Quarterly

Quarterly

ADVOCATING ON THE HILL | THE INFUSION EXPERIENCE | STRESS REDUCTION TECHNIQUES

Journal of the International Pemphigus & Pemphigoid Foundation

QUARTER 2: 2024

PEMPHIGUS.ORG

Quarterly

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Foundation 03 Message from the Executive Director Patrick Dunn

Advocacy 04 Advocating at the Coalition of Skin Diseases Hill Day

Awareness 05 Accelerating Awareness: The Vital Role of Participation in Dermatology, Nursing, and Oral Medicine Research Meetings Becky Strong

Feature 07 Wound Care and Dressings Brittney Schultz, MD

11 The Infusion Experience: A Personal Perspective Esther Nelson

14 Dealing with Stress and Moving Towards Recovery: Stress Reduction Techniques and Finding a Local Mental Health Provider Melissa M. Chudzinski, MA

14 Research & Treatments 17 Lessons Learned from Failed Clinical Trials

Victoria Werth, MD, and Radhika Gupta

Founder’s Corner 20 Finding Hope and New Beginnings Janet Segall

Spotlight 22 Four Questions with Katerina Patsatsi, MD, MSc, PhD 23 Peer Coach Spotlight: Scott Taub

Quarterly

The Journal of International Pemphigus & Pemphigoid Foundation

ISSUE #115 | Q2 2024 The IPPF is a US 501(c)(3) nonprofit organization. EIN: 94-3207871 BOARD OF DIRECTORS Staci White, President Sonia Tramel, Treasurer David Baron Laurence Gallu Badri Rengarajan, MD Michael Rigas, PharmD Ramesh Swamy Mindy Unger QUARTERLY STAFF Patrick Dunn, Editor-in-Chief, editor@pemphigus.org Anna Lane, Managing Editor Toby Speed, Copyeditor Fred Wish, Copyeditor CONTRIBUTING WRITERS Melissa M. Chudzinski, MA Patrick Dunn Radhika Gupta Esther Nelson Katerina Patsatsi, MD Brittney Schultz, MD Janet Segall Becky Strong Scott Taub Victoria Werth, MD IPPF STAFF Patrick Dunn, MFA, Executive Director Nelly Filippov, Administrative Manager Anna Lane, MSc, Marketing and Communications Manager Mei Ling Moore, Peer Coach Bryon Scott, Awareness Ambassador Coordinator Janet Segall, Peer Coach Rebecca Strong, Outreach Director Scott Taub, Peer Coach Amethyst Yale, Community Engagement Manager Marc Yale, Research and Advocacy Coordinator INTERNATIONAL PEMPHIGUS & PEMPHIGOID FOUNDATION 915 Highland Pointe Dr, Suite 250, Roseville, CA 95678 Toll free: 855-4PEMPHIGUS tel: 916-922-1298 info@pemphigus.org | www.pemphigus.org The Quarterly is published four times per year in both print and digital formats. The material presented is not intended as medical advice or to promote one product or service over another. Readers should consult their physicians before making changes to their health regimen. The contents of the Quarterly cannot be reproduced or copied without written permission from the IPPF. Inquiries should be directed to: 915 Highland Pointe Dr, Suite 250, Roseville, CA 95678 USA. The opinions of contributors are not necessarily those of the IPPF. Electronic versions of the Quarterly are available at pemphigus. org/quarterly. If you would like to submit a story for consideration, please contact our editors prior to submitting your story: editor@ pemphigus.org © 2024 International Pemphigus & Pemphigoid Foundation Printed in the USA by our friends at SUNDANCE PRESS, Tucson, AZ. www.sundancepress.com

Message from the Executive Director Dear Reader, Welcome to the latest edition of the Quarterly. Since publishing the previous issue, I’m happy to share that registration has opened for the 2024 IPPF Patient Education Conference. As you may have heard, this will be our first in-person patient conference since 2019. The agenda is almost finalized, and excitement is high at the IPPF virtual office. Not only will this be the first time in five years that we will gather the pemphigus and pemphigoid community, but it will also be the first time since 2019 that the majority of the IPPF staff will be in one place. It has certainly been too long, and we hope you’ll join us in what is sure to be a great celebration!

All of our current conference information is available at https:// go.pemphigus.org/conference2024. This includes topics and schedules, as well as hotel and travel information for Newport Beach, CA. But don’t worry if you are unable to travel. We still have a virtual option, and all registrations include access to recordings of conference sessions.

Recent Quarterly articles can serve as great starting points for the topics we will dive deeper into at the conference. In this issue, just about every article has a corresponding conference session.

However, as important as education is for managing a chronic rare disease, it’s often the personal connections that provide the hope needed to feel less alone. This is no small thing. In fact, for some people, it’s everything. It’s also the root of everything we do at the IPPF. Sincerely, Patrick Dunn, IPPF Executive Director patrick@pemphigus.org

Advocacy

Advocating at the Coalition of Skin Diseases Hill Day The IPPF attended to ask Congress for access to more care and treatments, and to request funding for dermatological research The following advocacy efforts are outlined on the Coalition of Skin Diseases’ (CSD) website (skincoalition.org/advocacy/): early every patient with a skin disease experiences significant barriers to accessing the healthcare they need. Patients are often unable to access specialists, medications and durable medical equipment, or the full range of services they need to manage their health outcomes.

The CSD supports state and federal initiatives that expand patients’ access to health coverage and health care; remove financial, administrative, and legal barriers to care, providers, and treatment alternatives; and give patients a voice in their care.

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CSD supports legislation that would: Limit Insurance Practices that Act as Barriers to Care, such as prior authorization, step therapy and non-medical switching; placing commonsense guardrails around these protocols and putting patients ahead of health insurer profits.

Establish Guidelines to Protect Patients from High Out-of-Pocket Costs by ensuring transparency around spending, capping out-of-pocket costs, prohibiting co-pay accumulator programs, and extending these protections to the Medicare program.

Improve Access to Care through initiatives that provide patients with greater access to providers, like telehealth services and network adequacy, and timeliness requirements for insurers.

Promote Equity by implementing policies that confront and address inequities of the nation’s healthcare

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Marc Yale, IPPF Advocacy and Research Coordinator, and Patrick Dunn, IPPF Executive Director traveled to Washington, DC, for the Coalition of Skin Diseases (CSD) Hill Day event.

system in providing access to appropriate care for all dermatological patients.

Expand Covered Benefits to Include Co-Morbid Conditions of Skin Disease. Some skin diseases co-occur frequently with other co-morbid conditions, and many create enormous challenges for patients’ mental wellness. CSD supports expanding essential benefits to include coverage for co-morbid conditions and mental health treatment.

