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ITP Awareness Special Edition The Platelet News VOL. 18; NO. 3 FALL 2016 In This Issue Message From the Executive Director 2 Communicating with your Doctor 3 Making a Difference 4 Walk/Run News 8 PDSA Launches First ITP Natural History Study Registry Teen Session at ITP Conference 2016 10 By Alexandra Kruse ITP in Adults: Q&A 11 Starting this winter, you can join thousands of patients living with ITP from across the globe and register with the Platelet Disorder Support Association’s (PDSA) “ITP Natural History Study Registry” to advance research and improve the quality of life of ITP patients. Purple Power Spreads New ITP Awareness! Special Photo Spread 12 The ITP Natural History Study Registry is an international, patient-consented registry of individuals with ITP. The registry aims to collect data on the natural progression of ITP, enabling PDSA to gather data on diagnosis and treatment, management of care, quality of life, and clinician reporting. The registry is administered by PDSA and overseen by the (CONTINUED ON PAGE 7) National Organization for Rare Disorders (NORD), the US Food It’s Official! ITP Advocacy and Support is a Worldwide Initiative In Appreciation 14 Fundraising News 15 PDSA’s First Research Coordinator Alexandra Kruse 16 In Memory / In Honor 17 New Patient-Centered Research Guidelines for ITP Research Proposals 17 Local Support Groups 18 Support Group News 19 Q&A with ICON Pediatrician Dr. Klaassen 20 GLOBAL PARTNERS MEET AS THE INTERNATIONAL ITP ALLIANCE By Nancy Potthast PDSA’s ITP Poke-R Club Abstract One of the Top Posters at NORD Summit 14 International delegates from 8 countries attended the ITP Global Support Group Meeting on Friday, September 16, 2016 at The Shaw Country House Hotel in the U.K. Representatives from International ITP Alliance member organizations included Denmark, Finland, India, Italy, Netherlands, Sweden, United Kingdom, and United States of America. Ahhhh, September. It’s that magical time of year when purple fills the hearts of a rare community with strength, pride and a sense of belonging — when warriors and advocates come together to raise awareness for immune thrombocytopenia (ITP). Just six short years ago, PDSA requested and received the designation of September as National ITP Awareness Month with the last Friday of the month named as Sport Purple for Platelets Day and for the first time this year, the last week of September was established as Global ITP Awareness Week. In all its purple splendor, it seems quite natural that September would also be the month in which (after many years of collaboration) patient support and advocacy groups from around the world should come together as the International ITP Alliance. (CONTINUED ON PAGE 7) IMPORTANT: The information contained in this newsletter is for educational purposes only. For advice on your unique medical condition please consult a health care professional. What Our Friends Are Saying on Facebook 21 Are You Looking for Great Holiday Gifts? 21 Advocacy Alert 22 ITP in Kids Q&A 23 Living with ITP – Patients Share Their Stories 26 Kids Korner 27 2 The Platelet News www.pdsa.org From the Executive Director Ten years ago when I was serving as a PDSA board member and working in the media industry, there was a rumor that actress Julia Roberts had ITP. Not that I would ever wish ITP on anyone, but we were excited with the possibility that one of the biggest stars on the planet would bring much needed awareness for ITP, and help raise funding for research. I sent a media kit with information and resources about ITP and PDSA to Julia’s publicist, but I never heard back. I can’t say whether Julia Roberts had ITP. I have heard rumors that there are several well-known people living with ITP, but keep their diagnosis a secret because of fear that having a chronic illness might impact their career. At the recent NORD (National Organization for Rare Disorders) Summit, parent advocate Kristen Gray shared with 600 conference attendees her family's personal and inspiring story to try and cure Batten disease. Kristen and her husband Gordon founded The Charlotte & Gwenyth Gray Foundation after learning that both their daughters had this aggressive, degenerative and fatal brain disease. Less than 10 children in the world currently live with Batten disease. Almost one year to the day after learning that their older daughter had been diagnosed with Batten disease, the Grays raised $3.2 million dollars and were funding a gene therapy clinical trial. The Gray’s story is very compelling and I was astonished that they could raise so much money in such a short period of time for an extremely rare disease. I know a mother’s (and father’s) love can move mountains, but how were they able to accomplish so much in such a short period of time? Then Kristen shared with the audience that her husband was a big Hollywood producer. He enlisted