mancuso ME, Schmitt C, JimĂŠnez-Yuste V, Kempton C, Dhalluin C, Callaghan MU, Bujan W, Shima M, Adamkewicz JI, Asikanius E, Levy GG, Kruse-Jarres R. Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors. N Engl J Med (2018) 379(9):811-822. 8. Chromogenic factor VIII with human reagents: https://www.aniara.com/PROD/a221402-ruo.html

BLOODLINES


Factor your way: empowering you to achieve your goals By Jorge Caicedo, Takeda

What is hemophilia? Hemophilia is a rare genetic bleeding disorder that prevents blood from clotting normally. The most common types of hemophilia are hemophilia A and B.1

400,000 individuals with hemophilia worldwide*

At the time of a bleed, proteins in your blood called clotting factors are switched on in a step-by-step fashion to form a clot and stop the bleeding (Figure 1).2, 3 However, if you have hemophilia, your blood lacks certain clotting factors (such as factors VIII and IX in the case of hemophilia A and B, respectively) and you may bleed for a longer time after an injury than you would otherwise.4, 5

Replacing what is missing with factor therapy The most common type of treatment for hemophilia is factor replacement therapy.7 Factor replacement therapy is the standard of care for hemophilia,7 with decades of real-world use as a proven treatment with an established safety profile.8, 9

Factor therapy replaces the missing blood-clotting proteins that are naturally found in your blood.10 Since it is administered via an intravenous injection i.e. directly into your bloodstream, the proteins are available immediately for use.10

Factor therapy can be given to control or treat an ongoing bleed (called on-demand therapy).6

Factor therapy can be administered regularly at fixed intervals to prevent further bleeds and complications (called prophylactic therapy).6

Patient without hemophilia: Your clotting factors work together in a ‘step-by-step’ process to stop bleeding.

Factor therapy can also be used to prevent and manage bleeds before and after surgery.7 Patient with hemophilia: In hemophilia, either factor VIII or IX is missing, the cascade is interrupted, and bleeding continues. FIGURE 1 The blood clotting cascade.

National Hemophilia Foundation 2015 *National Hemophilia Foundation 2015

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FIGURE 2 An individualized treatment approach to hemophilia.12

Other treatments for hemophilia More recently, the first non-factor therapy option became available for treating hemophilia A patients.11 It mimics part of the function of FVIII by bridging other factors in order to reduce bleeding. This therapy is administered subcutaneously and can also be used prophylactically.

Individualizing your treatment with factor Over the last 60 years there have been many advances in the treatment of hemophilia. Prophylaxis with factor has allowed many patients to reduce their bleeds and preserve long-term joint health.12, 13 It has allowed patients to pursue a lifestyle of their choice, and engage in a wide range of activities. While some patients are already achieving their desired treatment goals, there is an opportunity for more patients to achieve their goals by individualizing treatment to their unique needs.12 Individualized treatment takes into account many factors such as a person’s body weight, age, joint status, activity levels (sports, type of work, hobbies etc.), and bleeding frequency (Figure 2).12 When participating in sports, such as soccer, basketball or tennis, your risk of bleeding is increased; a factor activity level in your blood of 50% or higher is thought to reduce the risk of bleeds (factor activity levels over 50%

Age and weight

Joint status

Patient goals

Physical activity and lifestyle

Individualized treatment

Bleeding frequency

Pharmacokinetics

Genetics

are considered part of the normal range).12 Similarly, if you are due to undergo surgery, a factor level of 80% and above is recommended.14 By understanding how your body responds to factor therapy and with knowledge of your lifestyle and activity levels, your healthcare provider (HCP) can determine how much and how often you require factor and individualize your prophylaxis to meet your specific needs.12 Sticking to your prophylaxis will help you stay healthy and active; more importantly, it will help preserve your joints by significantly reducing bleeding.12

Individualizing treatment based on your own specific needs can help you to be adherent to your therapy so that you can continue doing the things you enjoy most.

What is pharmacokinetics? Pharmacokinetics (or PK) describe how fast or slow your body processes a medication.15 By measuring the levels of factor in your blood after an infusion your HCP is able to determine your unique PK profile.12 Your PK profile shows how much factor is available in your blood at various times after an infusion and allows your HCP to adjust the dose and timing(s) of your infusions to ensure that you have optimum coverage when you need it.12

BLOODLINES


Individualizing factor therapy can make a world of difference

CASE STUDY

Josh

Age 15 years Josh is an active adolescent who enjoys sports. Josh is currently on every-other-day Factor VIII dosing (25 IU/kg) but has recently been experiencing an increased number of bleeds, particularly in his ankles. When he visited his HCP, he was asked whether anything had changed. Josh said that as he wants to try out for his school tennis team he has been practicing 3 nights a week in an effort to improve his game. Prior to this Josh was practicing once on Friday and occasionally on the weekends. Based on these discussions, Josh’s hematologist realized that due to an increase in Josh’s practice schedule, Josh may not always be receiving optimal coverage from his current dosing regimen. Josh’s hematologist also noted that Josh had grown considerably since his last visit and recommended adjusting Josh’s dose of Factor VIII and changing his infusion schedule to better reflect his new practice times.

Since switching to the new dosing schedule, Josh’s bleeding has been reduced even with his increased activity. 35 IU/kg

35 IU/kg

35 IU/kg

FACTOR LEVEL

100%

50%

25%

0% AM

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FIGURE 3 Dosing schedule: three times a week Factor VIII (35 IU/kg).

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Together Josh and his hematologist reviewed Josh’s blood factor levels; they took a few blood samples and were able to determine what Josh’s levels would be at various time points following an infusion. Based on this information, Josh and his doctor agreed to an individualized dosing schedule that was acceptable to both Josh and his parents and one that was appropriate for Josh’s current activity level. Josh’s new dose is 35 IU/kg three times a week (Figure 3).


Individualizing factor therapy can make a world of difference

CASE STUDY

Chip

Age 35 years

Extended half-life: Extended half-life factor or EHLs are designed to work longer than standard half-life factor after they have been infused, thus requiring less frequent infusions.16

Chip is an IT consultant. His weekdays are fairly busy. A couple of nights a week, he plays pick-up basketball with his friends. He tries to run at least once during the weekend.