Ensure Coverage for Medical Equipment Devices Associated with Skin Disease by requiring insurers to cover cranial prostheses as durable medical equipment for patients experiencing hair loss due to a skin disease and including medically recommended skin disease therapies on insurer’s formularies.

Preserve Protections for Patients with Insurance Coverage by opposing the elimination of important federal coverage protections currently in place for patients and their families.

Increase Health Insurance Coverage Options for Uninsured Americans by supporting state and federal efforts to eliminate the coverage gap in all states.

Modernize Federally Regulated Health Insurance Policies to Better Protect Patients ensuring that ERISA plans cover the same benefits under the same cost-sharing rules as state-regulated policies.

www.pemphigus.org

Awareness

Accelerating Awareness: The Vital Role of Participation in Dermatology, Nursing, and Oral Medicine Research Meetings Becky Strong

Certain conditions can be particularly challenging to diagnose and manage in dermatology, oral medicine, and nursing. Among these are pemphigus and pemphigoid (P/P), a group of rare autoimmune blistering diseases affecting the skin and mucous membranes. While P/P may not be as widely known as more common conditions, such as eczema or psoriasis, we know from personal experience that their impact can be profound.

One key strategy in tackling these diseases is raising awareness among those who will see these diseases first: medical, dental, oral health, nursing professionals, and researchers in these fields. This is why it’s crucial that the IPPF participates in events each year. Recently, we attended the following meetings: the 2024 American Academy of Dermatology (AAD) Annual Meeting; the 2024 International Association for Dental Research/ American Association for Dental, Oral, and Craniofacial

An invaluable aspect of these meetings is hearing directly from P/P patients.

Becky Strong, IPPF Outreach Director, presented at the Interdisciplinary Pemphigus & Pemphigoid Symposium in New Orleans, LA.

Research/Canadian Association for Dental Research (IADR/AADOCR/CADR) General Session & Exhibition event; the American Academy of Oral Medicine (AAOM) 2024 Annual Conference; and the 2024 Dermatology Nurses’ Association (DNA) Annual Convention. These events provide platforms for sharing vital knowledge, experiences, and advancements in the field, ultimately leading to better understanding and management of conditions like P/P. In addition to attending the meetings, we also had the opportunity to be a part of multidisciplinary panels at the IADR/AADOCR/CADR and the AAOM meetings. Having experts from multiple disciplines helps healthcare professionals understand the network of experts that patients need in order to take care of their whole person. These meetings encourage networking between multiple fields and help to grow the understanding of the connection between oral and overall health.

Accelerating Diagnosis Times One of the most significant challenges in managing P/P is the delay in diagnosis. Patients often endure months, if not years, of uncertainty and misdiagnoses before receiving an accurate diagnosis. This delay prolongs suffering and keeps patients like us from leading CONTINUED

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“normal” and productive lives. There isn’t one magic pill that works to control everybody’s disease and put us into remission quickly, so learning about common treatments, as well as repurposed medications and new medications in the pipeline, can help doctors manage our disease better—and help the IPPF learn where to direct research and advocacy efforts.

By participating in these meetings, healthcare professionals gain access to the latest diagnostic techniques and guidelines from experts in our field, many of whom are members of the IPPF Medical Advisory Council. They can learn about the subtle clinical signs that may indicate P/P and the appropriate diagnostic tests to confirm the diagnosis more promptly.

They also learn empathy from what patients endure to find a diagnosis and treatment. This knowledge can significantly accelerate diagnosis times and improve patient outcomes. But most importantly at these events, we’re able to evaluate what is needed to help foster future research.

The IPPF works diligently to network with healthcare professionals and other patient organizations. This helps grow the network of P/P providers worldwide.

Networking at these events provides us with the opportunity to learn from other organizations regarding professional education, their patient and community support initiatives, and advocacy goals.

Sharing the Patient Experience

Encouraging Research

An invaluable aspect of these meetings is hearing directly from P/P patients. Their experiences provide unique insights into their challenges, the impact on their quality of life, how living with P/P has affected family and social relationships, and their care and support needs.

When medical and dental professionals engage with patient perspectives, they develop a deeper understanding of diseases beyond textbooks and research papers. This engagement fosters empathy and drives healthcare providers to tailor their approaches to better meet patients’ needs. There is a sincere interest in learning how they can better advocate and support their patients with P/P.

Research meetings are essential for the IPPF to foster collaboration and drive medical and dental research advancements. It allows the IPPF to share P/P patients’ needs, new findings, discuss challenges, and brainstorm solutions. By accelerating progress in understanding the underlying mechanisms of P/P, researchers can develop more effective treatments and, hopefully, one day, a cure.

Participation in national and international dermatology, oral medicine, nursing, and research meetings is instrumental in bringing P/P awareness to the forefront, accelerating P/P diagnosis times, sharing the patient experience, accessing support from similar organizations, and encouraging medical and dental research. By actively engaging in these events, the IPPF can drive healthcare professionals to make a significant difference in the lives of patients living with these challenging conditions.

IPPF Support The IPPF plays a crucial role in supporting patients and healthcare professionals. Through the participation in these meetings, the IPPF can provide educational opportunities and resources for healthcare providers; inform the medical, dental, nursing, and research communities about IPPF patient support services; and help lead advocacy efforts.

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When medical and dental professionals engage with patient perspectives, they develop a deeper understanding of diseases beyond textbooks and research papers.

Becky Strong is the IPPF Outreach Director. She was diagnosed with PV in 2010 and is currently in remission.

She lives in Michigan with her family.

www.pemphigus.org

Feature

Wound Care and Dressings Brittney Schultz, MD

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onditions like pemphigus and pemphigoid (P/P) lead to blisters and wounds on the skin and mucosal surfaces, such as the mouth. These are frequently painful and have significant impacts on the ability to perform normal activities of daily living. It can be challenging and time-intensive to care for these lesions. We hope to review some tips for managing these lesions below. Please remember that all advice should be discussed with your doctor to determine if these recommendations are appropriate for your or your loved one’s case.

Dressing Basics There are several types of dressings, including gauze, hydrocolloid, foams, alginate, films, and nonadherent (or nonstick) dressings. Within each type of dressing, there are many brand names. Each type of dressing has different benefits and is better suited for certain wounds.

Several frequently used dressings are reviewed in further detail below. Please note this is not an all-inclusive list.