his celebrity friends like Jennifer Garner, Dwayne Johnson, Rihanna and Jon Hamm to host fundraisers. People were motivated to donate to their cause because their daughters had been handed a death sentence and time was not on their side. PDSA might not have any big celebrities to promote our cause, but what we do have are thousands of “stars” who quietly support our organization with their generous donations and our Awareness Champions who raise funds and awareness about ITP. Over the past 18 years, PDSA has done an incredible job of supporting and educating ITP patients and their families. But we need to do more. ITP is an invisible illness — most of us with the disease “look fine” and it’s not considered a fatal disease. However, while dying from ITP is rare, it still happens. I know patients and caregivers don’t want to hear that, but it is the cruel reality of this disease. Just in the past couple of months PDSA received donations in memory of three ITP patients — one was a ten-month old boy, Talen Chao, who passed away two days after being diagnosed with ITP from catastrophic bleeding in his brain. His mom, Anna Symuong-Chao, contacted PDSA looking for answers and wanted to share her family’s story. And what about the ITP teen who is hospitalized on a monthly basis with excessive bleeding or the young adult who cannot work, participate in “normal” activities, and barely leaves the house because of his ITP? (CONTINUED ON THE NEXT PAGE) PDSA Board Members Peter Pruitt, Chairman, Coral Gables, FL Jay Charness, Vice Chair, Arvada, CO Kim Everett, Treasurer, Mishicot, WI Beth Siegelbaum, Secretary, Norwalk, CT Karen Avrick, Woodbury, NY Brian Bamesberger, Loch Lloyd, MO Jamie Harwell, Helena, MT Diane Joseph, McLean, VA Linda McGuirl, Basking Ridge, NJ Dale Paynter, Cambridge, ON, Canada Join… Your valuable donation will enable PDSA to enhance the programs and services that help those suffering with ITP and other platelet disorders. For more information about making a gift of $1,000 or more, contact Caroline Kruse, Executive Director, at (877) 528-3538 toll free or by e-mail, [email protected]. Thank you to sponsor for supporting the PDSA Website: www.pdsa.org Platelet Disorder Support Association The Platelet News Carol Hoxie, Managing Editor Platelet Disorder Support Association Caroline Kruse, Executive Director Brenda Foster, Administrative Manager Carol Hoxie, Communication Specialist Alexandra Kruse, Research Coordinator Nancy Potthast, Director of Marketing Jody Shy, Programs & Events Manager Sue Zemon, Chief Financial Officer Jeff Cooper, Web site and Social Media Manager phone 1-87-PLATELET 3 ITP Conference 2016: Part 2 In this fall issue PDSA continues its coverage of highlights from presentations at the 2016 ITP Conference in Orlando, FL on July 8-10. Part 1 of the highlights appeared in the summer issue of The Platelet News. To hear full details of the 2016 conference presentations be sure to order your copy of the Conference USB Flash Drive now available: https://www.pdsa.org/products-a-publications/the-plateletstore.html#!/2016-ITP-Conference-Video/p/69192534/category=4605129 Communicating with your Doctor: What Every ITP Patient Wants Their Doctor to Know ITP CONFERENCE 2016 • DRS. DONALD ARNOLD, JAMES BUSSEL, TERRY GERNSHEIMER, CRAIG KESSLER, DAVID KUTER, HOWARD LIEBMAN, AND JOHN SEMPLE By Alexandra Kruse Joan Young, Founder If any of the PDSA medical advisors wanted to quit their day job (and we hope that they wouldn’t!), we now know they have a promising career in acting thanks to the ITP Conference Saturday session, “Communicating with Your Doctor.” Each physician team role-played common scenarios that ITP patients experience with their hematologist. The Platelet News is published quarterly by A nonprofit organization dedicated to enhancing the lives of people with ITP and other platelet disorders through education, advocacy, research, and support. The first ITP patient, Donald (Dr. Arnold), has been bruising for the past month. He was admitted to the ER with a platelet count of 2,000 and feels afraid. He was referred to Dr. Craig Kessler, who inquired about Donald’s family history of low platelets. Dr. Kessler examined Donald’s blood smear (CONTINUED ON PAGE 6) PDSA medical advisors Dr. John Semple (L) and Dr. David Kuter (R) in this entertaining conference session team role-played common scenarios that ITP patients experience with their hematologist Thank you to our 2016 National ITP Conference Sponsors Platinum Sponsor Gold Sponsor Gold Sponsor Silver Sponsor For more information contact: PDSA 8751 Brecksville Road, Suite 150 Cleveland, OH 44141 Phone: 1-87-PLATELET or (440) 746-9003 Fax: (844) 270-1277 www.pdsa.org • [email protected] PDSA is a 501(c)3 organization. All contributions are tax deductible. From the Executive Director CONTINUED FROM PAGE 2 Thanks to the financial contributions of so many, we are excited to announce that we