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Summer 2018 I Volume 11

A CHES Publication

New Inhibitor Treatments: Weighing



PTSD: Is It Real for Inhibitor Families? How We Can Report an Adverse Event Clinical Trials: For Inhibitor Patients After the Shock: Inhibitor Camp for All The Access to Marketplace Insurance Act: Allowing Charities to be Charitable

In Memory of

Jane Cotter Forbes

Editors’ Note: We wish to thank Jane Cotter Forbes for her contribution of It’s Easy article (pg.18.) Sadly, on July 8, 2018 Jane suddenly passed away from a hemorrhagic stroke. She leaves her husband Ogden Michael Forbes and 6 beautiful children. As the co-founder of Helpful Hemophilia RNs on FaceBook, a prolific writer and tireless advocate for the hemophilia community, her absence will be profoundly missed.

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! We are thrilled to be celebrating our 5th year of LifeLines for Health! It has grown from a newsletter to a full-fledged magazine; and remains the only publication specific to the inhibitor community! Here at CHES, our hearts and commitment to those managing an active inhibitor has never been stronger. Our four (4) national programs in 2018 will explore topics such as ALL current methods of inhibitor management, insurance coverage of new products, grief, self-infusion, aqua therapy, and mindfulness and pain to mention a few. We will be offering a new program, After the Shock, an inhibitor family camp that will allow us to invite families younger than age six for the first time. This will be a CHES run program replacing Inhibitor Family Camp at Victory Junction. Our new 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 2018. Leverage and Momentum are scheduled for September and July respectively. 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. CHES’ mission is to inspire awareness and self-reliance for patients with chronic health conditions, their families, and their communities. Accuracy, empathy and integrity are the core principles of our company. Our programs are meant to augment the wonderful resources available and are as always, free of charge to attendees. There is no marketing or sales representatives at any inhibitor camp programs, just the way you like them! As individuals managing an inhibitor, we are constantly assessing treatments to manage an inhibitor. Genentech’s announcement for FDA approval and manufacturing for their new product, HemLibra in November, has taken our community by storm. Yet, 6 people have died and serious adverse events have been reported for individuals using this product. Many unanswered questions remain, including whether immune tolerance is possible while using this by-passing product. Most inhibitor experts agree, that tolerization remains the central goal for inhibitor eradication. Dr. Jonathan Bernstein from CCMD addresses new treatment risks and benefits for current and upcoming products. We begin a two-part series on PTSD in this edition. For so many of us, the reality of needle and procedure trauma, pain, and fear create an endless circle of anxiety and stress. PTSD is a very real diagnosis for those coping with acute medical challenges. The first step towards control is recognizing the symptoms and seeking professional assistance for not just the acutely affected individual, but the entire family. As those living with these diagnoses, we know support is needed for everyone! We address some of these strategies in Family Matters and the Mind-Body Connection. With school beginning again shortly, if you haven’t established a 504 plan for your child, now is the time as they transition to a new school grade in the fall. For high school students entering college, the transition between the parent and the school advocacy relationship now falls completely to the student. These action items must be implemented before the student begins school. What’s the Plan explains the concept of mitigating circumstances, a change to the federal law that ensures a higher-level of protection for those with disabilities and advocating effectively for federally regulated accommodations and modifications.

(Continued on page 3)


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 2018. More details on each of our programs can be found on our website: www.CHES.education

Friday, June. 22nd - Monday, the 25th, 2018 (Ended) Camp Zeke - Lakewood, PA

After the Shock provides parents and caregivers of newly 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.



Wed., Sept. 5th - Sat., the 9th, 2018 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. 28th - Monday, Oct. 1st, 2018 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.


Friday, July 20th - Sunday, the 22nd, 2018 (Ended) Union Station Hotel - St. Louis, MO

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 12 I New Inhibitor Treatments: Weighing Risks & Benefits Dr. Jonathan Bernstein outlines the risks and benefits of the new hot topic drugs and treatments hitting the market that individuals with inhibitors can consider.


COMMUNITY CHATTER 4 I LadyBugs at World Federation of Hemophilia CHES continues to spread worldwide awareness through LadyBugs (a support program for women affected by bleeding disorders) that women do bleed. A poster was presented at the World Federation of Hemophilia’s Symposium reporting a need for better treatment and resources.

6 I After the Shock: Inhibitor Camp for All Yasmin Pavri shares her thoughts and feelings on CHES’ first camp program for children of all ages 0-18 with an inhibitor. She hopes this pilot program will become a keeper.

FUN & INSPIRATION 11 I An Archer’s Dedication Camper, Evan Chung chose to author a school assignment about his archery experience at CHES’ Inhibitor Family Camp reminding us that our triumphs deserve celebration.

WHAT’S the PLAN? 26 I A Mitigating WHAT?? In January 2009, Congress amended Section 504 of the Rehabilitation Act of 1973 to not consider the effects of any mitigating measures that a student may require. How did this change affect your civil rights?  

WHAT’S NEW 29 I The Access to Marketplace Insurance Act: Allowing Charities to be Charitable A new law for marketplace customers has created a real hardship for many within the past few years. But PSI has been fighting back with H.R. 3976.

30 I Clinical Trials: For Inhibitor Patients Clinical trials are on the rise but not just for products. New treatment methods are just as exciting and may just revolutionize your regimen.


BLOODLINES 18 I It’s Easy! How We Can Report an Adverse Event Ever wonder why or how to report an adverse reaction to a drug? So easy, even YOU can report one.

32 I PTSD and Chronic Health Conditions Traumatic stress effects the brain and having an inhibitor creates a multitude of traumas. Mindfulness strategies that can help.

FAMILY MATTERS 20 I PTSD: Is It Real for Inhibitor Families? We’ve heard the questions from families traumatized over and over as to whether or not they may be affected by PTSD. Dr. Gary McClain explains the symptoms and when to seek help.





Summer 2018


Discover Learn more about our focus on the rare bleeding disorder community “Our goal is to help better the daily lives of patients living with hemophilia with inhibitors or severe von Willebrand disease.”

Discover us at hemabio.com or call 1-855-717-HEMA (4362). © 2017 HEMA Biologics, LLC HB061-0617

HEMA-930 HEMA_JA_BLOOD_horiz_FINAL v2.indd 1

7/24/17 12:04 PM

Letter From the Editors (continued) Ever wonder how to file an adverse reaction report to the FDA for a drug? The process is interestingly very simple and an important step in maintaining drug safety. In Bloodlines, read how you as a consumer may participate in this process. What are some of the clinical trials actively recruiting for new products that may provide new treatments for those of us managing an inhibitor? There are many, and if you aren’t comfortable with switching to what is currently available, you have choices. Check out our What’s New article. CHES neither endorses or 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. As always, we challenge you to see the inspirational and lighter side of life with an inhibitor in Fun and Inspiration. Community Chatter highlights our first After the ShockSM inhibitor family camp for our program in June. We always appreciate the voices and perspective from members of this community.

What do you know of HR 397? If you haven’t heard of it, you need to, as it potentially could affect premium assistance programs from non-profits such as PSI that are a desperately needed lifeline to many. Change is inevitable and the threats to those managing a chronic illness have increased. Your voice is just as important now as ever. When you receive emails from your local chapter, HFA or NHF, take the time to make the calls. May you enjoy the remaining lazy days that summer brings. Moments to step out of the regimented routine that the school year brings and sit back to appreciate ice-cream or watermelon on a hot summer night. Do you have an idea, comment or suggestion? We really want to hear from you! Share your thoughts at info@ches.education - Janet Brewer & Eric Lowe

“Truth can be stated in a thousand different ways, yet each one can be true.” -Swami Vivekananda jbrewer@ches.education l elowe@ches.education

LadyBugs F e d e r World ation

by Janet Brewer, M.Ed




in Glasgow, Scotland - May 20-24, 2018


or the second time, CHES has presented a poster abstract at WFH on the LadyBugs Program. LadyBugs was founded in 1998 by Barb Forss, an amazing woman who received her diagnosis of severe FVII deficiency at the age of 47. She had been told her entire life that “only males have hemophilia”. Armed with her new knowledge, Barb became an outspoken advocate for women with all bleeding disorders. Although CHES has assisted Barb and LadyBugs for over a decade; we officially assumed it as a program in 2016. Similar to all of our programs, the LadyBugs Program satisfaction rating was 97% in 2017.