Chip has been on his new treatment regimen for 2 months and so far, has not had a bleed and is happy with his infusion regimen. 40 IU/kg

Target Joint: A joint that has had frequent recurrent bleeding episodes.17

40 IU/kg

100%

FACTOR LEVEL

Chip is currently on a three-times-aweek Factor VIII dosing (30 IU/kg). He would like to reduce his frequency of infusions, without increasing the risk of bleeding while maintaining his current lifestyle. During his college days, Chip was not adherent to his prophylaxis regimen and developed a target joint as a result. However, with better adherence to his prophylaxis regimen, Chip has experienced fewer bleeds in the target joint, which has since reversed. During his annual visit, Chip and his hematologist reviewed his PK profile. Based on his blood factor levels, Chip’s hematologist suggested he consider a longer-acting factor therapy (called extended half-life Factor VIII). Chip’s new dose is 40 IU/kg twice a week (Figure 4); he is able to plan the time of his infusions such that he gets optimum coverage when he most needs it.

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FIGURE 4 Dosing schedule: twice a week EHL Factor VIII (40 IU/kg).

Supporting literature: 1. Livnat T, et al. Blood Cells Mol Dis. 2017;67:63–68; 2. Smith SA1, Travers RJ1, Morrissey JH1. Crit Rev Biochem Mol Biol. 2015;50(4):326–336; 3. Berg JM, Tymoczko JL, Stryer L. Biochemistry. 5th edition. New York: W H Freeman; 2002; 4. Hemophilia | National Heart, Lung, and Blood Institute (NHLBI). Available at: https://www.nhlbi.nih.gov/health-topics/hemophilia. Accessed March 21st 2019; 5. Hemophilia. Available at: https://www.mayoclinic.org/diseases-conditions/ hemophilia/symptoms-causes/syc-20373327. Accessed March 21st 2019; 6. Peyvandi F, Garagiola I, Young G. Lancet. 2016;9(388):187–97; 7. Rodriguez-Merchan E. Arch Bone Jt Surg. 2018; 6(3): 157–160; 8. Pipe SW. Hematology Am Soc Hematol Educ Program. 2016;2016(1):650–656;

9. Franchini M. Expert Rev Hematol. 2014;7(5):573–581; 10. Erik B. Rolstad. Am J Mens Health. 2015;9(6):486–495; 11. MASAC Document #255. National Hemophilia Foundation. Available at: www.hemophilia.org; Accessed March 2019; 12. Valentino LA. Haemophilia. 2014;20:607–615; 13. Manco-Johnson MJ, Soucie JM, Gill JC. Blood. 2017;129(17):2368–2374; 14. Farrkh A, et al. Gen Dent. 2016;64(4):14–17; 15. Pharmacokinetics definition. Available at: https://en.oxforddictionaries.com/ definition/pharmacokinetics. Accessed April 5 2019. 16. Weyand AC, Pipe SW. Blood. 2019;133(5):389–398; 17. Mulder K, Llinás A. Haemophilia. 2004;10(Suppl 4):152–156.

Commitment to our community is always a priority for Takeda Hematology (previously part of Shire and Baxalta). As a leader in hemophilia research, Takeda continues to innovate on your behalf, developing programs and services that support your efforts each step of the way. Takeda is focused on providing advanced hematology treatments for today and innovating for the future. Copyright © 2019 Takeda Pharmaceutical Company Limited. All rights reserved. All trademarks are the property of their respective owners. S46630 4/2019

BLOODLINES


Your Identity,

Your Child’s

Identity A

s a therapist, I often talk to my clients about how they view themselves, their identity. This topic often leads to more discussion of a related topic, selfesteem. Identity, as well as self-esteem, can be big issues for those living with bleeding disorders, as well as their parents. Let’s start with a definition of both of these terms. Your identity is basically who you think you are, how you define yourself or, in therapy terms, how you perceive yourself. As you can imagine, how you self-identify has a big impact on how you feel emotionally and how you relate to other people. And as you have most likely experienced, identity as a person with a bleeding disorder can come down to a struggle between identifying as “normal” and identifying as a “patient.” Now for a quick definition of self-esteem. Self-esteem basically comes down to the value you place on yourself. As such, your self-esteem also has a whole lot to do with how you feel and how you interact with others. Self-esteem is an issue for those with bleeding disorders. The development of both identity and self-esteem begins during childhood. Yet another reason why a child living with a bleeding disorder might find themselves especially challenged as they forge their self-identity, well into adulthood. To get the conversation started, I will give you a couple of examples from my own work.

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by Dr. Gary McClain, PhD

FAMILY MATTERS


Who am I? What am I? Bill walked into the lunchroom at work and saw a few of his co-workers sitting at a table together. He bought a sandwich and sat down with them. They were laughing and talking about the World Series, which was going to start the next week. Bill was just about to jump into the conversation when one of his co-workers said: “Bill, you look a little more tired than usual. How are you feeling?” “I’m fine,” Bill answered. He tried not to act too annoyed. “I was just about to tell you who I think has the best chance to win this weekend.” And then he did. That night, he told his wife, Maryann, about what had happened. “Do I have bleeding disorder tattooed on my forehead? And what makes them think I’m tired?” he asked. “Gosh, Maryann, I am holding up my end at the job. So why can’t they get that I live my life pretty much like they do? I’m not an invalid.” Tommy is thirteen and in junior high. He has been pressing his parents to let him participate in sports. They have discouraged him, out of concern that constant bumps and falls will lead to bleeds. Last week, Tommy pushed

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harder, and told them he was planning to try out for a team. His parents told him that under no conditions would they allow him to participate in sports. “I can’t stand this,” Tommy said to them. “I’m just trying to be like everybody else and have some fun and you are treating like I’m sick or weak or something. Are you going to wrap me in bubble wrap so that the other guys know I can’t handle myself?” Olivia’s parents had a similar experience. She is in the fifth grade, and recently had a bleeding episode at school and had to miss a few days. When she returned, some of the other kids had questions that weren’t comfortable for her to answer. A few weren’t so kind. “My teacher asked me in front of everybody if I wanted to stay in during recess. Now everybody thinks I am sick or something. I just want to be normal like everybody else.” And let’s not forget that parents also struggle with questions about identity. First, they are often concerned about their children, whether as parents they are doing everything they need to do to help their children have a healthy sense of themselves, to feel capable and empowered, and not to adopt an identity as a “patient.”