Gauze • E x a m p l e s : p e t r o l a t u m ( V a s e l i n e ®) g a u z e , Adaptic®, Xeroform® • Advantages: used to keep wound base moist

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Not occlusive, needs to have an additional dressing on top of the gauze to keep it secured

Hydrocolloid • Example: Duoderm® • Advantages: keeps wound base moist, absorbs some drainage, can be cut to fit wound, can leave in place for a few days • Not good for wounds with significant drainage • Hydrogels such as Restore® have similar benefits Foams • Example: Mepilex® • Advantages: keeps wound base moist, absorbs more drainage, may need to be changed frequently if wound has significant drainage • Better for wounds with more drainage • Alginates such as Algisite® have similar benefits Nonadherent dressings • Examples: Telfa®, ABD (abdominal) pads • Advantages: do not stick to surrounding skin so it’s easier to remove without pulling skin, good for heavy drainage • Need to be secured in place (Kerlix ®, clothing, Hidrawear®) CONTINUED

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Films • Example: Tegaderm® • Advantages: keeps wound base moist, flexible (good for difficult to cover areas, such as joints), transparent (makes it easier to monitor the wound), can leave in place for a few days • Not good for draining wounds

Wound Care Tips Tip #1: Moist is better than dry and crusty. Cover your wounds.

Moist, covered wounds heal better than dry, crusty wounds. For this reason, we recommend covering wounds (if a patient can) with some form of dressing (we will get creative below). We also recommend applying some form of lubricating ointment or gauze beneath this dressing as this helps the wound heal and makes it easier to remove the dressing. This can be difficult to do depending on the location or extent of your wounds, so this advice should be adapted to your situation. For example, it would be impractical to cover wounds in the mouth but moisturization can still be utilized on the lips.

There are additional specifics to achieve these tips below.

• For open areas on the skin, a common regimen might entail topical steroid ointment applied to the wound, followed by petrolatum (Vaseline®) gauze, then a nonstick dressing such as Telfa®, followed by Kerlix® to secure it in place.

• If wounds cover a large surface area, other ideas to hold the dressings in place instead of Kerlix® could include a tight-fitting T-shirt, camisole, or leggings.

For extremities like arms or legs, tubular bandages such as Tubigrip® can help secure dressings as well.

Hidrawear® is a clothing brand originally designed for patients with a different skin condition that also leads to draining wounds on the skin and can be used to hold dressings in place. This clothing is more expensive, but insurance coverage is sometimes possible.

• If wounds are over a joint such as an elbow or knee, films such as Tegaderm® can keep the wound protected, as a film dressing is more flexible.

• In general, we try to avoid popping blisters. If a blister must be popped (or pops on its own), we

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recommend leaving the blister skin on top of the sore as a natural bandage. You can discuss this with your doctor if it would be reasonable for your case to use a small sterile needle to pop certain blisters when they are very large/tense.

Tip #2: Topical steroids come in all shapes and sizes (or rather, potencies and formulations).

Find the one that works for you!

Topical steroids are frequently used in the treatment of P/P. It can sound like alphabet soup to remember all their different names. A few key things to know are that topical steroids come in different potencies or strengths.

Different strengths are better for different body locations (in general, we use weaker steroids on thinner skin such as face, groin, armpits, and stronger steroids for thicker skin such as hands and feet). Additionally, topical steroids come in different formulations, such as gels, creams, ointments, lotions, oils, and solutions. Different formulations are better for different body locations.

Some general recommendations for use of topical steroids are below, but please note that it is very common for different patients to have different preferences. The most important thing is finding what works for you.

• You can apply topical steroids to open wounds.

• Ointments are generally preferred for the skin as they work better and do not contain alcohol, so they are less irritating to the skin. If the greasiness of the ointment is not ideal for you, creams are also a good option but sometimes burn when applied.

• For lesions in the scalp, solutions, or oils are often easier to use.

• For lesions in the mouth, gels or swish/spit solutions are often easier to use.

• If you have disease involvement of the gums, consider using a dental tray to apply your medicated gel. This needs to be a dental tray that will reach the gums (not a teeth-whitening tray).

• If you have more localized lesions in the mouth, you can pat the mucosal surface dry with a piece of gauze, apply the medicated gel, and then hold a piece of non-stick dressing such as Telfa® or hold a cotton swab on top of the medicated gel for a few minutes so it can absorb better.

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Ask for help. Caring for your skin and mucosal surfaces can be a full-time job. You should not have to do it alone.

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Consider saline nasal spray for sores in the nose and saline eye drops for irritation in the eyes.

Tip #3: Modify your personal products and practice to fit your needs.

Conditions such as P/P can have a dramatic effect on your activities of daily life. It can be helpful to modify some of these activities if possible, to reduce the pain associated with your lesions.

• Baths may be better tolerated than showers.

• Consider toddler toothbrushes and toddler toothpaste as these are gentler. Sodium lauryl sulfate (SLS)-free toothpaste can also be less irritating.

• Flossing tape can be less painful than floss.

• Consider using a peri-bottle (examples: Frida Mom®, Lansinoh®; used by women postpartum) or a small watering can with a long spout when urinating if genital sores are present.

• Certain foods or beverages can be very irritating if you have sores in the mouth. The exact triggers are different for each patient, but foods that can often be irritating include spicy, citrus, and crunchy foods (like chips). More information can be found on the IPPF website at pemphigus.org/nutrition.

Tip #4: Consider antimicrobial soaks.

It is normal to have some bacteria on the skin, but the presence or overgrowth of bacteria can make it harder for wounds to heal. Wounds can also become colonized.

Antimicrobial soaks, such as dilute bleach or dilute vinegar soaks, can be effective in many skin conditions due to their anti-inflammatory and antimicrobial effects.

We frequently use dilute bleach baths in children with eczema. It is like going in a swimming pool with chlorine.

If you are experiencing wounds with drainage, please ask your doctor about using these options.

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Dilute bleach baths. ◊ Add ¼ to ½ cup bleach to a tub of warm water (approximately 40 gallons), soak for 5-10 minutes, rinse off completely with warm water. ◊ Alternatively, you can add ½ to 1 teaspoon of bleach into a large bowl (~4 gallons), soak the washcloth in the bowl, apply the washcloth to areas of concern for 5-10 minutes, and rinse off completely with warm water. ◊ You may add a few teaspoons of salt for less irritation or to reduce bleach quantity. ◊ Please use regular or “unconcentrated” household bleach.

Dilute vinegar soaks. ◊ Add 1 tablespoon of vinegar with 8 ounces of water, soak the washcloth in the bowl, apply the washcloth to areas of concern for 5-10 minutes, and rinse off completely with warm water.

Tip #5: You are not alone. Ask for help.

This is the most important tip. Caring for your skin and mucosal surfaces can be a full-time job. You should not have to do it alone.

• Depending on the location of your lesions, it is often helpful to have a specialist dedicated to monitoring and treating that location. For example, your team may include your dermatologist, but also an ophthalmologist, dentist/periodontist, and urologist/gynecologist. We are all wanting and willing to work together to care for you.