Pictured: Janet Brewer

In 2016, CHES offered the LadyBugs program in three (3) regions within the United States, partnering with local chapters in Oregon, Texas and New York.

These three programs were small, but successful, with an overwhelming request for MORE. The first and only National LadyBugs Women’s Summit became a reality on November 4-6, 2018 in Newport News,VA. Funding from Aptevo, Bioverativ, CSL Behring, Grifols, HEMABiologics, Kedrion, Novo Nordisk, Octapharma, Shire, and Soleo made this inaugural event possible. Separate tracks for vonWillebrands, FVIII/FIX and Rare Bleeding Disorders to include FV, FVII, GT, FXI and platelet storage pool were presented by Virginia Kraus, Dr. Joanna Davis (who presented Dr. Robert Sidonio’s slides), and Dr. Diane Nugent. Sessions on mindfulness, advocacy, self-infusion and self-care were offered. Saturday evening, we gathered for a paint night celebration. Science is finally confirming what we as women have known for years, we bleed. The “family business” of prolonged periods and anemia are NOT normal! On November 3, 2016, MASAC released a Recommendation Regarding Girls and Women with Inherited Bleeding Disorders #245 indicating that “Inherited bleeding disorders are under-recognized as causes of bleeding in girls and women. However, recent data have documented that 50% of girls and women who are carriers for hemophilia A or B have factor VIII or IX levels below 50%, putting them in the category of mild hemophilia and causing them to have an increased risk of bleeding, especially during menstruation and at the time of an accident, surgery, or delivery.” https://www.hemophilia.org/ResearchersHealthcare-Providers/Medical-and-Scientific-AdvisoryCouncil-MASAC/MASAC-Recommendations/M CHES is committed to increasing awareness, as well as advocating for more research and education for women with any bleeding disorder through the LadyBugs Program.





Summer 2018


Women with All Bleeding Disorders Require Education Authors: Janet H. Brewer M.Ed Organization: Comprehensive Health Education Services



Women have long been an underserved population within the bleeding disorders community. The common conception is that only men have hemophilia, even within sections of the bleeding disorder community. Medical professionals discount women’s claims of abnormal bleeding, leaving us to feel undervalued and demeaned. Within the last decade, increased studies support what women have known all along-WE bleed. In the US, annual, national meetings feature a women’s track, through the assistance of Hemophilia Federation of America local chapter meetings feature women only retreats. Our collective voices are at last being heard!

To propel all women with bleeding disorders issues to the forefront, we set out in 2016, by partnering with three local chapters to offer a one day LadyBugs Program. Sessions on FVIII/FIX carrier status, stress reduction to include mindfulness and yoga, self-infusion, and a rap session. Session and program evaluations were distributed to determine participant satisfaction. These programs were well received in their communities, but the request remained: We Need More. To accomplish this, the first national LadyBugs Women’s Summit was offered for an entire weekend in November 2017, that included breakout sessions for vWD, FVIII/FIX, and other related blood disorders to include: FV, GT, FX, FXI and Storage Pool deficiency. •For the national LadyBugs Summit, program and session evaluations were dispensed to evaluate satisfaction. •A pre-and post-program survey was distributed to evaluate efficacy.

PRE SURVEY & POST SURVEY GRAPHS PRE SURVEY QUESTIONS Q1. I have a deep understanding of my condition.

Q2. I feel successful advocating for myself to get my medical needs met.

Q3. I have a strong support network that understands what I need.

Q4. I am aware of the effects of stress on my body.

Q5. I can confidently infuse myself or loved one.


RESULTS • Cumulative program evaluations from the one day, 2016 LadyBugs programs indicated a 96.7%

satisfaction rating based on 32 attendees.

• The 2017 national LadyBugs Women’s Summit indicated a 96.9% overall satisfaction rating based on 20 attendees. • Pre-and post-survey data indicated a 5%-30% increase in participant’s understanding of their own condition, their ability to self-advocate and creation of a strong support network.

CONCLUSIONS • Women with all bleeding disorders need and are demanding a voice when it comes to diagnosis, identification and treatment respective to their own health. • National programs organized by and for women with any bleeding disorder deserve the attention and resources necessary to elevate their treatment to the same level as men with bleeding disorders.



chool was over less than 24 hours before we left to participate in “After the Shock 2018” camp organized by CHES. We were tired from a long school/work year and were happy to take some time off from our regular routine. We reached the camp site after almost a 2 ½ hour drive. The final 40 minutes of which was very scenic. It made us excited and eager to know what the camp site would be like. Camp Zeke (located in the foothills of the Poconos) was where CHES chose to host the camp, and it was gorgeous! Scenic, serene, clean, and extremely comfortable!! Every staff member of the camp was dressed with a smile and was welcoming in nature. Great job CHES for this find. Janet Brewer, Eric Lowe, Joan Ward, and Chris Brewer - the hard work and effort you put into pulling this camp together showed and every arrangement was flawless. Adult sessions were very well thought out and kid’s activities had so much variety. While parents were learning about developing coping skills to manage deep emotions dealing with the shock of inhibitors, kids were mentored and monitored by their family pals who were not just supervising them but were befriending them. While parents listened to guest speakers who were throwing light on new treatments for inhibitors, or how to deal with insurance and to be a great advocate for themselves and the community, or





Summer 2018

“Inhibitor Camp for All” by Yasmin C. Pavri


sometimes just hearing each other’s stories; kids were either fishing, doing archery, engaging in arts and crafts or even cooking some delicious desserts. There were outdoor field games and the lake had kayaking and canoeing. There was something for everyone! There was a ping pong table. There was a basketball court. There was a soccer field. And if you got tired, there was even a place to go relax and do some yoga with professional guidance. It can’t get better!! I would also like to give a big shout out to Chad Brown, Adam Smith (with Music for the Cause), and Krystyn Strother for being more than presenters. They got so friendly with all these kids and went out of their way to know each one of them personally. One special family pal was Gabe Anspaugh! Unfortunately, my children

are older now and did not require a family pal with them all the time, but Gabe was their friend, support, helper, fellow community member and someone who related to all these young boys with hemophilia with inhibitors. He was such a positive and high-spirited person that no one around him could sit sad or feel lonely. It was like he could put life into a rock. He is the Rock Star of “After the Shock” camp, just like how Lee was to “The Painted Turtle” location when we attended Inhibitor Family Camp in 2015. There were also these super nurses, Kathy & Brianna, who led the infusion sessions on both days making sure all our children had their factor and were ready to roll for the day. Some even got their big stick award at this camp. Hurray!! We were a variety of families, each displaying strengths in their own ways and motivating others in the room. I personally made a new friend, a good friend in the community who I know I can share my concerns with and who can depend on me when they need a friend. On behalf of my husband Cyrus, my children Farah & Porus, and on behalf of all those that attended this camp, we sincerely want to thank you CHES for giving us this opportunity of learning, sharing, laughing, and most of all having fun together. We look forward to returning to this camp again next year.

Yasmin’s wishboat made during CHES’ signature Parent Cafe’ session. Upon the conclusion of camp, we launched them off of the lake along with our secret wishes of life.