It is also human nature for the parent of a child with a bleeding disorder to have questions about their own identities. To view also themselves as “normal” parents. With a day-to-day life that is similar to other families around them, sharing the same financial concerns, routines, goals and dreams, able to take an active role at school and in their communities. Yet, as parents of a child with a bleeding disorder, they have unique concerns and responsibilities that cause their lives to be very different in many ways. And as I stated earlier, how you think of yourself has a direct connection to how you feel about yourself. Again, the relationship between identity and self-esteem. Having a strong sense of our own identity is a task for adults, and parents, as well as children.

Maintaining Your Identify Here are some ideas for exercising your own identity muscles:

Review your own foundation Most likely, you have days when you ask yourself questions like “Do I know what the heck I’m supposed to be doing?” and “Am I any good at all at wearing all these hats?” This might be a good time to do a self-inventory. Think about: Your support network. Your healthcare team. Your ability to make good decisions when you’re faced with a challenge. Your inner resilience. The circumstances of life don’t have to lead to an identity crisis when you have a strong foundation. Your foundation is what’s real. It’s what you have to fall back on if things don’t go the way you want them to. Also keep in mind: If you are a parent, having a clear connection to your own foundation helps you to be there for your children, and also sets an empowered example for them.

Get Support Get connected with people who can listen without judging you or trying to tell you how you should feel. Including other people in the bleeding disorder community. Share your experiences. If you’re feeling like your identity is increasingly shaky, you might also want to schedule some time with a mental health professional to talk it out and learn some new coping techniques.

Look for the Lesson When something doesn’t go as you had hoped, look at this as an opportunity to learn. Learning experiences can include how you felt, and coped, with insensitivity at work, like Bill did, or the experiences of Olivia and Tommy and their parents. Remember the lesson so you can apply it in the future. Experience is the best teacher. While you’re at it, be willing to be the first to apologize.

Love Yourself Everyday Show yourself some kindness. Make sure you are building something you enjoy into each day. Along with people to enjoy it with. Remind yourself of what’s good in your life. Give yourself some words of encouragement.

Have a Sense of Humor It helps to be able to turn a frustrating situation into a humorous one. You can do this by being willing to look beyond your initial reactions and find the humor. For example, with a smile, Bill might have said to his co-workers: “Bring the stretcher. While I recover, I’ll give you all lots of moral support while you finish up the work.” And then, he could have changed the subject back to the world series. Humor is empowering!

FAMILY MATTERS


Give Yourself a Break Ask that voice of kindness for a little pep talk. Remind yourself: “I’m human. I’m trying hard. This is not an easy road. And I’m doing the best I can, even if not everything I do is perfect.” Having trouble conjuring up that voice? It might help to sit down with a sheet of paper and do some journaling. Give yourself a positive self-talk script you can read when you need a lift. Try some affirmations. Start out with: “I am a work in progress. I get better every day.” While you’re at it, why not give your self-esteem a boost?

Beware: Compare and Despair It’s only human nature to compare ourselves to others. But when we do that, we set ourselves up to come up short. There’s always somebody who, at least on the surface, seems to be doing a little better than you are. Sure, you’re living with a bleeding disorder and they’re not. And yes, absolutely, life is not fair. We’re all on our own unique path in life. Focus your attention on your path, that’s the way to success.

Helping Your Child to Have a Positive Identity Parents can have a profound impact on the development of their children’s identity. Yes, I know this sounds like a pretty big job. Here are some ideas to help you:

Encourage Involvement in Decision-Making Children with bleeding disorders often feel as if they are bound by limitations, which can contribute to the feeling they are being labeled a patient and not a whole person. Provide your child with a sense of control, whenever possible by, allowing for some choices in daily routines, in diet, and in self-care. Encourage shared decision making. Explain the options and the boundaries, share information, and listen to your child’s concerns and preferences. Doing this will help your child to integrate their bleeding disorder self-care into their daily life, in a way that supports your child’s other self-determined interests and priorities. Knowledge is power!

Teach All Children to Be Advocates Children with bleeding disorders need to learn to speak up for themselves so that teachers and other adults outside the home are aware of any needs and limitations. They also need to learn to deal with the questions and comments that will inevitably come their way, as do their brothers and sisters. Teach your chronically-ill child how to be a self-advocate through role-playing at

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home. By doing this, you are also teaching your child to make it clear to others who they are as people and how they want to be thought of. How’s that for an identity builder? Being an advocate reinforces your child’s self-esteem.

Remember that Not Everything is a Medical Issue Children are human beings, not medical conditions, and communications don’t all have to revolve around your child’s chronic condition. Lots of the issues that come up with kids and teens are developmental. Maintain your perspective on your child as a whole person. Avoid falling into the trap of assuming that everything that happens to them is somehow


connected to their bleeding disorder. For example, all teens are in the process of figuring themselves out, and the struggle for independence is something all families with teens are coping with. While you’re at it, don’t allow other adults your child comes into contact with, at school, in the community, in your extended family, to reduce them to a walking and talking bleeding disorder. Creating a positive identity is a daily process.

Avoid the Urge to Catastrophize Let’s face it, you’re a parent. So, it is only natural that when something happens to your child, you feel like it is also happening to you. But what may seem devastating and alarming to you may be something that your child is taking in stride. After all, they are living day to day with their bleeding disorder. So, try to take emotional cues from your child. Sure, be diligent in working with your child to manage their bleeding disorder. As the saying goes, slow and steady wins the race. Set an example for your child by not keeping your finger poised over the panic button.

Encourage Your Child to Do a Self-Inventory In other words, to review their own foundation. Frequently. Sit down together and discuss their accomplishments, skills, the qualities they most value about themselves, the qualities that others value. Make a list together and then pull it out when they have those moments when they aren’t feeling all that great about themselves. This is a great way to help your child take ownership of their identity and understand the connection between taking positive steps toward forming their own personal identity. Keep in mind that self-esteem comes from within, from believing in yourself. So, review the evidence together often. This exercise is, by extension, also a selfesteem builder.