are now at a place where we can start funding and conducting several research studies on an annual basis, and have even added a research coordinator to our staff. There are still so many unanswered questions when it comes to ITP, and we hope this new research will yield some much needed answers. Caroline Kruse, Executive Director Platelet Disorder Support Association 4 The Platelet News www.pdsa.org Making a Difference George R. Buchanan, MD By Caroline Kruse Dr. George Buchanan, pediatric hematologist and ITP specialist, has served as President of the American Society of Pediatric HematologyOncology (ASPHO) and twice on the Executive Committee of the American Society of Hematology (ASH). His national honors include the Distinguished Career Award from ASPHO, the Mentoring Award in Clinical Research from ASH, and the Arnold Gold Foundation Award for Humanism in Medicine from the American Association of Medical Colleges. Dr. Buchanan has retired from the University of Texas at Southwestern Medical Center in Dallas, TX. Bleeding severity and quality of life are key factors when an ITP expert physician and patient/caregiver consider a treatment plan. Unfortunately, it wasn’t always that way. Less than 10 years ago most hematologists relied solely on the platelet count (and some still do) when deciding whether to treat or not to treat. But not Dr. George Buchanan. Calling himself a ‘radical nihilistic “non-treater”,’ Dr. Buchanan has worked tirelessly for 40 years to improve the lives of children with ITP. Dr. Buchanan, a pediatric hematologist, recently retired from the University of Texas Southwestern Medical Center in Dallas TX. In honor of Dr. Buchanan’s distinguished career and numerous contributions to the field of ITP research and advancement of patient care, I asked him to answer a few questions about his lifework. Q: Why did you decide to specialize in ITP and what has been your approach to treating children with ITP? A: My introduction to ITP was during my pediatrics residency in Chicago in the early 1970’s. After encountering several affected children with ITP I came across and was influenced by a 1966 publication in Journal of Pediatrics by Drs. Jeanne Lusher, then a fellow in pediatric hematology in Detroit, and her mentor Wolf Zuelzer, a legendary figure who was one of the “fathers” of pediatric hematology as a distinct discipline. They described the extensive use of steroids to treat ITP, indicating that it could temporarily raise the platelet count but was associated with many undesirable side effects. Although they did not perform a rigorous randomized study (virtually no one did in those days!), their conclusion was that steroids should be avoided in most children with ITP given that the quite substantial side effects did not usually justify their use in such a self-limited condition. This experience influenced me greatly to become a hematologist and paved the way to my approach to managing ITP (which continues to the present) as a “minimalist” or “non-interventionist” who treats the patient, not the platelet count, but instead uses bleeding severity and quality of life as the primary determinants of management. Q: What research have you been personally involved in and what do you hope your contributions or legacy will be in regards to ITP? A: After completing my hematologyoncology training in 1977 I accepted a faculty position at UT Southwestern and Children’s Medical Center Dallas, where I have since remained. For years I (CONTINUED ON THE NEXT PAGE) Medical Advisors Donald M. Arnold, M.D. McMaster University Hamilton, Ontario, Canada James Bussel, M.D. Weill Medical College, Cornell University New York, NY Douglas Cines, M.D. University of Pennsylvania Hospital Philadelphia, PA Amy Geddis, M.D., Ph.D. Seattle Children’s Hospital Seattle, WA Terry Gernsheimer, M.D. Puget Sound Blood Center • Seattle, WA Andra H. James, M.D. Duke University • Durham, NC Craig Kessler, M.D. Georgetown University Hospital Washington, DC David Kuter, M.D., D.Phil. Massachusetts General Hospital Boston, MA Michele P. Lambert, M.D. Children’s Hospital of Philadelphia (CHOP) Philadelphia, PA Howard A. Liebman, M.D. Norris Cancer Center Hospital Los Angeles, CA Diane J. Nugent, M.D. Children’s Hospital of North Orange County (CHOC) • Orange, CA Drew Provan, M.D. Barts & The London Queen Mary’s School of Medicine & Dentistry London, UK John Semple, Ph.D. St. Michael’s Hospital • Toronto, Canada Michael Tarantino, M.D. Bleeding & Clotting Disorders Institute Peoria, IL Emeritus Robert McMillan, M.D. The Scripps Research Institute • La Jolla,CA Platelet Disorder Support Association “For more than 40 years, Dr. Buchanan has made countless, meaningful contributions to our understanding of pediatric benign and malignant hematology, especially on the optimal management of ITP in children. Like many pediatric hematologists in the US and around the world, I am fortunate to have