Summer 2018

CHES is proud to partner with these generous sponsors to make After the Shock not just a dream but a reality.




DO YOU OR SOMEONE YOU LOVE HAVE HEMOPHILIA A WITH AN INHIBITOR TO FACTOR VIII? YOU MIGHT BE INTERESTED IN OUR CLINICAL RESEARCH STUDY. Factor VIII inhibitors are antibodies that people with hemophilia A can develop in response to their Factor VIII treatment. They interfere with Factor VIII and prevent it from working the way it should. The development of FVIII inhibitors is one of the most serious complications for people with hemophilia A. The INITIATE clinical study is examining the potential advantages of utilizing a patient-specific laboratory test called batch-selection to decrease the time to success in Immune Tolerance Induction (ITI) for hemophilia A patients with inhibitors to FVIII.


For more information, or if you would like to be considered for participation in the study, please contact us at 916-734-3880. We will be available to discuss any concerns or answer any questions.

Or go to www.initiateusa.com to learn more.


Archer’s Dedication at

Our inhibitor programs are all about pushing your boundaries to try something you may have never done before. Read about Evan Chung’s achievement at Inhibitor Family Camp at Victory Junction. This experience had such a profound effect on him, he wrote an essay about it! Way to go Evan-you demonstrate such determination! We are proud of you! -Janet Brewer


was running down a steep hill at 6:30pm. I was going to archery at Inhibitor Family Camp at Victory Junction. My dad and I came there together. My dad and I had to wait for a while. Finally, I got a spot. It was pretty windy there. I was feeling pretty excited because I just got a spot, then I tried reaching a bow, but I couldn’t reach one because I wasn’t tall, and I was only 6 ¾ years old. I tried many more times, but I couldn’t make it. Then my dad took one bow down, and I said I wanted a different bow. But he said it’s not my size and he also said the one he picked was the right size for me. Dad picked a blue bow.

by Evan Chung

It was almost lunch time, so I thought I’d better hurry up. But I didn’t waste any arrows. I did my first shot out of twelve arrows. Blue part, bad. Then I shot my second arrow. Red part, not that bad! Well now let’s

Then came Larry the archery councilor at camp. He said to my dad “good!” I don’t know why. Then Larry gave me twelve arrows. Then he told me how to do archery. Then he said to try to get a bullseye which is when you get your arrow in the middle of your target. Once at a different camp called Camp Bold Eagle, there was archery too, but I never got a bullseye there. In fact, I never got a bullseye, ever. Anyway, I was really determined to even get one bullseye because my dad told me if I get one bullseye, I can get a gold medal from Larry. Anyway, I shot my first arrow. Really surprising! I did better than last time! Then I shot my next arrow. Then I started going crazy because I just got my arrow in the red part of the target. My dad said, “calm down, Evan” so I calmed down, but in my thoughts, I just kept going crazy. That caused me to waste some arrows because I was going crazy. I only had three arrows left. I shot an arrow. Red part. I shot another arrow. Blue part. Worse. It was my last arrow… and I got the red part. I sighed. “Parent, pick-up!” shouted Larry. Then all of the parents went to pick up the arrows. They also pulled them off the targets.





Summer 2018

skip to the ninth arrow. I got it in the yellow, but it was not a bullseye. I was on my second to last arrow. I took a deep breath, then set up and shot. Then… “twang!” It hit right in the middle of the bullseye! Then I started going really crazy. I shouted, “Larry! Larry! I got a bullseye!” Of course, he didn’t hear me because he was working with another kid. Then I shouted again. This time he heard me. He gave me a gold medal. It was the first medal I ever got. I felt really proud. “I did It!”, I thought. Dad said it was time for lunch. Then we ate lunch. I played with my medal. I felt really proud.


New Inhibitor Treatments:




Long-Term Effects

Thrombotic Event

Quality of Life (Reduced bleeding, Sub-q injections, and more)


ver the past several years, hemophilia care has been turned on its head as new opportunities and ideas for the treatment of hemophilia have emerged. During this time, we have seen the greatest change in hemophilia care since Judith Graham Poole first helped to develop cryoprecipitate and observed that Factor VIII could be made cheaply from this product. These new changes include new factors, new bypass products that can be used subcutaneously, and gene therapy. Finally, we now may have two new ways to treat bleeding diseases that have no treatment at this time.





Summer 2018


By: Dr. Jonathan Bernstein, Md

Disqualificaction of Other Treatments

Inaccurate Lab Readings

Potential Death


With these new treatments come choices where we must weigh the risks versus the benefits of these drugs.

However, research protocols are strict and can have no variance. When a drug is available for use in the medical community, the way it is used by doctors and patients often differ from the strict rules for use that were specified by the research protocol. How many of you have not used your medication in the way you were instructed by your doctor? I know I have – but, of course, doctors are the worst patients. When patients and doctors vary in how they use a drug, this leads to increased risk of something bad happening, and possibly death. Because of our concerns when we first introduce a drug to our patients, we tend to be more conservative in prescribing these drugs. Companies are now required to do what is called “phase 4 research� to try and capture the changes in use over time of a drug or treatment.

Whenever a doctor (or a patient) considers the use of a medicine, they should look at the risks to the patient and weigh these against the advantages or benefit to the patient. Sometimes it is hard to know what the risk might be when a drug is first being used to treat patients. We can look at the research that has been done to test the new drug in a well-defined protocol and see if any significant adverse events were reported.





Summer 2018

Hemlibra The first of these newer drugs, Hemlibra, had problems early on with one death, and the development of both microangiopathic hemolytic anemia and blood clots. These adverse events occurred when Hemlibra was used with a high dose of FEIBA. This combination of a drug that activates Factor Xa (Hemlibra) and a drug containing both Factor Xa and prothrombin (FEIBA), at high doses seems to activate compliment and cause increased clotting and the development of microangiopathic hemolytic anemia (which always seems to involve compliment activation). With further studies, Genentech, the company that developed and tested this drug, was able to eliminate these serious complications, by decreasing the dose of FEIBA, or by avoiding its use completely. Many of you know that 5 patients have died who received this drug off study under compassionate use. While it is stated that these patients’ deaths were not caused by Hemlibra, it is curious that so many deaths occurred in the Factor VIII inhibitor group, when so few were exposed to the drug. Little information has been released on the causes of deaths in these patients. We also have no data on whether we can tolerize patients while on these drugs. Without tolerance, these patients may never be allowed onto gene therapy protocols, as they might have a greater risk of negative outcomes. Hemlibra has also solved many of the problems that patients with Factor VIII inhibitors have. Patients on this drug are able to give themselves subcutaneous (SQ) shots once a week, with few if any breakthrough bleeds. Many patients who were bleeding once or twice a week are now going from a month to years without bleeding. This decreases the damage to muscles and joints, improves quality of life, improves compliance (much easier to give a subcutaneous shot), and can make patients more independent. Thus, in deciding whether to use this drug we must make a choice weighing risks versus benefits. Patients who are doing well on current treatment with tolerance of their inhibitors, should probably avoid Hemlibra. However, for patients with higher titer inhibitors or

frequent breakthrough bleeds, this drug’s benefits will most likely be greater than the risks as long as patients follow instructions for use.