Encourage your child to have a sense of humor Teach them that both kids and adults say things that are not very well thought out, mainly because they aren’t educated about bleeding disorders. And that sometimes the best response is just to reply to a dumb comment with a joke. Olivia could have informed her classmates: “I got bit by a shark. I need to be more careful while swimming.” Again, humor is empowering.

Also talk to your kids about comparing and despairing Kids are in a constant state of comparing themselves to their peers. Help your child to recognize what’s unique about themselves. Sure, their condition sets them apart, but so do other qualities. Teach them that each of us are on our own path, with our own challenges and opportunities. Their peers have challenges, too. Your identity. Your child’s identity. Stay focused on the big picture of life. Embrace all of who you are. If you are a parent, encourage your child to do the same. Avoid labels, especially the “patient” label. Think of your bleeding disorder as a small wedge of a very big pie, that is, only one aspect of your identity. One aspect of your child’s identity. And one aspect of your identity as a parent. We’re all in this together!

Gary McClain, PhD, is a therapist, patient advocate, and author, specializing in helping clients deal with the emotional impact of chronic and life-threatening illnesses, as well as their families and professional caregivers. He works with them to understand and cope with their emotions, to learn about their lifestyle and treatment options, to maintain compliance with medical regimens, to communicate effectively with the medical establishment, to communicate better with other family members, and to listen to their own inner voice as they make decisions about the future. He writes articles for healthcare publications and websites, facilitates discussions in social health communities, and conducts workshops on living with chronic conditions, Chronic Communication Skillssm. He maintains a Website, www.JustGotDiagnosed.com.

FAMILY MATTERS


TRANSITIONING

from

High School t o

College or Work

M

ost transitions can cause stress but transitioning from high school to college can be a scary one! In high school, the laws require that staff identify a student with a disability. Higher education is not required to identify students with disabilities. At the college level, it falls upon the student to be their own advocate. This may be difficult for some parents who have been used to being their childâ&#x20AC;&#x2122;s advocate for 17+ years! Because of privacy laws (FERPA), parents do not have access to their childrenâ&#x20AC;&#x2122;s records or are able to make decisions for their child. It is important that students with disabilities begin to advocate for themselves in high school so that they learn how to navigate difficult situations.

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by Lisa Cosseboom, M.Ed. & CAGS School Psychologist & Special Education Evaluation Team Chairperson

Spring/Summer 2019

The American with Disabilities Act (ADA), section 504 requires colleges who receive federal funding to provide equal access and reasonable accommodations to a student with a disability. Many private higher education institutions do not receive federal assistance and therefore do not necessarily have to offer a student with a disability, accommodations. However, even if a college is exempt from Section 504 requirements, a student may be covered under other disability rights legislation.


Here are some examples of accommodations that may be available at the college level: •

Use of note-takers for class lectures

Making audio recordings of lectures

Use of a laptop computer in the classroom

Taking exams in a distraction-reduced room

Accessible testing locations

Course substitutions

Accessible software

These accommodations are not automatically given to a student with a disability. The student must seek out and apply for reasonable accommodations through the college’s disability services department. A student does not need to disclose that they have a disability when applying to college. Once a student begins at a college, they will want to go to the Disability Services Center at the university. Be prepared to have supporting documentation of the disability. Each college has their own documentation requirements. Some

examples may include a high school 504 plan, doctors’ documentation etc. However, keep in mind that the documentation must be recent (generally within 3 years). It is highly recommended that families review colleges’ disability services prior to choosing which institution will best support their child’s educational future.

Vocational Rehabilitation for People with Disabilities The Federal Programs of Social Security Disability Insurance (SSDI) and Supplemental Security Income (SSI) offer many options for services. Each state receives funding from these federal programs that are distributed into state programs. If a person is already receiving either SSDI or SSI, they are automatically qualified to receive Vocational Rehabilitation (VR) services. Vocational Rehab services include preference evaluations, job training and job placement. The mission of VR is to assist an individual in receiving the support they need and guidance to access that support.

financial assistance while you are getting some vocational rehab services

transportation needed to get to some vocational rehab services

help transitioning from school to work (for students)

personal assistance services

rehabilitation technology services and devices

supported employment services, and

To qualify for Vocational Rehabilitation, an individual must have a physical or mental condition that causes a “substantial impediment” to work and be able to benefit from VR services to gain employment. Each state has their own processes and procedures. When researching, look into your state’s Department of Health and Human Services or Education Department.

help finding a job

a personal assessment of your disability(ies) to see if you are eligible and to determine how VR can help you

job counseling, guidance, and referral services

physical and mental rehabilitation

vocational (job) and other training

on-the-job training

In my home state, Massachusetts, Vocational Rehab services are administered under the Massachusetts Rehabilitation Commission (MRC). They explain that “Vocational Rehabilitation (VR) helps people with physical, psychiatric, and learning disabilities find and keep a job. VR helps you identify job goals based on your interests and skills and explore college and vocational training. It also reduces or removes barriers to employment. Priority is given to people who have the most severe disabilities in areas such as communication, mobility, work tolerance, and work skills.” Similar to applying for disability services in college, documentation including school records, medical history, evaluations etc. are required to become eligible for services.

Helpful links: http://www.askearn.org/state-vocational-rehabilitation-agencies/ https://www.ssa.gov/benefits/disability

WHAT’S the PLAN?