• Your primary care doctor is vital to the success of your care team.

• Insurance will sometimes cover home health services for wound dressing changes.

• Additional mental health support is often needed during this difficult time.

• Find a specialist through the IPPF or talk to a peer coach.

Brittney Schultz, MD, is an Assistant Professor of Dermatology and Director of the Autoimmune Blistering Disease Clinic at the University of Minnesota. She is also a Staff Dermatologist at the Minneapolis Department of Veterans Affairs Medical Center.

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www.pemphigus.org

Feature

The Infusion Experience: A Personal Perspective Esther Nelson

Dear Patient, I would like to share my personal experience with infusion treatments for pemphigus vulgaris (PV), including details about what to expect throughout the process. There is a lot to know, but don’t worry—it’s going to be okay. Really. I’ve been through infusions many times and I’m fine. You will be too. Please remember that I am a patient, not a doctor, and I’m sharing what has worked for me with the hope that you will find this helpful. It is important to work directly with a dermatology specialist in pemphigus, if possible. The IPPF maintains a Find a Doctor list (pemphigus.org/ find-a-doctor/) if you need assistance locating a medical professional.

I have been a PV patient since September 2013 and my most recent flare was from November 2023 through March/April 2024. Over the years, I have received both Rituxan® (rituximab) (pemphigus.org/rituxan/) and intravenous immunoglobulin (IVIG) therapy

(pemphigus.org/treatments/). I have had many infusions in the outpatient area of a major hospital (Mount Sinai in New York City) and then recently in a private infusion center (the American Infusion Center in New York City). Dr. Annette Czernik is an excellent specialist who I worked with for many years. She is an IPPF Medical Advisory Council member and recently presented on IVIG for a Patient Education Series webinar (pemphigus. org/patient-education-webinars/). My current doctor, a dermatology specialist in pemphigus, is Dr. Jacob Levitt (in New York City). He has been very responsive and helpful to me.

Rituximab and IVIG Rituximab is a Food and Drug Administration (FDA)-approved medication for PV given via infusions that recalibrates the immune system. It’s like pushing a button, and over 3 to 6 months it takes you back to what your body’s immune system was and should be: CONTINUED

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Feature normal and working well. Life may feel like it was before your diagnosis. Instead of rituximab, some doctors or insurance companies may prescribe or approve a similar infusion called a biosimilar. An example of this is Truxima®, which I had last year. For more details about biosimilars and generic medications, visit the IPPF’s Patient Resources webpage (pemphigus.org/ patient-resources/).

IVIG is also given intravenously. It is clear colored even though it comes from human plasma and contains antibodies which may heal you quicker than rituximab. I found that the IVIG infusions went smoothly and helped to heal me quicker while the rituximab was doing its job.

As a result of my experiences, I would like to share some tips with you:

One month before the infusion: •

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It is important to make sure that your doctor prescribed the correct infusion. In a hospital, it may be easier because they most likely have a department that deals with infusion insurance. Work with your doctor on this.

If you’re going to receive the infusion in a hospital, it may take up to a month before you find out the infusion dates and type of medicine. If it’s in a private infusion center, you will likely receive the infusion much sooner.

Your insurance company, doctor, and the infusion provider may bring up the topic of biosimilars. This can get complicated. However, speak to your doctor about this.

While you are waiting to receive an infusion, you might be experiencing difficulties with eating food.

Consult with your doctor if you’re having problems in your mouth. In my experience, it was helpful for me to avoid salty, hot, cold, or crunchy foods.

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Approximately one week before the infusion: •

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Visit the infusion center in person. This may help you feel more comfortable with the surroundings by knowing what to expect and meeting the staff members. ◊ Determine whether the room has a bed, reclining chair, and/or a television. You want to feel comfortable since you will be spending most of the day in the room. ◊ Ask if they provide lunch or snacks. If they don’t, I recommend bringing food with you. ◊ Especially if this is your first infusion, I recommend talking to someone you trust and feel comfortable with such as a family member or friend.

One day before the infusion: •

There is a lot to know, but don’t worry—it’s going to be okay.

Really.

◊ Cooking in the microwave became my new best friend for many months. The food was quick, soft, and easy to digest. It just slid down my throat. I also prioritized eating enough protein, such as chicken and salmon. If you’re vegan or vegetarian, it’s important to figure out which foods give you the vitamins that you need. Also consider protein shakes with high protein levels.

(Been there, done that!) ◊ You may also talk to your pharmacist about what to eat (and when) if you’re taking other medication. Not all doctors will provide these details, and it’s okay to ask. ◊ Food is medicine and you are your own doctor, too.

Consult with your doctor about your medication dosages (including vitamins) for the day of the infusion.

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I recommend drinking a lot of water. It’s important to be hydrated because when you get to the infusion, you want the nurses to find your veins easily and being hydrated helps with that.

Gather items to take with you to the infusion such as a bottle of water, something to read or a game to play, food (something you can eat with one hand), something to do that is relaxing (and preferably not

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The day of the infusion: • •

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We are here for you and understand what you are going through.

related to your job), comfortable clothes, and a cozy sweater or light blanket.

I also recommend telling a friend, relative, or someone about the infusion. This might help you feel better emotionally.

Check with your doctor about what you should eat the morning of the infusion.

Get a good night’s sleep. It’s going to be okay.

Arrive a bit early. Relax.

The staff will welcome you to a room or chair. Relax.

It’s okay. They know what they’re doing. Chit chat with them! Let them get to know you. They can talk and distract you. Tell them if you’re scared. I was scared, and I’ve always hated needles. Make jokes if it helps!

They may give you a blood test. They will likely test your blood pressure and your heart rate, at least at the beginning, maybe every 15 minutes for the first hour. Then they will look at your veins. They are the “vein whisperers.” But if your veins don’t speak to them, they will give you a heating pad which you’ll put on both hands to warm them so the veins come to the surface, ready to face the day. It’s okay. Breathe.

Before the infusion starts, ask the infusion staff to administer the medication as slowly as possible. In my experience when the speed was raised every 15 minutes, the infusion felt like a waterfall rushing and gushing through my veins.

To be more comfortable, it’s a good idea to ask for some or all of the following: a pillow to put under your arm with the IV, an extra pillow for your back, a blanket, instructions for using the bed or chair, snacks, a call-bell (if you need the nurse), and something to cover your arm if seeing the needles bothers you.

On a big metal pole, they will set up a bag of saline solution (salt water), a bag of liquid antihistamine, and a bottle of the infusion medication. The antihistamine can help with preventing hives or allergic responses. They might also give you a pain reliever and water so your stomach doesn’t feel queasy. I recommend verifying the infusion

Journal of the International Pemphigus & Pemphigoid Foundation

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medication by looking at the bottle. They may give you some type of prednisone orally.