Fitusiran Another drug on the horizon is Alnylam’s/Sanofi’s Fitusiran. This drug increases the thrombin level in the blood by using small interfering RNA (siRNA) to silence the ability of the liver to produce Antithrombin III through RNA interference. It is similar to a process that the body uses to decrease production of certain proteins. This increases the thrombin level in the blood making up for the level lost when one is deficient in a factor. It can be used in patients with inhibitors of factors VIII and IX, as well as to treat patients with factor deficiencies and platelet disorders that can cause excessive bleeding. It is also given subcutaneously. It is currently being studied in a clinical trial protocol at this time. There has been one death in a patient with hemophilia A. This is thought to have occurred when the patient had a sinus venous thrombosis, which then hemorrhaged, resulting in the patient’s death. Protocols and education were put in place to decrease the chance that this would happen again. The decision to use this drug must be decided by both the patient and the physician after balancing risks against benefits to decide if the risks are greater than or less than the benefits. In patients with factor IX deficiency and an inhibitor, this would allow bypass and improve clotting greatly. In patients with The Human Liver

no treatment Antithrombin than antifibrinolytics, such as Factor

Thrombin other

= Fitusiran (with siRNA) Fitusiran silences the liver’s ability to produce antithrombin while thrombin is still produced. For illustration purposes, only.


with no treatment other than antifibrinolytics, such as Factor V patients, as well as severe platelet disorders, such as Glanzmann’s Thrombasthenia and Bernard Soulier, Fitusiran may greatly improve quality of life and outcome.

who has undergone gene therapy may need to have boosts of the vector to maintain success. However, it is possible that patients might develop antibodies to the vector so that they are no longer candidates for repeat boosts of the vector.

Hemlibra and Fitusiran may also both make it hard to measure Factor VIII and inhibitors as they both bypass what we usually measure to determine levels. New methods must be discovered that will allow us to find the levels in patients on these drugs.

Some patients may not be candidates for treatment with gene therapy. If one has an antibody to the vector the body may reject the treatment. Some companies are attempting to develop several vectors that would avoid this problem. If a hemophilia patient has an inhibitor it could either cause the inhibitor level to increase, or could allow the immune system to see a constant amount of factor and tolerize these patients. I believe that we will see both occur.

Gene Therapy The next set of treatments on the horizon are gene therapies for Factor VIII and IX deficiency. Both will soon be available. Studies are ongoing with increasing success. Gene therapy will only be offered to individuals above the age of 18 for the foreseeable future. This approach has been a long time coming. However, there are still substantial risks associated with gene therapy. In the past, patients (with disorders other than hemophilia) have developed cancers when treated with a retroviral vector (viral DNA that carries the gene into the cells). Genes carried by these vectors into the DNA often inserted themselves next to genes that allowed unrestrictive division, a hallmark of cancer. The improved treatments that were then developed avoid this problem by either not inserting into the genome or having more specific insertion. This is a vast improvement. One company estimated that only one in ten thousand would insert incorrectly. However, there may be millions of these vectors, that will have to be given. This might mean that several hundred could insert incorrectly. If this happens, it is possible that years later a patient could develop a malignancy – the most likely site of cancer would be the liver as these vectors insert the changes into liver cells. Next, although less worrisome, is that with time a patient

Plasma-Derived Factor Finally, a trial comparing plasma Factor VIII to Recombinant Factor VIII showed that plasma Factor VIII was less likely to cause inhibitors than Recombinant Factor VIII. However, much has changed since the study was started. New factors, some of which had longer half-lives, became available. These factors were not compared to plasma. It is believed that humanized factor VIII or extended half-life Factor VIII may develop as few inhibitors as plasma does, and these may also be as good at tolerizing patients as plasma is. If studies prove this to be the case, we may find that more patients convert to these drugs. Thus, we now have a wealth of new treatments available with many others on the horizon. There will be risks and benefits, as is true of all drugs, even relatively benign drugs like acetaminophen or aspirin. When patients are given a chance to treat themselves with these drugs, they must weigh the risks against the benefits and decide whether to use the drug. They must also follow the instructions given to them by their doctors and the drug companies so that they can avoid side effects as much as possible.

Jonathan Bernstein, MD is the medical director of the Hemophilia and Thrombosis Treatment Center at Connecticut Children’s Medical Center. Dr. Bernstein is new from the West Coast where he was Medical Director of a Hemophilia Treatment Center for 18 years. He has taken care of patients with all types of bleeding and clotting disorders and has developed several treatment strategies for patients with rare bleeding disorders.





Summer 2018

Talk to your healthcare provider to FIND OUT IF KOĀTE IS RIGHT FOR YOUR CHILD.

© 2017 Kedrion Biopharma Inc. All Rights Reserved. June 2017 KT-0514-00-2017B

It’s Easy!

How WE Can Report an Adverse Event


by Jane Cotter Forbes

here are many medications for people with MedWatch within 24–48 hours. bleeding disorders in the marketplace today and In considering the filing of a voluntary report for an our pipelines are bursting with new therapies and AE to the FDA, here are some points for guidance! medications. Some medications have been available for many years while others [Should you or a loved one be currently experiencing a life-threatening adverse event, are newly FDA approved. attend to it immediately by contacting your treating physician and/or calling 911.] If you or your loved ones experience a serious adverse IT IS EASY. First you go directly to the FDA event (AE) while using a bleeding disorders medication, MedWatch website [https://www.accessdata.fda. it is always a good idea to voluntarily report an AE to gov/scripts/medwatch/index.cfm?action=reporting. the Food and Drug Administration (FDA) MedWatch home] and click on form 3500B. Then you start filling consumer voluntary reporting program. AE’s should in the blanks. When you are done, you hit send. In be reported as soon as possible to MedWatch using particular, the website asks for a lot number, an NDC Form FDA 3500B by those who are consumers and number, the strength, the units, and the quantity (how medical personnel for the following situations: death, many doses or vials of the medication were used). life-threatening medical events (e.g., anaphylactic Most of this information can be found from the container of the medication and/or on the prescription label. The website asks for a detailed description of the AE or serious problem as well any and all medical reports (if available). You will need to include a list of all other medications in use. This website also suggests not throwing the medication away because the FDA may request what remains for further inspection.

reactions), AEs that require hospitalization (short or long term), disability or permanent damage, congenital anomaly and/or birth defect, and any required medical intervention to prevent permanent impairment (e.g., hemorrhage). If you are not sure if the AE is related to the medications you are using, it still is a good idea to voluntarily submit a report to MedWatch just in case. Mandatory reporting is required by law by userfacilities, importers, distributors, and manufacturers. AE’s for these organizations need to be reported to





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In addition, medication that comes with faulty equipment or devices such as needle breaking or ineffective transfer device to be reported. If you are not able to use the website addressed herein, you are free to call the FDA at 1-800-FDA-1088 in order to file a report. “The FDA encourages patients to report AEs as soon as possible. If the AE team gets a cluster of reports about the same drug in a short period of time, it will be able to respond more quickly.” IT TAKES ONLY A FEW MINUTES. Depending on the details of what you submit and the explanation of the AE, it will mostly likely take only 10 to 15 minutes to complete.

IT IS IMPORTANT. It is the fundamental way by which American people can alert the FDA of any and all AEs after having taken a pharmaceutical medication. This information is gathered, reviewed, analyzed, and a determination is readily made if further action is needed. All AE reports are kept in the FDA database. IT IS APPRECIATED. The FDA truly welcomes this information, for it provides ready and immediate feedback on medications which they have approved. It is an essential feedback mechanism. The FDA MedWatch system also allows for consumers or patients to report voluntarily and independently from their doctors. The FDA “understands that for a variety of reasons, you may not wish to have the form filled out by your health care provider, or your health care provider may choose not to complete the form. IT IS REWARDING. There is a genuine sense of personal fulfillment in having participated in this safety program for not only the American people, but for people worldwide, who might also have access to such medications. It is a tangible means by which we can protect ourselves from harmful medications.

occurring with a particular drug. Some examples include: •

A new serious AE which is not listed in the drug’s package insert

An increase in the reporting rate of an event already occurring with a drug

A new report of how an FDA approved drug interacts with other medications.