ATHN 7

Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Hemophilia

by Janet Brewer, M.Ed ClinicalTrials.gov identifier (NCT number): NCT03619863

T

he American Thrombosis and Hemostasis Network has initiated the Natural History Study for the purpose of following patients with Hemophilia A/B with or without inhibitors for a period of 4-6 years who receive their care from hemophilia treatment center physicians across the United States. The primary purpose of the study is to assess the safety of FDA-approved treatment products and their outcomes to include: replacement clotting factor products, bypassing agents and non-factor products. Treatment regiments will be at the discretion of the patient and their hemophilia caregivers. There will be no treatment products or compensation offered; inhibitor testing however, will be provided by the CDC at no cost to patients. Data collection will include eligibility, demographics, medical history, hemophilia history (genotype and family history), inhibitor history and immune tolerance induction (ITI) treatment regimen (if applicable), co-morbidities at baseline (i.e., HIV, Hepatitis C), detailed treatment product(s) usage, bleeding events, surgical procedures, and *EUHASS adverse events and other adverse events of special interest. Data collection will also include patientreported outcome questionnaires regarding health-related quality of life, treatment use and patient perceptions of treatment products. Patients will be seen at baseline, annually, and at study exit. Patients will also receive routine quarterly follow-up phone calls from HTC staff to review medical history, bleed events, and product treatment history. A number of sub-studies are planned with pharmaceutical sponsors to collect information from patients about their specific product use. Participation in these sub-studies (Product Specific Modules) is optional and sub-study visits will be planned to coincide with HTC visits. The modules will collect information from patients about their perception and use of treatment products, physical activity levels and other general health questions. This data will be collected via questionnaire. (https://clinicaltrials.gov/ct2/show/NCT03619863)

Secondary outcome measures will include: • Effectiveness of non-factor products, bypassing agents and clotting factor replacement products based on the participant’s number of bleeding events and/or annualized bleed rate (ABR). • Dosing regimens for hemophilia treatment products and total amount utilized for prophylaxis and treatment of bleeds. •

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Target joint monitoring


CRITERIA

Real world effectiveness of treatment products as measured by the number and types of medical visits and/or hospitalizations per year. The study is currently open to 280 participants in 38 ATHN-affiliated HTCs. Patients will be seen at baseline, annually and at study exit. Criteria: •

No age restrictions

Open to men and women

Inclusion Criteria: 1. Congenital hemophilia A or B of any severity with or without inhibitors, receiving a current therapy, a non-factor product, or for whom use of a non-factor product is a possibility; 2. Able to give informed consent (by patient or parent/ authorized guardian); and 3. Co-enrollment in the ATHN dataset

Exclusion Criteria: 1. Presence of any known bleeding disorder other than congenital hemophilia A or B; 2. Presence of concurrent hemophilia and a second hemostatic defect (low Von Willebrand Factor (VWF) without Von Willebrand disease (VWD) diagnosis is not excluded); and 3. Unable or unwilling to comply with the study protocol

Contact: Angela Riedel - Ph: 309.361.4375, ariedel@athn.org Primary Investigators are: Tyler Buckner, MD, MSc Hemophilia and Thrombosis Center/ University of Colorado Anschutz Medical Campus

Nikki Hirsh, MS, CCRC - nhirsh@athn.org Michael Recht, MD, PhD The Hemophilia Center at Oregon Health & Science University

For a list of the 38 study locations please refer to: https://clinicaltrials.gov/ct2/show/study/NCT03619863?show_locs=Y - locn *EUHASS adverse events include: : death, factor inhibitor development, venous thrombosis, allergic reactions, treatmentemergent side effects, new malignancies, cardiovascular events, and blood-borne infections. Other treatment-related events to be documented on the ATHN Adverse Event Module Form including thrombotic microangiopathies, injection site reactions, drug-induced liver injury and anti-drug antibodies.

WHAT’S NEW?


Advocacy to Protect

by James Romano Dir. of Government Relations

PATIENT ASSISTANCE

S

ince the passage of the Patient Protection and Affordable Care Act (Public Law, 111-148) the pendulum has swung on the perception of patient assistance programs. Once a beacon of hope in the social safety net, governmental sympathy and stakeholder interest has largely vanished. Today the conventional wisdom for these programs have oscillated between the false theory that these programs are somehow no longer necessary or needed to the dangerous notion that patient assistance actually hurts patients. Health Insurance Providers in their quest to reduce their costs and maximize profits have influenced and benefited from the pendulum shift. The health insurance industry will not be satisfied until as many patients with rare disorders and chronic conditions are forcibly transitioned onto public programs such as Medicare and Medicaid.

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Patient Services Incorporated has met the challenge laid down by the much larger health insurance industry in an attempt to protect the patient assistance model that the organization founded 30 years ago. PSI is a national nonprofit patient assistance organization that raises private donations into disorder specific programs. PSI utilizes those donations to provide financial assistance to patients to subsidize the high cost of health insurance premiums; out of pocket expenses such as deductibles, copayments and coinsurance; as well as ancillary supplies and travel expenses. PSI also helps patients to obtain their disability benefits for certain conditions through our ACCESS Program. This Pendulum shift first manifested itself approximately 5 years ago in 2014 with the rule promulgated by the Centers for Medicare and Medicaid Services (CMS) entitled: Patient Protection and Affordable Care Act: Third Party Payment of Qualified Health Plan Premiums. This rule mandated that health insurance providers in the state and federal insurance marketplaces accept insurance premium and cost sharing assistance from State Ryan White/HIV Programs; Indian Tribes & Tribal Organizations; as well as any other government program. The rule also states that health insurance providers can impose contractual prohibitions onto any other entities such as nonprofit organizations like PSI. This rule serves to undermine the reform of the pre-existing condition exclusion included in the Affordable Care Act because this policy disproportionately affects those with rare

and chronic illnesses who rely on these programs to obtain needed treatments and therapies while maintaining their way of life.

Almost immediately PSI was contacted by plans informing our charitable assistance organization that the plan would no longer accept our assistance. Since the release of the rule over 100 plans in 43 states have implemented the prohibition. Insurance providers have gone to the extremes while implementing this prohibition including threatening patients with litigation to recoup claims and forcing patients to sign attestations under the penalty of perjury that the patient receives no outside support in paying their premium. The target of this nefarious rule are people with rare and chronic conditions living their lives as best they can while Health Insurance providers are developing ways to prevent them from

WHATâ&#x20AC;&#x2122;S NEW?


obtaining the needed treatments. Even though PSI has fought to modify or overturn the rule, the insurance industry has been emboldened in their actions and have attempted to spread the prohibition into other insurance coverage markets including the Medicare Supplement/ Medigap market as well as Non-ACA individual market. Besides the CMS Rule, health insurance providers have looked for other creative ways to limit access to specialty treatments for patients again placing profits above lives. The implementation of Copayment Accumulators; Fail First policies and prohibitions on copayment cards by manufacturers are examples of this creativity to push more

costs onto patients. These initiatives have one common thread running through themâ&#x20AC;&#x201D;ensuring that patients must jump through more hoops to obtain their treatment or worse preventing the access of treatments all together. PSI has stepped forward to defend the patient assistance model we created. The mission of PSI in the advocacy field has been to advocate for government policies at the state and federal level that will end these egregious practices and ensure easier access for patients living with rare and chronic illnesses. PSI was the first organization to recognize the threat the CMS rule would pose to patient assistance. PSI has led the

Let charities be charitable!