If you need to go to the restroom, take it slowly because you will be taking the pole with you.

Be careful how you move your hand with the IV attached. Ask for help if you need it.

The antihistamine might make you feel sleepy so you may decide to take a nap. That’s fine; it will help pass the time.

Try to relax by reading, calling people on the phone, watching a movie, chatting with the medical staff, and eating lunch or snacks. In my experience, an IVIG infusion lasted about 4-5 hours, while Rituxan® lasted about 6-7 hours.

When the infusion is over, try to relax and determine how you feel and whether you need someone to drive you home.

I found it helpful to drink water the rest of the day.

The day after the infusion: It’s also important to stay hydrated on this day too.

One side effect the day after is getting a migraine, which I had once and never want to have again. I believe that staying hydrated will help you feel fine.

So dear patient, I hope this information has been helpful. Reach out to me (egn5@hotmail.com) or the IPPF if you have additional questions. We are here for you and understand what you are going through. There are IPPF support groups throughout the US that meet virtually. Visit the IPPF Events calendar (pemphigus. org/events/) for information on meetings, webinars, and events to connect with us. It’s going to be okay. You’ll see.

Sincerely, Esther Nelson Esther Nelson, a PV patient and the Tri-State Support Group Leader, enjoys the outdoors, traveling, learning languages, and cooking. She teaches English to immigrants.

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Feature

Dealing with Stress and Moving Towards Recovery: Stress Reduction Techniques and Finding a Local Mental Health Provider Melissa M. Chudzinski, MA

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ullous pemphigoid (BP) has been shown to cause psychological distress for some patients, as some patients may have experienced a loss of ability to perform tasks, along with managing pain and scarring from the disease (Kouris et al., 2016). Patients may also experience difficulties adjusting to a daily routine, as well as increased stress regarding the disease and their individual prognosis (Kouris et al., 2016). Additionally, Kouris and colleagues (2016) explained that “both clinical symptoms and the effects of treatment can have a strong impact on physical and emotional status. They may lead to functional limitations, a need for increased family support, stress, and exclusion from social activities” (p. 601).

It is important for patients, families, and providers to be aware of the impacts of BP on psychological wellbeing (including the effects that the disease may have on levels of stress and anxiety). By taking a more holistic

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biopsychosocial approach, individuals living with BP, their families, and their providers may be better able to address, advocate, and cope with additional perceived barriers that may arise.

There are many different beneficial ways that an individual can cope with stress. Some different activities to assist with stress include visual arts (such as painting or photography), textile arts (knitting, crocheting, quilting, sewing, and embroidery), horticultural arts (including gardening), music therapy, and mindfulnessbased techniques (such as breathing exercises to aid in relaxation).

Mindfulness activities have been shown to be helpful in stress reduction. Some mindfulness-based activities include box breathing, body scan meditation, progressive muscle relaxation, yoga, and focusing on the senses (what you can see, smell, feel, taste, and hear around you).

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Below is an example of box breathing and how to practice it: 1. Breathe out slowly, releasing all the air from your lungs.

2. Breathe in through your nose as you slowly count to four in your head. Try to focus on the air passing through your lungs.

3. Hold your breath for a count of four.

4. Exhale for another count of four.

5. Hold your breath again for a count of four.

6. Repeat for three to four rounds.

(Cleveland Clinic, 2021) Box breathing can be beneficial for short-term stress relief, as it allows the body and mind to get into a calm state. This technique can be difficult at first, but over time with practice it can become easier.

Another mindfulness activity often used for stress relief is body scan meditation. This occurs when an individual focuses on a part of their body and nothing else. For example, someone sitting down could begin to focus on their toes or feet and how they feel on the ground. Then, slowly, the person begins to focus on other parts of the body such as how their legs feel against the chair, how their back feels, and so forth. Additionally, with progressive muscle relaxation an individual can also tense and relax their muscles. Practitioners recommend tensing and relaxing muscle groups one at a time, usually starting with tensing then relaxing the lower portion of the body and moving upwards. This type of activity should not be painful, and the tensing of the muscles should not be to a level of discomfort. If you find that this relaxation technique does not feel comfortable, stop, and try a different technique.

Practicing yoga has also been found to be effective for dealing with stress. The practice of yoga can assist with stress relief through balancing the body’s sympatheticparasympathetic systems. Yoga can allow one to become more aware of their body and the present moment. It can also help relieve physical tension that is kept in the body when a person is feeling stressed or overwhelmed. There are many free beginner yoga videos online to help you get started with the practice of yoga such as yogajournal. com/yoga-videos/best-yoga-youtube-channels/.

Sometimes it can be difficult to know about different stress-relieving activities. There are several phone apps

The practice of yoga can assist with stress relief through balancing the body’s sympathetic-parasympathetic systems. that are available to assist with stress-reduction activities and mindfulness-based activities. Some of these apps include Headspace (headspace.com), Calm (calm. com), Healthy Minds Program (hminnovations.org/ meditation-app), and Smiling Mind. Additionally, some of the best ways to reduce stress include participating in healthy activities that you enjoy and find meaningful.

Being involved in craft projects, going outside for fresh air, spending time with family and friends, and reading are a few examples. Last, if you are feeling stressed and overwhelmed, it can always be helpful to talk these feelings through with someone (such as a family member, friend, or professional).

Individuals with BP who may be experiencing difficulties in adjustment and stress may benefit from seeking a clinical health psychologist, clinical psychologist, or mental health counselor. Coping strategies and self-regulation techniques may assist individuals in the short term to help relieve some high levels of stress and anxiety (such as the use of mindfulness-stress reduction) (Norouzi et al., 2020), while psychotherapy (such as Acceptance and Commitment Therapy or Cognitive Behavioral Therapy) may assist with stress and adjustment in the long-term.

It is important for anyone struggling with anxiety, stress, feelings of loneliness, and depression to connect with a mental health professional to better address their psychological symptoms and distress.

Sometimes navigating the healthcare system can be confusing and difficult. Some resources for finding a provider include connecting with your local psychological association (they sometimes have a “Find a Provider” list on their website). Another way to find a local psychologist that is a good fit for your needs is by using the American Psychological Association’s Psychologist Locator (locator.apa.org/). Here you can CONTINUED

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search your local zip code and find psychologists in your area. Usually, the locator will display the names of local psychologists along with their phone numbers, emails, insurances they accept, and a brief biography about who they are and how they practice. Here are some questions to consider when looking for a provider: 1. Has this provider worked with individuals with autoimmune diseases?