A product’s name, packaging, or labeling leading to an AE needs to be adjusted, relabeled, and added safety information needs to be included.

Depending on the information found during the FDA’s review of the safety signals and the severity of the AE, the FDA may decide to: Update the drug’s package insert or labeling •

Place restrictions on the drug’s distribution

Release a drug safety communication to inform the public

Withdraw the drug from the market

IT IS FOR EVERYONE. All Americans using prescription drugs approved by the FDA are encouraged to report all adverse events or serious problems. SO WHAT HAPPENS AFTER AN AE REPORT IS FILED? The FDA reviews all of the submitted MedWatch reports for safety signals - similar adverse events

Examples of AEs: •


cardiac arrhythmias

catastrophic bleeding

debilitating migraines

heart attack

kidney damage

TMA (Thrombotic Microangiopathy)

anaphylaxis reaction

liver damage

shortness of breath

serious infections


extreme nausea



excessive vomiting


swelling in extremities

DIC (Disseminated intravascular coagulation

cancers including rare cancers

blood clot formation

chest pain

anti-drug antibody (inhibitors)



PTSD Is It Real for Inhibitor Families?





Summer 2018

by Dr. Gary McClain, PhD


iving with a chronic condition like a bleeding disorder sure packs an emotional wallop. Not one but repeated emotional wallops. Over time, those wallops can wear you down, and impact your wellness – physical, emotional, spiritual.

One potential outcome is Post Traumatic Stress Disorder, known as PTSD. I know that’s a scary term. And believe me, my intent is not to scare you but to alert you of a potential condition and, more important, inform you about what you can do if you are concerned that you or a family member might be experiencing it. Research has shown that individuals living with chronic conditions can and do experience PTSD. So, two important points to know: PTSD is treatable! And you are not alone!

What Is PTSD? We generally think of PTSD in regard to men and women serving our country in the military. There’s a good reason for that. PTSD is an ongoing issue for those who have been in war zones. So, I am going to give you first the traditional definition of PTSD, followed by what PTSD means when it is caused by the experience of living with a chronic condition. Basically, PTSD is caused by exposure to a traumatic event. After this event, individuals with PTSD continue to be stressed or frightened even when they are not in real danger. The classic example of PTSD is someone who hears a noise and has an extreme reaction, as if exposed to danger when, in reality, they are not. Symptoms of PTSD can result soon after the original traumatic event, or they may develop years later. For a diagnosis of PTSD, the symptoms must last more than one month. And they must be severe enough to interfere with daily functioning, like going to work and basic self-care, and relationships.

What Are the Symptoms of PTSD? PTSD is a serious diagnosis and it is not made haphazardly. Mental health professionals have a very specific set of symptoms they assess before they determine that an individual is suffering from PTSD. Here are examples of symptoms that are associated with PTSD: • Unwanted memories • Loss of pleasure • Extreme reactions to reminders of events experienced as traumatic • Irritability • Changes in thinking or in mood after an event experienced as traumatic • Self-blame • Anxiety that occurs in anticipation of an event • Sleep difficulty • Anxiety about doing the right thing • Isolating oneself from other people


Keep in mind that these can be symptoms of stress, or anxiety, or of other mental health conditions. Again, only a mental health professional who has been specifically trained to diagnose and treat PTSD can make this diagnosis. And again, the diagnosis is made based on the presence of multiple symptoms in a specific combination. All the more reason not to attempt to self-diagnose.

The Cause of PTSD Can Be Cumulative This is a really important thing to keep in mind for individuals living with a chronic condition like a bleeding disorder. Receiving a diagnosis can in itself be experienced as a traumatic event. In that case, the cause and effect are clear. But notice that I use the term “experienced as a traumatic event.” That’s important to keep in mind if you or a loved one has a chronic condition. The stress of events that at face value are relatively minor can build up over time. At some point, one relatively simple event can be what my mom used to call, “the straw that broke the camel’s back.” In other words, if you are constantly in situations that result in stress, you may find yourself less and less able to cope. You may just be worn down emotionally over time. Consequently, at some point, you may begin to experience these events as traumatic. Here are some examples of events that can, over time, lead to a trauma reaction: •

Running out of critical medical supplies, like factor

Problems with insurance coverage

A sudden onset of a bleed

A canceled doctor’s appointment

A major life change, like difficulty in performing basic tasks

Uncertainty, like financial concerns

An unhelpful medical professional

Multiple failures infusing factor

I suspect you have experienced one or more of these events. And you probably have your own coping skills to help you get through them. But over time, experiencing one after the other, if not at the same time, you may find yourself less able to cope.





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Here’s what I think of as the PTSD mindset: Your chronic condition may feel over time like a lurking enemy, and you may perceive yourself as being under siege, an ambush right around the corner. You may find yourself being what therapists call hypervigilant, constantly watching out for signs of attack. On the other hand, denial of emotions, feelings that are pushed down and not acknowledged, but tearing you apart inside, can also contribute to PTSD.

Why Would I Experience PTSD? In other words, why me and not you? Or, why you and not me? There isn’t any one answer to that question. Susceptibility to a diagnosis of PTSD is based on a range of factors. These factors include: •

Your basic personality make-up

The number of events

The frequency of events

Your overall coping skills. Some of us are more resilient than others

Your relationship with the healthcare professionals you are working with

What I want to emphasize is that PTSD is a mental illness. And like a physical illness, it is not something you choose. It does not occur because you are weak. And most important, it is treatable.

Another Caution: Caregivers Can Also Experience PTSD In my own clinical experience, I am often as concerned about the mental health of caregivers, if not at times more concerned, as am I about the person living with the condition. So, if you are a caregiver, monitor yourself for signs that your ability to cope day-to-day is at risk.

What Can I Do? First, Reach Out to a Mental Health Professional When I write mental health articles, I usually provide guidelines regarding what you can do to help yourself if you are experiencing whatever issue I am discussing. But I am reversing that order for PTSD. As I described,

PTSD can have a major impact on your day-to-day functioning. It can not only interfere with your quality of life, it can affect your functioning to the point where it is difficult to effectively cope with the demands of everyday life. If you suspect you are experiencing PTSD, it’s really important to reach out to a mental health professional – counselor, social worker, psychologist, psychiatrist. A trained clinician would have a talk with and evaluate your symptoms, discuss with you what’s going on, how you are feeling. After talking with you, a clinician will make a diagnosis. And them work with you on a treatment plan for the road ahead. Again, your first step is reaching out to a professional.


Building Your Foundation for Healthy Coping Living with a chronic condition like a bleeding disorder can turn your life into what feels like an endless roller coaster, and not the fun kind. You know more about this than I do. These ups and downs can be emotionally exhausting. But you can be in the best position to cope emotionally if you have a strong foundation in place. The following are some ideas for better coping. Please understand that the ideas that follow are not intended to be quick bandages or “cures” for PTSD, only a mental health professional can provide adequate assistance to someone with PTSD. However, having a foundation for healthy coping can help you get through those rough spots that, over time, can contribute to developing PTSD.

Cope Better with Stressful Situations Step back. Ask yourself: Is this a catastrophe or am I magnifying a small inconvenience or annoyance and giving it the power to wreck my peace of mind? Keep in mind that PTSD can cause your thoughts to go quickly to a dark place. Be aware of your symptoms and do something about them. Each of us has our own unique way of reacting to a stressful situation. Bursts of anger. Hands balling up into fists. Sweating. Hyperventilating. Stomach clutching. Where do you feel your stress? Be conscious of how your body is reacting. Take calming breaths. Relax those tightening muscles. Give yourself a talking to. Ask yourself a series of questions: How am I feeling? What triggered all these feelings? What are these emotions making me want to do? And if I took that action, what would the consequences be? What you are doing here is





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engaging your rational mind to help you handle your feelings before they handle you. Watch how this process helps bring you back to yourself. Self-monitor. Be aware of your own emotional state – and whether you might be especially vulnerable to stressful thoughts so that you can be mentally prepared to manage them. And staying aware of what’s going on around you to discern where your mental energy needs to go and where it doesn’t. When you’re not awake, it’s like being trapped in a pinball machine, getting bounced here and there and back again. You can’t control your thoughts, but you don’t have to let them control you.