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advocacy efforts over the last five years to overturn this rule. PSI created the Marketplace Access Project Coalition to bring together like-minded patient advocacy groups to support legislative efforts to help patients utilize these programs. PSI was successful in developing the Access to Marketplace Insurance Act (HR 3742 & HR 3976), a bill that has been introduced in the last two Congresses by Kevin Cramer (R-ND). Congressman, now Senator, Cramer recognized the need to strengthen the social safety net not diminish it and coined our battle cry—Let Charities be Charitable. This legislation would amend the Affordable Care Act and add three more entities to the list that CMS established through the rule. Health insurance providers must accept premium and cost sharing assistance from the entities stated in the rule but also three additional entities-- Non-Profit Organizations; Places of Worship and Civic Organizations. The legislation had strong bipartisan support and garnered 147 and 177 cosponsors respectively in Congress. However, the health insurance plans worked overtime to prevent the legislation from passing. Since 2012, PSI has hosted our yearly advocacy fly-in to promote the model and assistance programs. PSI brings patients from all over the country to advocate with their Members of Congress and Senators on the importance of the patient assistance they receive and what would happen if that assistance was limited or even prohibited. To continue to advocate for assistance for patients, PSI is partnering with other entities such as the Hereditary Angioedema Association; the Pulmonary Hypertension Association and Good Days to form the coalition—United for Charitable Assistance. UCA will work on the state and federal levels to promote and protect charitable assistance for patients for many years to come. PSI has worked to understand why CMS has promulgated such a rule. CMS has stated that they are concerned that

charities would flood the plans with sick patients—contrary to the stated goals of the Affordable Care Act. PSI has worked with Congress through the House and Senate Appropriations Committees to include report language in the FY 2016 and FY 2017 Joint Explanatory Statements, asking the agency to explain to Congress its reasoning for excluding nonprofit organizations from the listed entities. CMS ignored the request for information from Congress and never provided any explanation. Congress continued to press the issue sending at least six sign-on letters including the signatures of hundreds of Members of Congress asking for CMS to modify the rule. To this moment CMS has ignored Congress and has ignored the calls from the patient advocacy community to fix this rule. PSI will continue its leadership in passing the Access to Marketplace Insurance Act as well as promoting charitable assistance in Congress and in state legislatures. If you would like to become involved, please contact the PSI Government Relations team at Advocacy@uneedpsi.org. Since the authorship of this article, we have the following updates to add. In March 2019, Virginia and West Virginia became the first two (2) states to ban copay accumulators. Several states, AZ, CT, IN, KY and RI, have pending state legislation to ban as well! On April 18th, CMS released their Notice of Benefit and Payment Parameters Rule for 2020, which limits use of brand name drugs that have equally effective generics. Because factor does not have a generic alternative, it should be exempt from the Accumulator Adjuster. https://www.cms.gov/newsroom/press-releases/cmsissues-final-rule-2020-annual-notice-benefit-and-paymentparameters

Editor’s note: Please view the PSI You Tube Accumulator Adjustor video on this topic: https://video.search.yahoo.com/yhs/search?fr=yhs-sz-002&hsimp=yhs-002&hsp art=sz&p=PSI+you+tube+accumulator+adjustor - id=1&vid=563fcff548e5a6ed0b 912afc8cc8247f&action=click or NHF’s YouTube video https://www.hemophilia.org/Newsroom/Advocacy-Legislative-News/CopayAccumulator-Adjustments-What-are-they-and-how-they-can-affect-you

WHAT’S NEW?


How

Mindfulness Keeps Us in the MOMENT

by Krystyn Strother

H

ave you ever heard the saying “don’t let the future steal your present,”? Consider it for a moment. This statement holds so much potency. Many of us could benefit from repeating these words as it shows the importance and power of mindfulness. If you have never heard of mindfulness before, it is the practice of being present in the moment.

The Importance of Staying Present in the Moment Simple in concept, more challenging in practice, mindfulness allows us access to live in the moment, and the tools to cope with those moments when they may be undesirable. When we spend our time dwelling on the past or worrying about the future we cultivate anxiety, worry, stress, and fear. These emotions can contribute to problems with sleep, immunity, and mental health, to list just a few. One negative thought typically triggers another and another and so on, initiating a domino effect that can lead you into a spiral of thoughts. One way to keep those thoughts at bay is through mindfulness. Mindfulness allows you the opportunity to quickly recognize these thoughts and anchor to the present moment. Worry is generally about the future, sometimes the past, and it is near impossible to be anxious or worried when your mind is focused on what is simply happening in the present moment.

“Don’t let the future steal your present.” Navigating the What If’s Go With the What Is. When you or your child has a chronic illness, it may seem impossible to not worry about what the future holds. When the mind isn’t dwelling on the future it can often get stuck in the past, ruminating over past mistakes. The what if’s of the past and future Keep us

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disconnected from the present. We can’t change the past, so why live in it? There are no guarantees for the future so why jump to conclusions? Of course, it is human nature and wise to plan for the future. It is also smart to learn from our past mistakes. However, it is irrational and potentially detrimental to worry about that over which we have no control – e.g., the past and the future. Living in the moment allows us to be present, mindful, and experience the passing of time. Whatever emotion or thought or physical sensation you are experiencing, whether positive or negative, over time, has to pass.

Practical Tips for Everyday Mindfulness Pay attention. It’s hard to slow down and notice things in a busy world. Try to take the time to experience your environment with all of your senses — touch, sound, sight, smell and taste. For example, when you eat a favorite food, take the time to smell, taste and truly enjoy it. Live in the moment. Try to intentionally bring an open, accepting and discerning attention to everything you do. Find joy in simple pleasures.