2. Is this provider experienced in working with certain age ranges?

3. Does the provider accept my insurance?

4. Does the provider have experience working with adjustment disorders, anxiety, or chronic stress?

5. Does the provider offer telehealth, in-person therapy, or both?

6. What therapies does the provider use in their practice?

References 1. Akarsu, S., Özbağçivan, Ö., Dolaş, N., & Aktan, Ş.

(2017). Possible triggering factors and comorbidities in newly diagnosed autoimmune bullous diseases.

Turkish Journal of Medical Sciences, 47(3), 832-840.

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2. Cleveland Clinic. (2021, August 16). How box breathing can help you destress. https://health. clevelandclinic.org/box-breathing-benefits 3. Kouris, A., Platsidaki, E., Christodoulou, C., Armyra, K., Korkoliakou, P., Stefanaki, C., & Kontochristopoulos, G. (2016). Quality of life, depression, anxiety and loneliness in patients with bullous pemphigoid. A case control study. Anais Brasileiros de Dermatologia, 91, 601-603.

4. Norouzi, E., Gerber, M., Masrour, F. F., Vaezmosavi, M., Pühse, U., & Brand, S. (2020). Implementation of a mindfulness-based stress reduction (MBSR) program to reduce stress, anxiety, and depression and to improve psychological well-being among retired Iranian football players. Psychology of Sport and Exercise, 47, 101636.

5. Ujiie, H. (2023). What’s new in the pathogeneses and triggering factors of bullous pemphigoid. The Journal of Dermatology, 50(2), 140-149.

Melissa is a graduate student finishing her Doctor of Psychology degree in clinical psychology in Buffalo, NY. She is a volunteer for the IPPF.

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Research & Treatments

Lessons Learned from Failed Clinical Trials Victoria Werth, MD, and Radhika Gupta

Clinical trials and patients with pemphigus or pemphigoid Pemphigus and pemphigoid (P/P) comprise a group of rare, potentially life-threatening blistering autoimmune diseases which have significant physical and psychological impacts on patients and their families. The treatment options currently available for patients are limited to systemic corticosteroids, immunosuppressants, and immunomodulants, all of which have dramatically improved patient outcomes but are associated with significant adverse effects, such as metabolic complications and increased risk of infections, in part due to the high doses required for efficacy. In the past few decades, rituximab has been the only therapy approved for moderate to severe pemphigus vulgaris (PV), but it still requires the concomitant use of corticosteroids and is associated with disease relapses.

Thus, there remains a high unmet need for therapies that provide both a sustained clinical response and have a favorable safety profile.

For the US Food and Drug Administration (FDA) to approve new therapies, they must be tested in rigorous clinical trials that assess the therapy’s efficacy and safety.

In these trials, the therapies are often compared to a placebo or standard of care regimen to assess whether there is a meaningful benefit provided over and above existing treatment options. Every trial pre-specifies certain “endpoints” that must be met to indicate successful achievement of the desired benefit. Some of the factors to consider when evaluating these clinical trials include: 1. Study Design (How was the clinical trial designed?): Are patients randomized to the study groups? Is there a control or placebo group? How many patients were studied? What criteria were used to include or exclude patients during recruitment?

How long was the therapy given? How was the therapy administered? What dose was given?

2. Endpoint Selection (What metrics were used to determine the efficacy or safety of the therapy in the CONTINUED

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Research & Treatments

There remains a high unmet need for therapies that provide both a sustained clinical response and have a favorable safety profile. trial?): Do the outcomes that are used to assess the efficacy of the treatment matter to patients? Are they too stringent to allow adequate measurement of therapeutics? Do they reflect disease activity? Do they capture relapses? Do they capture quality of life measures?

3. Data Reporting (What patient data was reported from the clinical trial?): How long was the follow-up period after treatment? Do they report the adverse effects experienced by patients? Are the adverse effects reported considered mild, moderate, or severe?

4. Mechanism of Action (How does the therapy work to help patients with pemphigus or pemphigoid?): Is there a reason to believe that the mechanism of this therapy even works? Is it suspected to provide symptomatic relief or sustained efficacy? Will it require the use of concomitant medications such as corticosteroids?

What have we learned from trials for patients with P/P that have failed?

Over the past few decades, autoimmunity has become an area of significant research efforts. While no new therapies besides rituximab have been approved by the FDA for pemphigus, trials have been ongoing; some of which are showing promising initial data readouts.

Equally as important though is understanding why many prior clinical trials have “failed.” Here, we explain some of the salient reasons why some trials have been discontinued.

Development of several therapies have been discontinued due to lack of performance in clinical trials. Dompé Farmaceutici S.p.A was evaluating an oral small molecule targeting IL-8 receptors CXCR1 and CXCR2 called DF2156A for patients with active bullous

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pemphigoid (BP), and then later terminated their Phase 2 (NCT01571895) due to lack of efficacy after only onethird of patients had been enrolled at the target dosage.

Similarly, Novartis’s Phase 2 study (NCT01688882) evaluating QGE031/ligelizumab (humanized IgG1 anti-IgE mAb) for patients with PV and the National Institute of Allergy and Infectious Diseases’ (NIAID) Phase 2 study (NCT00283712) evaluating infliximab for patients with PV were both terminated due to lack of efficacy. Sanofi’s Phase 3 study (NCT03762265) for rilzabrutinib/PRN1008 (BTK inhibitor) for patients with moderate to severe PV or pemphigus foliaceous (PF) was stopped because the primary endpoint, which was complete remission, was not significantly different between rilzabrutinib and the placebo. In this case, the poor performance may have been due to the design of the study in which patients in the placebo arm received corticosteroids (CS), making it harder for the intervention arm to show a meaningful benefit. However, results from a prespecified sensitivity analysis using CS dose ≤5 mg/d did show a significant difference between the treatment and the control groups. AstraZeneca’s Phase 3 study (NCT04612790) of benralizumab (anti-IL5 mAb) for patients with BP was also terminated because the primary endpoint was not met, a similar outcome to the Phase 2 trial assessing mepolizumab (anti-IL5) for patients with BP. In the latter trial, the proportion of patients free of relapse did not differ between the intervention and placebo arms. There is reason to believe that the mechanism of these two therapies is not well suited for BP. Other trials have been terminated due to companyspecific reasons. Clinical trials are expensive endeavors to undertake, and substantial funding is required throughout the development process. The University of California Irvine terminated their Phase 1 study (NCT01313923) of sirolimus (mTOR inhibitor) in patients with PV due to lack of funding. Similarly, Immune Pharmaceutical was evaluating bertilimumab (anti-eotaxin-1 mAb) for patients with BV in a Phase 2 trial (NCT02226146) that was terminated due to company bankruptcy.