learned from Dr. Buchanan over the years. He is truly a legend in the field.” – PDSA Medical Advisor Michael Tarantino, MD, Medical Director and President, The Bleeding and Clotting Disorders Institute “Dr. Buchanan has been a leader in the field of pediatric hematology. He has conducted numerous research studies that have improved the care of children. He especially focused on understanding the disease from the perspective of the patient and learning how to capture clinically relevant information. In ITP for example, he led the way in capturing important outcomes like bleeding symptoms and changed practice by focusing treatment decisions on symptoms rather than the platelet count. As a clinician he engaged families in shared decision making, taking the time to discuss the disease and treatment options, and understand their goals and concerns. He has trained over 80 fellows and has made an impact on the career of many individuals. As his mentee, he was always supportive and you worked hard because he believed in your success.” – Cindy Neunert, MD MSCS, Columbia University Medical Center, Chair of the Pediatric ITP Consortium of North America (ICON) phone 1-87-PLATELET 5 Making a Difference CONTINUED FROM PAGE 4 practiced oncology as well as hematology and was Division Director between 1980 and 2011. Our program was a large one, so I had the opportunity to see and care for many children with ITP and noted, like Dr. Lusher and many others, that most of them did quite well without steroid treatment. By this time the debate (steroids vs no steroids) was intense in the childhood ITP world, as were other controversial issues such as the need for a bone marrow exam to “rule out” leukemia and whether hospitalization was necessary when the platelet count was extremely low. Being a bit frustrated by the lack of formal prospective studies in children with newly diagnosed ITP, I designed and conducted a clinical trial randomizing such patients to steroids or a placebo/ sugar pill and measured over the next four weeks their platelet counts, bleeding times, and clinical “bleeding scores.” Upon its publication in 1984 it was the first (or one of the first) to randomize ITP patients to steroids versus no drug treatment and to quantitatively assess bleeding severity. The 1980’s were when IVIg was introduced and shown to be beneficial (and in most ways superior to steroids) as a means to promptly raise the platelet count. Although I endorsed its use to treat significant bleeding or prevent it upon injury or need for surgery, I was cautious in view of its great expense, adverse effects, requirement for an intravenous infusion, and widespread acceptance by many ITP treaters. In editorials, commentaries, and face to face “debates” with colleagues I stuck by a “non-interventionist” philosophy, which was shortly thereafter challenged by the introduction of anti-D immunoglobulin as a seemingly more “user friendly” means of quickly raising the platelet count (albeit introducing intravascular hemolysis as a potentially serious adverse effect). My reputation as radical nihilistic “non-treater” probably continued to grow, yet during the 1990’s it was gratifying to me that many others shared similar views, as manifested by their willingness to “protest” in a letterto-the-editor in Blood accompanying its publication in 1996 of guidelines recommending routine steroid or IVIg treatment of children with newly diagnosed ITP. Although the debates have continued regarding ITP treatment philosophy, I and many others subsequently “moderated” our views such that an “era of good feeling” began to prevail in the pediatric ITP arena and — much to everyone’s satisfaction — has continued to the present. Interaction among ITP “treaters” and investigators is now rarely heated and often very productive. Organizations such as the Platelet Disorder Support Association (PDSA), the ITP Support (CONTINUED ON THE NEXT PAGE) PDSA’s Executive Director Caroline Kruse with Dr. George Buchanan at the American Society of Pediatric Hematology/Oncology’s (ASPHO) 29th Annual Meeting in Minneapolis, May 2016 6 The Platelet News www.pdsa.org Communicating with your Doctor CONTINUED FROM PAGE 3 and didn’t notice abnormalities on his red or white cells — were there other autoimmune symptoms that could contribute? Donald thought his diagnosis was a mistake and mentioned his physician wanted to perform a bone marrow examination and started him on prednisone, which made him feel hyperactive. Dr. Kessler informed Donald that most hematologists don’t perform bone marrow biopsies on ITP patients anymore. He explained that controversy on how to treat ITP patients exists because of the variety of treatment options. Dr. Kessler recommended the next step is raising Donald’s platelet count to minimize bleeding and that Donald needs to give up his hobby of playing hockey. ITP patient, Terry (Dr. Gernsheimer), has taken prednisone for six months