When the reminder goes off, stop whatever you are doing for thirty seconds and focus your awareness on the present moment. What is happening right then? What is the temperature? How are you breathing? What do you taste? Check in with yourself and ask yourself about how you are feeling emotionally and physically, and what you are thinking about. Mindfulness is all about being accepting, non-critical and open, so be kind to yourself and curious about your experience. How do you attain mindfulness? There is no definitive “achievement” of mindfulness, especially when the essence of it is to empty your mind. Mindfulness is just a state of being. Use the times when you are feeling worried or anxious as an opportunity to return to the present moment. Remind yourself that regardless of what happens in the future, you will be able to handle it. After all, you always have been able to handle whatever life has thrown at you. You are resilient. You are strong. You are capable.

Photo courtesy of krystynstrother.com and Stark Photography

Accept yourself. Treat yourself the way you would treat a good friend. Focus on your breathing. When you have negative thoughts, try to sit down, take a deep breath and close your eyes. Focus on your breath as it moves in and out of your body. Sitting and breathing for even just a minute can help. Some of the same things that can quickly disconnect us from the present moment can also serve as a reminder for you to return to the moment. Make your mobile phone or computer your mindfulness machine by setting reminders that go off through the day to encourage you to notice the moment.

Krystyn Strother is the former program director at HUSH Meditation, strategic designer/author of the HUSH meditation curriculum, is a certified meditation instructor, co-founder of NOMAD, “Adventures in Wellness”, and yoga instructor. Krystyn’s yoga classes range from Vinyasa to Yin. In addition to her regularly scheduled classes, Krystyn guest teaches at several yoga teacher training programs throughout the country, speaks at conferences on mindfulness and stress reduction practices, teaches specialized workshops, facilitates yoga + adventure retreats, and conducts continuing education classes for currently registered RYTs. Krystyn holds a certificate of completion in the Yoga of Awareness For Chronic Pain, an evidence-based program sponsored by the Department of Anesthesiology at OHSU. Read more about Krystyn at krystynstrother.com

MIND BODY CONNECTION


UNLOCKING YOUR SELF-POTENTIAL ONLY ADVATE® HAS 15 YEARS OF EXPERIENCE IN THE REAL WORLD AS A RECOMBINANT FACTOR VIII1 • Proven in a pivotal clinical trial to reduce the number of bleeding episodes in children and adults when used prophylactically 2* • Third-generation full-length molecule, similar to the factor VIII that occurs naturally in the body1,2 *Multicenter, open-label, prospective, randomized, 2-arm controlled trial of 53 previously treated patients with severe to moderately severe hemophilia A. Two different ADVATE prophylaxis regimens (standard, 20-40 IU/kg every 48 hours, or pharmacokinetic-driven, 20-80 IU/kg every 72 hours) were compared with on-demand treatment. Patients underwent 6 months of on-demand treatment before 12 months of prophylaxis.2

The market leader in hemophilia A treatment† † Based

on 2016 data published July 2017.3

ADVATE Important Information What is ADVATE?

• ADVATE is a medicine used to replace clotting factor (factor VIII or antihemophilic factor) that is missing in people with hemophilia A (also called “classic” hemophilia). • ADVATE is used to prevent and control bleeding in adults and children (0-16 years) with hemophilia A. Your healthcare provider (HCP) may give you ADVATE when you have surgery. • ADVATE can reduce the number of bleeding episodes in adults and children (0-16 years) when used regularly (prophylaxis). ADVATE is not used to treat von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION Who should not use ADVATE?

Do not use ADVATE if you: • Are allergic to mice or hamsters. • Are allergic to any ingredients in ADVATE. Tell your HCP if you are pregnant or breastfeeding because ADVATE may not be right for you.

What should I tell my HCP before using ADVATE?

Tell your HCP if you: • Have or have had any medical problems. • Take any medicines, including prescription and non-prescription medicines, such as over-the-counter medicines, supplements or herbal remedies. • Have any allergies, including allergies to mice or hamsters. • Are breastfeeding. It is not known if ADVATE passes into your milk and if it can harm your baby. • Are or become pregnant. It is not known if ADVATE may harm your unborn baby. • Have been told that you have inhibitors to factor VIII (because ADVATE may not work for you).

What important information do I need to know about ADVATE?

• You can have an allergic reaction to ADVATE. Call your HCP right away and stop treatment if you get a rash or hives, itching, tightness of the throat, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea or fainting. • Do not attempt to infuse yourself with ADVATE unless you have been taught by your HCP or hemophilia center.

What else should I know about ADVATE and Hemophilia A?

• Your body may form inhibitors to factor VIII. An inhibitor is part of the body’s normal defense system. If you form inhibitors, it may stop ADVATE from working properly. Talk with your HCP to make sure you are carefully monitored with blood tests for the development of inhibitors to factor VIII.

©2019 Shire US Inc., Lexington, MA 02421. All rights reserved. 1-800-828-2088. SHIRE and the Shire Logo are registered trademarks of Shire Pharmaceutical Holdings Ireland Limited or its affiliates. ADVATE and MYPKFIT and Logo are trademarks or registered trademarks of Baxalta Incorporated, a wholly owned, indirect subsidiary of Shire plc. S41004 02/19

The myPKFiTTM for Patients Mobile Application offers visibility into your personalized ADVATE treatment. Talk to your doctor to see if myPKFiT for ADVATE may be right for you. Learn more at ADVATE.com. What are possible side effects of ADVATE?