Manage Your Anxiety Use your breath. Anxiety can get the best of you if you allow yourself to get caught up in the rush of anxious feelings and don’t maintain your perspective. Say to yourself: “Yup, I’m anxious.” And then breathe it out. This will help you to stay in the moment and not get caught up in anxious thinking that can lead to more anxiety and keep you stuck in that anxious place. Ask yourself a simple question: What’s the best way to channel my anxious feelings? Think about constructive ways to put your anxiety to use and to distract yourself at the same time. Is there a task you could get involved in to take your attention away from anxious thoughts? Turn it on, turn it off. Think of your anxiety as a laser beam, and decide where it can be put to the best use. Turning the beam on yourself, and amping up your anxious feelings by letting your thoughts run amok, isn’t the best use. When you’re feeling anxious, visualize yourself holding that laser beam. And remind yourself that you have a choice where you focus it.

Keep Calm Withdraw if you need to. Move yourself out of the center of the action. Physically, mentally, or both. Step back. You can even say something like, “I need a moment.” Use your breathing. That initial emotional rush can be overwhelming. Notice how you’re breathing. Little short breaths? Not breathing at all? Take a series of deep, calming breaths. In through your nose, out through your mouth. Do things that calm you down. Listen to music. Take a walk. Read. Do some relaxation exercises. Anything that helps you to stay calm, that helps you feel connected to your center. Calming yourself down is essential if you are experiencing symptoms of PTSD. Don’t fight your feelings. Okay, so you’re having a lot of feelings. The worst way to cope with emotions is by pushing them down or trying to pretend they aren’t there. Your feelings are your feelings. Good feelings, feelings you aren’t so proud of. Give yourself permission to feel them all. Remember that feelings may not represent reality. The feelings of the moment can make the world around you look pretty foggy. You may see things that aren’t real, like what someone else’s motives look like, or where a situation seems to be going. All the more reason to take a pause until the fog dissipates.

Try to be patient with yourself. Emotions are part of being human. Our way of coping with strong emotions is hardwired into us, based on years of practice. Some of us shut down, some of us wear our emotions on our sleeve. Learning how to cope with emotions in a healthier manner takes practice. And keep in mind, if you’re living with a chronic condition, you have that much more on your plate. So be patient with yourself.

Most of All, Stay Supported… If you’re living with a bleeding disorder and an inhibitor, you already know the value of staying connected to your support network. If you are experiencing strong emotions, and especially if you suspect you are experiencing PTSD, it is important to get emotional support. Professional support. Family support. Friends. And other members of the inhibitor community. The same if you are a caregiver. If you are a family member of someone you suspect may be experiencing PTSD, it’s important to get support for both your loved one and for yourself. Don’t go through this alone! You, your chronic condition, and your mental health. You’re dealing with a lot. And one stressful situation followed by another can take a big toll on your mental health. If it’s all starting to wear you down, do the bravest thing in the world and ask for HELP. PTSD, like other mental health conditions, is treatable.

Gary McClain, PhD, LMHC, Dr. 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 and Chronic Communicationssm. He maintains a Website, www.JustGotDiagnosed.com.


A Mitigating

WH aT ? ?

by Lisa Cosseboom, M.Ed. & C.A.G.S School Psychologist & Special Education Evaluation Team Chairperson

Child Find

Child Find is part of the 504 law in which a school district is responsible for evaluating students who have a suspected disability to determine if they need related services and accommodations under a 504 plan. This extends to students in private schools and who are homeschooled. The district is responsible for evaluating and offering appropriate services and accommodations in the public school setting if a private school student is identified as having a disability. Parents whose children don’t attend public schools may not understand what kind of help and services are available.

Section 504 of the Rehabilitation Act of 1973 Section 504 was initially enacted as one part of the American’s with Disabilities Act. It PROTECTS students with a disability and requires public institutions who receive federal funding. A plan is developed to facilitate EQUAL access to a disabled student as a non-disabled student. Section 504 was enacted to protect qualified individuals from discrimination based solely on their disability. Whether a particular student is protected

under Section 504 requires a determination that the student is an “individual with a disability” and that the student is “qualified” under Section 504. A student is “disabled” under the Section 504 regulations if the student meets any one of the three criteria listed in 34 CFR §104.3(j)(1). An individual is considered disabled under Section 504 if the individual: 1. has a physical or mental impairment which substantially limits one or more major life activities (SEE table 1 for “Major Life Activities”), 2. has a record of such an impairment, or 3. is regarded as having such an impairment. “Substantially limits” is measured against their same age, non-disabled peers in the general population without the benefit of medication or other mitigating measures such as accommodations.

Table 1 (Major Life Activites) “Major life activities, as defined in the Section 504 regulations at 34 C.F.R. 104.3(j)(2)(ii), include functions such as caring for one’s self, performing manual tasks, walking, seeing, hearing, speaking, breathing, learning, and working. This list is not exhaustive. Other functions can be major life activities for purposes of Section 504. In the Amendments Act (2008), Congress provided additional examples of general activities that are major life activities, including eating, sleeping, standing, lifting, bending, reading, concentrating, thinking, and communicating. Congress also provided a non-exhaustive list of examples of “major bodily functions” that are major life activities, such as the functions of the immune system, bleeding disorders, normal cell growth, digestive, bowel, bladder, neurological, brain, respiratory, circulatory, endocrine, and reproductive functions.” Children that have bleeding disorders have the right to a “free and appropriate public education (FAPE)” regardless of the type of disability or the severity. A 504 plan lasts for one school year and MUST be reviewed/ updated annually. A 504 plan protects your child and ensures that the school and school staff are implementing all accommodations your child requires!





Summer 2018

What should be in a 504 plan? The plan should outline what accommodations a student needs to access academics (instructional supports), the physical building space, transportation, testing (standardized and regular classroom), behavior etc. This is to ensure that students with a disability have equal access to all education as their non-disabled peers. For students with bleeding disorders, it is imperative that part of the 504 plan addresses self-management of health conditions in the school setting or school-sponsored events (field trips, dances, field day etc.). Additionally, if self-management is unattainable, the 504 plan should address how the student will have access to health management and by whom. It is also important that the plan address how the plan will be monitored and who the school contact person is. Many districts across the United States find it easier to say to a family that their child with bleeding disorders should not participate in physical education or recess. This is a civil rights violation! Your child has access to all the activities that their non-disabled peers have access to. It is recommended that a 504 plan specifically addresses physical activity as one part of the plan. For example, a student with a bleeding disorder might consider joining a swim team but not a football team, or can play golf but not soccer. It’s imperative that the 504 plan be a specific as possible! The 504 Plan would define the signs and symptoms of your child’s bleeding disorder (i.e. external bleeding, swelling, tingling etc.). Additionally, it should address an emergency care plan for the school about what should happen and who is responsible in the case of a bleed. There should be a coordinated plan between home and school regarding specificity around what to do with a bleed. There should be listed contact numbers for Hemophilia Treatment Center’s (if used) and/or pediatrician, hematologist and parents along with any other pertinent members of the Team. It

should discuss any accommodations necessary for the student to access the building (wheel chair accessible, elevators etc.) and who is responsible for keeping teachers and staff (including substitutes) up-to-date on the students’ 504 plan and/or health care plan. Often times, a 504 and health care plan overlap. Some districts insert the health care plan into their 504 document and vis a versa.