Companies like Alkahest had to discontinue their Phase 2 trial (NCT04499235) evaluating AKST4290 (CCR3 inhibitor) for patients with mild to moderate PV due to operational challenges. On the other hand, large

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corporations often shift their development strategy.

GlaxoSmithKline’s ofatumumab (anti-CD20 mAb) was acquired by Novartis. Novartis then terminated the asset while in Phase 3 trials (NCT02613910, NCT01920477) for patients with PV. To prioritize other assets in their portfolio, Novartis also terminated their Phase 2 study (NCT01930175) of ianalumab/ VAY736 (anti-BAFF-R mAb) in patients with PV. Some clinical trials have also been stopped due to lack of patient recruitment. This includes the NIAID’s Phase 1 trial (NCT03239470) investigating PolyTregs in patients with PV, Northwestern University’s Phase 1 trial (NCT00278642) investigating hematopoietic stem cells in patients with PV and PF, and Incyte Incorporation’s Phase 2 trial (NCT03780166) investigating parsaclisib (a PI3Kδ inhibitor) in patients with mild to moderate PV. In summary, when reflecting on the outcomes of prior P/P clinical trials, we have identified a few reasons why several therapies seemed to have “failed” to make it to the finish line.

• Systemic corticosteroids have been shown time and time again to provide relief from disease burden.

Trials for therapies such as [FcRn) and Sanofi’s rilzabrutinib (NCT03762265) have given patients in the placebo arm corticosteroids as well, making it harder for the intervention arm to meet primary endpoints and show clinically meaningful benefits.

To differentiate treatment from placebo arms in a trial it is important to have a low enough dose of corticosteroid at the end of the trial for a treatment to be called a success.

• Large-scale clinical trials that are required to adequately assess the safety and efficacy of new therapies are often conducted by biotech and pharmaceutical companies who have ever evolving financial positions and corporate goals. Assets

Clinical trials are expensive endeavors to undertake, and substantial funding is required throughout the development process.

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•

•

•

such as Novartis’s ianalumab (NCT01930175), GlaxoSmithKline’s (later Novartis’s) ofatumumab (NCT02613910, NCT01920477), and Immune Pharmaceutical’s bertilimumab (NCT02226146) are examples of potential therapies that were discontinued due to either a shift in corporate strategy and or inadequate funding.

While preclinical studies may support the potential role of a certain biological pathway leading to P/P, the drug may not perform as expected due to a flaw in the mechanism of action. This was seen in the case of mepolizumab and AstraZeneca’s benralizumab (NCT04612790).

Achievement of primary and secondary endpoints is an essential part of clinical trial success. Selecting appropriate endpoints and their corresponding threshold values is challenging. This process is in part dictated by the FDA. Pemphigus and pemphigoid are rare diseases and only a handful of institutions across the world have experts in the field caring for these patients.

This, along with prespecified inclusion/exclusion criteria and provider and patient preferences and restrictions, can make patient recruitment into trials difficult. For example, most patients with BP are elderly and it can be challenging for them to get to study sites.

LEGEND BAFF = B cell activating factor BTK = Bruton’s tyrosine kinase inhibitor mAb = monoclonal antibody PolyTregs = polyclonal regulatory T cells IL = interleukin Victoria Werth, MD, is a Professor of Dermatology and Medicine at the Hospital of the University of Pennsylvania and the Veteran’s Administration Medical Center. She is the Chief of Dermatology at the Philadelphia VA Hospital. Her clinical and research interests lie in autoimmune skin diseases, including autoimmune blistering diseases.

Radhika Gupta is a rising fourth-year medical student at the Perelman School of Medicine at the University of Pennsylvania. Radhika has a particular interest in autoimmune diseases and hopes to pursue the cutaneous manifestations of these diseases during residency.

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Founder’s Corner

Finding Hope and New Beginnings Janet Segall

W

hen I was diagnosed with pemphigus vulgaris (PV), I didn’t know anything about the disease or how I was going to deal with it. At 36 years old, I had plans other than having a rare disease diagnosis.

It seemed untenable for me to learn to navigate my life with PV and as a single parent.

Hope felt like something hard to come by. No one really understood what I was dealing with emotionally.

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Hope felt like something hard to come by. No one really understood what I was dealing with emotionally.

My family was sympathetic and helpful in many ways, but the first few years of dealing with this alone were daunting for me. There was no internet at that time, so information about PV was scarce. However, I found some stability on 30 mg of prednisone and the lesions went away, so my doctor and I figured out how to live by taking daily prednisone since that was the only choice.

We learned that even reducing the prednisone dosage by 2 mg caused a breakout, so I remained taking 30 mg for three years. Yes, three years on 30 mg of prednisone kept me disease free but I had side effects that were sometimes not pleasant for the people around me.

But without knowing it, I felt hope from the very beginning. After my initial diagnosis was confirmed, my first thoughts were that I didn’t want to die, and I was grateful that I didn’t have amyotrophic lateral sclerosis

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(ALS) or a multi-organ autoimmune disease. So, I had no choice but to find ways to be hopeful. I hoped that I would be able to stop taking prednisone at some point but focused on finding ways to successfully deal with the side effects. I investigated ways to de-stress, participated in biofeedback (a mind-body technique that helps control the body’s functions such as heart rate, breathing, and muscle responses), Chinese Herbal Medicine, hypnotherapy, group therapy, and diet programs because I gained so much weight on prednisone. And to some extent I was successful and made it through those first three years.

After the first three years, I went into remission for the next three years. I felt more “normal,” and went back to the workforce.

But unfortunately, the disease returned. However, I immediately knew what to do at the first sign of a mouth sore. My doctor and I came up with a plan for my prednisone dosage (no other effective drugs were available at that time).

Within a few months I was able to again achieve remission, and back to work I went. I even took a road trip across the country. We stopped at the Petrified Forest National Park in Arizona; saw Elvis’ house in Memphis, Tennessee; and stopped in Washington, DC, and New York City. On our return, we stopped at Niagara Falls in New York; a transcendental meditation spot in Fairfield, Iowa, called the Maharishi Peace Palace; South Dakota’s Corn Palace; Mount Rushmore National Memorial; Badlands National Park in South Dakota; and Yellowstone National Park. Driving home to California from Yellowstone was among the most beautiful scenery I had seen across America, and so hard to describe. On the way home we also stopped at the spectacular Grand Teton National Park. After visiting Jackson Hole, Wyoming, and Lake Tahoe, we arrived home after a magnificent trip. It secured my belief in hope, fortitude, and new beginnings.

Inevitably the disease returned after the trip, but I had the knowledge and understanding to deal with it.