• Side effects that have been reported with ADVATE include: cough, headache, joint swelling/aching, sore throat, fever, itching, unusual taste, dizziness, hematoma, abdominal pain, hot flashes, swelling of legs, diarrhea, chills, runny nose/congestion, nausea/vomiting, sweating, and rash. Tell your HCP about any side effects that bother you or do not go away or if your bleeding does not stop after taking ADVATE.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. For additional safety information, please see Important Facts about ADVATE on the following page and discuss with your HCP. For Full Prescribing Information, visit www.ADVATE.com

myPKFIT for ADVATE Patients Mobile Application Intended Use • The myPKFiT for Patients Mobile Application (“myPKFiT Mobile App”) is intended for use by patients with hemophilia A being treated with ADVATE [Antihemophilic Factor (Recombinant)] who are 16 years of age or older with a body weight of 45 kg or higher, and their caregivers. • The myPKFiT Mobile App is designed to make it convenient for you to record your infusion and bleed events, track your estimated Factor VIII levels following a prophylactic infusion, and export the data for review by your health care provider (“HCP”). • Your HCP can use the myPKFiT software to generate ADVATE dosage amount and frequency recommendations for routine prophylaxis using your age, body weight information, and laboratory tests that measure your Factor VIII clotting activity. Using myPKFiT software, HCPs can evaluate various prophylaxis dose regimens tailored to your individual needs and treatment plan. • myPKFiT Mobile App should only be used by hemophilia A patients treated with ADVATE, as per the ADVATE Prescribing Information. • myPKFiT Mobile App is not indicated for use by patients with von Willebrand disease. myPKFiT Mobile App should not be used by patients who have developed inhibitors to Factor VIII products. myPKFiT for Patients Mobile Application is Rx only. For safe and proper use of the myPKFiT Mobile App, please refer to the complete instructions for use in the User Manual. References: 1. Grillberger L, Kreil TR, Nasr S, Reiter M. Emerging trends in plasma-free manufacturing of recombinant protein therapeutics expressed in mammalian cells. Biotechnol J. 2009;4(2):186-201. 2. ADVATE Prescribing Information. 3. The Marketing Research Bureau, Inc. The plasma proteins market in the United States. 2016. 4. myPKFiT Mobile Application v2.0 User Manual. 2018.


What should I tell my healthcare provider before I use ADVATE?

Important facts about ADVATE [Antihemophilic Factor (Recombinant)] This leaflet summarizes important information about ADVATE. Please read it carefully before using this medicine. This information does not take the place of talking with your healthcare provider, and it does not include all of the important information about ADVATE. If you have any questions after reading this, ask your healthcare provider.

You should tell your healthcare provider if you: • Have or have had any medical problems. • Take any medicines, including prescription and non-prescription medicines, such as over-the-counter medicines, supplements or herbal remedies. • Have any allergies, including allergies to mice or hamsters. • Are breastfeeding. It is not known if ADVATE passes into your milk and if it can harm your baby. • Are pregnant or planning to become pregnant. It is not known if ADVATE may harm your unborn baby. • Have been told that you have inhibitors to factor VIII (because ADVATE may not work for you).

What is the most important information I need to know about ADVATE?

What are the possible side effects of ADVATE?

Do not attempt to do an infusion to yourself unless you have been taught how by your healthcare provider or hemophilia center.

Call your healthcare provider right away and stop treatment if you get a rash or hives, itching, tightness of the throat, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea or fainting.

You must carefully follow your healthcare provider’s instructions regarding the dose and schedule for infusing ADVATE so that your treatment will work best for you. What is ADVATE? ADVATE is a medicine used to replace clotting factor (factor VIII or antihemophilic factor) that is missing in people with hemophilia A (also called “classic” hemophilia). The product does not contain plasma or albumin. Hemophilia A is an inherited bleeding disorder that prevents blood from clotting normally. ADVATE is used to prevent and control bleeding in adults and children (0-16 years) with hemophilia A. Your healthcare provider may give you ADVATE when you have surgery. ADVATE can reduce the number of bleeding episodes in adults and children (0-16 years) when used regularly (prophylaxis). ADVATE is not used to treat von Willebrand disease.

Who should not use ADVATE? You should not use ADVATE if you: • Are allergic to mice or hamsters. • Are allergic to any ingredients in ADVATE. Tell your healthcare provider if you are pregnant or breastfeeding because ADVATE may not be right for you. How should I use ADVATE? ADVATE is given directly into the bloodstream. You may infuse ADVATE at a hemophilia treatment center, at your healthcare provider’s office or in your home. You should be trained on how to do infusions by your healthcare provider or hemophilia treatment center. Many people with hemophilia A learn to infuse their ADVATE by themselves or with the help of a family member. Your healthcare provider will tell you how much ADVATE to use based on your weight, the severity of your hemophilia A, and where you are bleeding. You may have to have blood tests done after getting ADVATE to be sure that your blood level of factor VIII is high enough to clot your blood. Call your healthcare provider right away if your bleeding does not stop after taking ADVATE.

You can have an allergic reaction to ADVATE.

Side effects that have been reported with ADVATE include: cough sore throat unusual taste abdominal pain diarrhea nausea/vomiting

headache fever dizziness hot flashes chills sweating

joint swelling/aching itching hematoma swelling of legs runny nose/congestion rash

Tell your healthcare provider about any side effects that bother you or do not go away. These are not all the possible side effects with ADVATE. You can ask your healthcare provider for information that is written for healthcare professionals. What else should I know about ADVATE and Hemophilia A? Your body may form inhibitors to factor VIII. An inhibitor is part of the body’s normal defense system. If you form inhibitors, it may stop ADVATE from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to factor VIII. Medicines are sometimes prescribed for purposes other than those listed here. Do not use ADVATE for a condition for which it is not prescribed. Do not share ADVATE with other people, even if they have the same symptoms that you have. The risk information provided here is not comprehensive. To learn more, talk with your health care provider or pharmacist about ADVATE. The FDA approved product labeling can be found at www.ADVATE.com or 1-888-4-ADVATE. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. ©2017 Shire US Inc., Lexington, MA 02421. All rights reserved. 1-800-828-2088. SHIRE and the Shire Logo are registered trademarks of Shire Pharmaceutical Holdings Ireland Limited or its affiliates. ADVATE and BAXALTA are trademarks of Baxalta Incorporated, a wholly owned, indirect subsidiary of Shire plc. Baxalta US Inc. Westlake Village, CA 91362 USA U.S. License No. 2020 Issued: 11/2016 S32554 06/17


89 E. Washington Street Hanson, MA 02341-1125

CHES Mission To Inspire awareness and selfreliance for patients with chronic health conditions, their families, and their communities.

Editors in Chief Janet Brewer, M.Ed Eric Lowe

Editor Janet Brewer, M.Ed

Publication Designer Eric Lowe

Contributing Writers Janet Brewer, M.Ed Jorge Caicedo (Takeda) Amy Gaytan Lisa Cosseboom, M.Ed., C.A.G.S Eric Lowe Dr. Gary McClain, PhD James Romano Krystyn Strother Mark Zatyrka

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