Mitigating Circumstances The Americans with Disabilities Amendment Act of 2008 updated eligibility based on positive mitigating circumstances. They ruled that the determination of a disability must focus on whether or not the student


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. © 2017 Aptevo BioTherapeutics LLC.

All rights reserved.

would be substantially limited in performing a major life activity REGARDLESS of what the mitigating circumstances are. Mitigating circumstances are things like medication (i.e. Factor), medical equipment/ Where to File a Complaint Online: http://www.ed.gov/about/offices/list/ocr/ complaintintro.html. Mail: U.S. Department of Education Office for Civil Rights Lyndon Baines Johnson Dept. of Education Bldg. 400 Maryland Avenue, SW Washington, DC 20202-1100 Telephone: 800-421-3481 Fax: 202-453-6012; TDD: 800-877-8339 Email: OCR@ed.gov





Summer 2018


devices, mobility devices etc. For comparison, refer to table 1 on pg. 30. The law also notes that it can take into consideration the negative mitigating circumstances which is considering the side-effect or burdens of the mitigating circumstances and whether there remains a need for the accommodation.

Post-Secondary 504 Information ADA rights carry into the college setting. However, it is the responsibility of the student to self-identify and request appropriate services to staff. It stays as confidential information and can not be used against the student. Generally, there is a Disability Services office on all college campuses. One thing to keep in mind, is that unlike 504 plans in public schools, there is not a written plan. The college student needs to advocate for himself/herself and provide documentation that can help the disability services center provide the accommodations needed. Although it is not a written plan per se, students have the rights to the accommodations needed!


The Access to Marketplace Insurance Act:

Allowing Charities to Be Charitable by PSI, Mandy Herbert & Jim Romano


atient Services Incorporated (PSI) is leading the effort to pass H.R. 3976, The Access to Marketplace Insurance Act, and protect the valued safety net of patient assistance. This legislation is in response to a regulation issued in 2014, during the implementation of the Affordable Care Act (ACA), that allowed Marketplace insurance companies to deny charitable third-party premium and cost-sharing assistance. The rule essentially provided a permission slip to health insurance providers to prohibit assistance by charities looking to help patients in need in order to shift sicker patients off their plans.

In response to this misguided policy, PSI created the Marketplace Access Program (MAP) coalition to bring together leading patient advocacy groups and patient assistance organizations dedicated to protecting charitable assistance for individuals suffering from chronic and life-threatening illnesses. The focal point for our coalition’s work is H.R. 3976, federal legislation that would require insurers to accept assistance from non-profits, places of worship, and local civic organizations. PSI has worked closely with H.R. 3976’s sponsor, Congressman Kevin Cramer (RND), to develop this legislation and stop this harmful policy from continuing.

Since 2014, the impact of this regulation has been We have made great progress on the bill, garnering considerable, with approximately 90 insurance plans over 130 cosponsors since introduction in October. in 43 states having implemented the prohibition. This rule allows insurers to evade the reforms of the ACA However, if we have any chance of passing this needed removing the pre-existing legislation, we need patients and other stakeholders who condition exclusions and care about patient assistance to make their voices heard. provides an open route to discriminate against PSI would welcome any assistance from readers who patients with higher healthcare costs simply because would like to get involved. If interested, please contact they receive assistance. Taking these discriminatory PSI at jromano@uneedpsi.org or (804) 521-7908 for practices, a step further, health insurance providers more information. have also attempted to expand this prohibition into other markets including the Medicare Supplemental Insurance Market.





Summer 2018


Clinical Trials: For Inhibitor Patients


by Ann Marie M. Minichiello

s research and medical advances progress, it is important to recognize that this momentum is not possible without the involvement of patients. Without clinical trials, new drugs and treatment options cannot be made available to those who would benefit from them. Presently, there are six clinical trials recruiting patients with hemophilia and inhibitors. The following studies are all “interventional” (clinical trials) with the primary purpose of treatment. The information on each of the six trials begins with its official title and drug being used.


ndividualized ITI based on Fviii(ATE) protection by VWF (INITIATE) with Wilate® began on June 1, 2017 and is estimated to be completed by December 1, 2022. Requiring 120 participants, the primary goal of this trial is to compare the outcome of individualized lot selection to random lot selection using Wilate® for immune tolerance induction (ITI). The principal investigation site for this study is the University of California, Davis. (Sponsor: UC Davis)


non-controlled, openlabel, multicenter, study of efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in severe hemophilia A subjects with inhibitors undergoing the first ITI treatment , utilizing biological: rFVIIIFc, began June 30, 2017 and is estimated to conclude December 2020. There are 30 participants needed for this study to describe the time to tolerization using rFVIIIFc within 12 months of ITI. There are 9 sites recruiting. (Sponsor: Bioverativ Therapeutics, Inc.)

Do different lot numbers of the same product work better than others for ITI?

Is there really a better FVIIa treatment regimen out there for bleeds?


phase III study on the safety, pharmacokinetics and efficacy of coagulation factor VIIa (recombinant) in congenital hemophilia A or B pediatric patients from birth to <12 years old with inhibitors to factor VIII or IX: PerSept 2, using biological: coagulation FVIIa (recombinant), began in December 2015 and was estimated to conclude August 30, 2017. Requiring 24 participants, the goal of this study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes. The principal investigation site is the University of Colorado, Denver, with 2 additional sites. (Sponsor: LFB USA, Inc.)

Just how effective is rFVIII with Fc for ITI?





Summer 2018


TLAS-INH: A phase 3 study to evaluate the efficacy and safety of Fitusiran in patients with hemophilia A or B, with inhibitory antibodies to factor VIII or IX in utilizing Fitusiran, recombinant factor VIIa and FEIBA. This study began January 8, 2018 and is estimated to be completed in July 2019. Requiring 54 participants, the goal is to determine the frequency of bleeding episodes in patients receiving Fitusiran for prophylaxis, compared with patients who continue with their regular medication. There are 5 sites recruiting. (Sponsor: Alnylam Pharmaceuticals)


phase IV, multicenter, singlearm, open-label study of Emicizumab (Hemlibra) prophylaxis in patients with hemophilia A and inhibitors undergoing minor surgical procedures with Emicizumab began March 31, 2018 and is estimated to conclude June 28, 2019. Thirty participants are needed for this study to evaluate whether participants receiving Emicizumab prophylaxis can safely undergo minor surgical procedures without prophylactic bypassing agents. There are 3 sites presently recruiting. (Sponsor: Genentech, Inc.)


phase III, multicenter, openlabel study of the efficacy and safety of B-domain deleted recombinant Porcine factor VIII (BAX 802) in subjects with congenital hemophilia A with factor VIII inhibitors undergoing surgical or other invasive procedures with biological: antihemophilic factor (recombinant), Porcine sequence (BAX 802), began December 22, 2016 with an estimated completion date of June 1, 2018. There are 15 participants needed for this study to evaluate the efficacy and safety of BAX 802 in patients who are undergoing surgical or other invasive procedures. There is 1 site recruiting in the U.S. (Sponsor: Baxalta, part of Shire)

Better bleed control for surgery?

What about this new “nonfactor” sub-Q product?

What about Porcine Factor?