I researched more about the disease and decided it was time to connect with others, including doctors, also living

Inevitably the disease returned after the trip, but I had the knowledge and understanding to deal with it.

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with the disease. It wasn’t easy, but it ended up being worth my time and energy despite being on prednisone and Imuran. This was a new beginning for me.

I never lost hope. I began finding people like me as the internet was just developing and we were able to reach out to each other. I found wonderful doctors who were willing to support the cause. And, to my amazement, the IPPF became a reality. Not only were we connecting with one another across the US, but internationally as well.

Before being diagnosed with PV, I never would have believed that I possessed such determination. As horrible as it was to be diagnosed with PV, it gave me a purpose in my life to find something good and positive to do. Sometimes we think we can’t do something we’ve always wanted to do—or something we never thought we could do. Starting the IPPF changed me. I grew into a different person in the sense that I found something within myself that I didn’t know I had.

I always believed in hope. Sometimes parts within us lie dormant and it takes something like a diagnosis to awaken our inner strength and find a path to our individual new beginnings!

Janet Segall is the Founder of the IPPF and a PV patient since 1983. She is an IPPF Peer Coach and the leader of the Northern California Support Group.

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Spotlight

Four Questions with Katerina Patsatsi, MD, MSc, PHD Our Spotlight section features a medical professional whose work regularly impacts the lives of pemphigus and pemphigoid (P/P) patients. Get to know a new physician, researcher, or other medical professional who knows these diseases best. This issue, we’re featuring Katerina Patsatsi, MD, MSc, PhD. Dr. Patsatsi is a Professor of Dermatology and Venereology at the Aristotle University School of Medicine, in Thessaloniki, Greece. She oversees the Autoimmune Skin/Bullous Diseases Unit and the Cutaneous Lymphoma Unit of the second Dermatology Department/Aristotle University School of Medicine and the Center of Expertise on Autoimmune Bullous Diseases, located at Papageorgiou General Hospital. Dr. Patsatsi was a fellow in Dermatopathology in Dermatologikum, Hamburg, and at the Ackerman Academy of Dermatopathology in New York. She was also a visiting scholar in the Autoimmune Skin Diseases Unit and in the Cutaneous Lymphoma Unit at the Department of Dermatology of the University of Pennsylvania. Her MSc was on Medical Research Methodology and her PhD on the diagnostic procedures of bullous pemphigoid.

She is the current co-chair of the European Academy of Dermatology and Venereology (EADV) Task Force on Autoimmune Bullous Diseases. She has recently initiated the formation of the Greek Bullous Diseases Study Group. Her main research fields include autoimmune bullous diseases, autoimmune skin diseases, cutaneous lymphomas, and inflammatory dermatoses (psoriasis, atopic dermatitis). She has published more than 160 peer-reviewed articles in peer-reviewed journals. She has participated as a principal investigator, as well as a sub-investigator in numerous clinical trials. She has also participated as a co-author in books published in English and Greek literature, and she has lectured at many international and national meetings.

How did you become interested in researching pemphigus and pemphigoid (P/P)?

Twenty years ago, when I was asked to choose the topic of my PhD thesis, I decided to study new diagnostic methods for bullous pemphigoid (BP). For this research I was awarded a scholarship by the Greek State Scholarships Foundation (IKY). At that time, we did not use any serological methods to follow the titer of BP autoantibodies and I was very excited to try new methods in sera and in blister fluid in elderly patients with BP.

What do you think the IPPF community should be researching?

I think that the unmet need requires the development of new treatments.

The authorities must be convinced that it is of crucial importance to find new molecules acting as monotherapy or additional to lower doses of systemic steroids.

What can patients do to get more involved in research?

Patients should be continuously informed and realize that getting involved in clinical trials is the way to help science, the IPPF community, and themselves in the ongoing research on targeted treatments.

What is one fun fact about yourself?

I never lose my positive mindset and my smile, even in difficult situations.

Patients should be continuously informed and realize that getting involved in clinical trials is the way to help science, the IPPF community, and themselves.

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Peer Coach Spotlight

Peer Coach Spotlight: Scott Taub Our Peer Coach Spotlight section features one of our IPPF Peer Coaches, volunteers who have learned to manage living with pemphigus and pemphigoid (P/P).

Peer Coaches share their tips and tricks, as well as IPPF resources and educational materials that help people affected by P/P to have meaningful discussions with their healthcare teams. Learn more at pemphigus.org/peercoaches. This issue, we asked Scott Taub to share more about himself.

Scott Taub became an IPPF Peer Coach in 2020. He was diagnosed with pemphigus vulgaris in 2013 by the eleventh doctor he saw after his symptoms started. Scott achieved remission in late 2016 with prednisone and azathioprine and has maintained remission without any systemic medications since 2017. Scott is an accounting and financial reporting consultant, helping large companies deal with complex transactions and accounting standards. He has been able to continue having a successful career while dealing with PV. He lives in the Chicago area with his wife and two sons.

How did you become involved with the IPPF? When I was finally diagnosed after three months of suffering from painful lesions in my mouth that made it difficult to eat or sleep, I looked for ways to find out more about PV. I found a Facebook group for those dealing with PV and saw postings from the IPPF, including announcements about the upcoming annual Patient Education Conference, which happened to be close to my home. I learned more from the conference than I had learned from my doctors or any other sources. I decided to help others with PV by discussing my experiences and answering questions in the Facebook group. I was glad to be offered the opportunity to continue helping others suffering with P/P as a Peer Coach.

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What is something that community members with P/P can do to better advocate for themselves?

Don’t be scared to ask your provider questions and engage with them in conversations about questions or concerns regarding the disease and your treatment. Utilize the IPPF resources, including the IPPF Guide to Pemphigus and Pemphigoid (pemphigus.org/patient-resources/) and archive of Patient Education Series webinars (pemphigus.org/patient-education-webinars/).

You can then bring up things you learned about with your provider. They are there to help you, and you shouldn’t feel like you need to wait for them to bring something up.

Which treatment(s) did you take in your rare disease journey?

I started with prednisone, as most of us do, and it gave me a lot of relief somewhat quickly. I also used a dexamethasone mouthwash to help and took doxycycline for a while. Of course, I quickly started looking for something that would allow me to get off prednisone, and the first drug I tried was mycophenolate (Cellcept®). After about one year, my doctor and I decided that wasn’t working, so I switched to azathioprine (Imuran). I made steady improvement on azathioprine and prednisone and went into remission. I then very slowly tapered off prednisone, and eventually dropped azathioprine as well. I’ve been in remission for over seven years and off all medications for over six years.

What is something fun or interesting about yourself?

I work as a financial accounting expert, have served on many national and international accounting working groups, and am the author of two very long accounting books.

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