The safety and validity of a study is the responsibility of the sponsor and investigators. As with any clinical trial, please consult your healthcare provider (HTC) before participating. For more information, please visit www.clinicaltrials.gov.


by Krystyn Strother

PTSD and Chronic Health Conditions


eing diagnosed with a medical problem can be distressing, even traumatic. The experience of trauma is entirely subjective but it can lead to post-traumatic stress disorder (PTSD). Let’s first look at the definition of PTSD and Trauma: Trauma is defined by the American Psychological Association (APA) as an emotional response to a terrible event.1 That’s a pretty broad definition and can be entirely subjective. PTSD or Post Traumatic Stress Disorder, is described as an intense response to trauma and can create severe distress and can limit one’s ability to function in life.2 PTSD from chronic pain or illness is very real and if you think about it, makes a lot of sense.3 Chronic illness and pain are all at once traumatic to the body, brain and emotions, and the level to which people experience symptoms are vast. Some folks do

well, while others struggle to get out of bed each day. One of the tools that we possess to cope with trauma is to work directly with the brain using mindfulness. Science has shown us, through a huge number of studies, that the brain does not become static once it reaches maturity. It continues to change based on our experiences and actions by way of neuroplasticity. They gray matter in our brain can get bigger or smaller; neural connections can become stronger or weaker. Every time you learn a new dance step you are creating a new neural pathway. We often hear about neuroplasticity in relation to using techniques to heal from injury, cope with chronic pain, deal with stress and anxiety. While creating new neural pathways does help us deal with these things, it can also work against us when we have negative experiences. When trauma is encountered, the brain creates new pathways and changes its structure in response to the event in a way that does not serve us well.4

Photo courtesy of krystynstrother.com and Stark Photography

There is a significant amount of data that supports using mindfulness as treatment for those affected by trauma and PTSD.5 Practicing mindfulness reverses the neurological patterns taking place with trauma and PTSD by decreasing areas of the brain that are more active in stressful situations and increasing activity in areas of the brain that regulate our emotion and decision making.6 Do a google search for “mindfulness brain scan” to see side by side images of brain function before and after practicing mindfulness. It is important to realize and reiterate that fear or anxiety around procedures, infusions, or other pokes and sticks is nothing to be ashamed of, and trying to get yourself or your child to accept an “it’s no big deal” attitude is often not the answer, especially if there is trauma and PTSD involved. Fortunately, there are tools that we can use to help our kids, and





Summer 2018

ourselves, become more comfortable. Practicing together as a family will help those with fear, anxiety, or trauma feel less alone and supported. 1.

Progressive relaxation



Tense the muscles of your body (arms, upper body, legs) and hold for a count of 10.


Release the tension and relax to the count of 20.

Belly breath a.

Relax your face and place one hand on your belly. Take long, slow, deep breaths in through your nose and out through your mouth. Feel the

Having an injection in my arm Thinking about having an injection Listening to someone talk about having an injection Watching someone else have an injection in real life Watching someone else have an injection on TV or online Looking at photos of injections Holding a needle Touching a needle

belly rise as you breathe in and gently drop as you breathe out. Don’t force the breath, let it be easy. Do this for 5 or more breaths. 3.

Fear wave a.

This is helpful for those that may have a fear of needles or experience anxiety around pokes and sticks. This can also be used as a template for other procedures.

We begin by noting what it is that makes us the most anxious. Rather than tackle the big stick first we can start with small things. Use the chart below and have your kiddo rank what scares them the most. This one is filled out as an example. 10 / 10 9 / 10 8 / 10 8 / 10 8 / 10 7/ 10 3 / 10 3 / 10

Developed by Dr. Jane Hutton, Clinical Psychologist. Dr. Katie Steel, Clinical Psychologist. Dr. Natali Chung. Hajar Habibi, Clinical Nurse Specialist. IMPARTS team at Guy’s, St Thomas’s and King’s College Hospitals.

Developed by Dr. Jane Hutton, Clinical Psychologist.

Dr. Katie Steel, Clinical Psychologist. Dr. Natali Begin with the least difficult situation. You can visualize this situation asChung. an island out at sea. You slowly paddle out in your Hajar Habibi, Clinical Nurse Specialist. IMPARTS team boat or surfboard (your kid’s watercraft of choice) toward this point. If anxiety at Guy’s, St Thomas’s and King’s College Hospitals. begins, hold where you are in the water and try a few rounds of progressive relaxation or belly breath. The anxiety doesn’t have to go away completely but it will begin to lessen and become more manageable. Continue moving towards the feared situation. Each bout of anxiety or fear is like with the least difficult situation. You can visualize this situation as an island a wave.Begin It builds and intensifies and then it will gradually reduce. Staying with the fear allows us to see how anxiety makes out at sea. You slowly paddle out in your boat or surfboard (your kid’s watercraft us feel and shows us that it is only temporary. Ride the waves of of choice) toward this point. If anxiety begins, hold where you are in the water the sea until you find yourself at the shore of this particular island.

and try a few rounds of progressive relaxation or belly breath. The anxiety doesn’t have to go away completely but it will begin to lessen and become more Whenmanageable. your childContinue feels confident with one situation, move on Krystyn Strother is the former program director moving towards the feared situation. Each bout of anxiety to the next. Eventually, with visualization practice, there be reduce. at HUSH Meditation, strategic designer/author of or fear is like a wave. It builds and intensifies and then it willwill gradually smoothStaying sailingwith towards these situations. Thisanxiety practice changes the fear allows us to see how makes us feelthe and shows us the HUSH meditation curriculum, is a certified way thethat brain reacts to the actual fear inducing situations when it is only temporary. Ride the waves of the sea until you find yourself at themeditation instructor, co-founder of NOMAD, they arise. Overcoming yourisland. fear will take some time and practice, shore of this particular “Adventures in Wellness”, and yoga instructor.

but in the long run, it will make life less stressful and you will feel less anxious. When your child feels confident with one situation, move on to the next. 1. 2. 3.

Krystyn’s yoga classes range from Vinyasa to Eventually, with visualization practice, there will be smooth sailing towards these Yin. In addition to her regularly scheduled classes, Krystyn guest teaches at several yoga situations. This practice changes the way the brain reacts to the actual fear when arise. Overcoming your fear will take some time teacher training programs throughout the country, (n.d.).inducing Retrievedsituations April 11, 2018, fromthey http://www.apa.org/topics/trauma/ practice, in thefrom long run, it will make life less stressful and you will feel speaks at conferences on mindfulness and (n.d.).and Retrieved Aprilbut 11, 2018, http://www.apa.org/topics/trauma/ stress reduction practices, teaches specialized less anxious. Alonzo, A. A. (2000, May). The experience of chronic illness and workshops, facilitates yoga + adventure retreats, post-traumatic stress disorder: The consequences of cumulative and conducts continuing education classes for adversity. Retrieved April 11, 2018, from https://www.ncbi.nlm.nih.gov/ currently registered RYTs. pubmed/10741582


Bremner, J. D. (2006, December). Retrieved April 11, 2018, from https:// www.ncbi.nlm.nih.gov/pmc/articles/PMC3181836/


(n.d.). Retrieved April 11, 2018, from http://www.bcosi.ca/resources/ publications-archive/publications-archive-2011/mindfulness-in-thetreatment-of-posttraumatic-stress-disorder-among-military-veterans


Ryback, D. (2006, October 1). Self-Determination and the Neurology of Mindfulness. Retrieved April 4, 2018, from https://doi. org/10.1177/0022167806290214

Krystyn holds a certificate of completion in the Yoga of Awareness For Chronic Pain, an evidencebased program sponsored by the Department of Anesthesiology at OHSU. Read more about Krystyn at krystynstrother.com


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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 Dr. Jonathan Bernstein, MD Janet Brewer, M.Ed Evan Chung Lisa Cosseboom, M.Ed., C.A.G.S Jane Cotter Forbes, RN Dr. Gary McClain, PhD Ann Marie M. Minichiello Yasmin C. Pavri Krystyn Strother

Contributing Materials Patient Services, Inc (PSI)

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