and is seeing Dr. Howard Liebman to seek new treatment. She’s worried about having cancer because she’s being treated at a cancer center. Dr. Liebman explained the close association between hematology and oncology medical training, meaning many hematology patients (without cancer) just receive treatment at cancer centers. Terry disclosed that prednisone has made her gain weight and feel sick, restless, and moody. Dr. Liebman reassured Terry of alternative approaches: some treatments have a high frequency of response and eliminate the need for steroids; others have lower responses but could result in remission. Dr. Liebman recommended Rituxan, explaining that although Rituxan was originally developed to treat immune system tumors, it’s now used in immune disorders to deplete B-cells. Splenectomy was also suggested but has potential long term risks and could be managed by a patient observant about immunizations, infections, and blood clots. Dr. Liebman recommended TPO-agents, which elevate platelet production so that Terry won’t have to take prednisone. Dr. Liebman clarified that although ITP therapies cannot cure ITP, treatment could improve Terry’s symptoms and quality of life. The last patient of the day, “Ms. Semple,” (Dr. John Semple) has had ITP with a count of 10,000 for 30 years and has tried steroids, IVIg, Rituxan®, and TPO. She didn’t want to remove her spleen because of her fear that the treatment won’t work. She saw Dr. David Kuter who reassured her that most patients initially see high rates of response and tried to calm her anxiety by telling Ms. Semple that her spleen could be taken out safely, either by getting her blood counts up with platelet transfusions or maybe having an older surgeon perform the operation, as older surgeons are more used to performing splenectomies with low platelet counts. After inquiring about Ms. Semple’s family history and bone marrow biopsy results, Dr. Kuter wanted to check for anti-platelet antibodies and thrombopoietin levels to validate her ITP diagnosis. Dr. Kuter also suggested combining therapies such as steroids and Nplate®/Promacta®, or enrolling in new experimental research studies. Although Ms. Semple felt that she had tried most of the common ITP therapies without any luck, there were still a variety of other treatments that remained and could allow her to live a normal life with ITP or cause remission. Making a Difference CONTINUED FROM PAGE 5 Association in the U.K., the Intercontinental ITP Cooperative Study Group in Switzerland (ICIS), and ITP Consortium of North America (ICON) have provided highly effective networking opportunities between investigators, clinicians, and patients with ITP. I have had the great fortune of being involved with each of these marvelous groups. ITP management guidelines that are evidence-based are now consulted and frequently employed as well as being subject to periodic review. New agents to effectively manage persistent and chronic ITP are available and undergoing study in children as well as adults. Bone marrow exam and hospitalization are no longer features of “routine” care in most centers. We’ve come a long way since my “initiation” to ITP 30+ years ago! Q: What does the future hold for you and will you still be involved in the world of ITP? A: An important obligation we have as academic leaders in the ITP arena is identifying, nurturing, and supporting the success of young investigators. I take special pride in the many past and current physician trainees in our Dallas program who have focused on ITP. As my career as an ITP doctor is winding down, I have not regretted for a moment my longstanding involvement with ITP patients, their families, and their primary physicians. I will no longer be doing clinical work seeing patients, but I hope to remain connected with PDSA (including their wonderful teleconferences with young ITP patients and their parents), ICON, ICIS, and the ASH Guidelines panel. I will also watch from the “sidelines” what I expect to be major advances during the coming years in better understanding the pathophysiology, treatment, and cure of ITP. It has been a wonderful privilege being a member of the international “ITP Family!” Platelet Disorder Support Association phone 1-87-PLATELET 7 September is ITP Awareness Month CONTINUED FROM PAGE 1 On Friday, September 16th, delegates from Denmark, Finland, India, Italy, Netherlands, Sweden, United Kingdom and the United States met at The Shaw Country House Hotel in Melksham, Wiltshire, United Kingdom for a meeting organized by the ITP Support Association. For many years, these patient groups, as well as others from around the world, have been collaborating to educate, support and advocate for the ITP community and International ITP Alliance members (pictured left to right): Barbara Lovrencic (Italy), PDSA Executive Director, Caroline Kruse, Monika Westerberg (Sweden), Marjo Holmala (Finland) and PDSA Marketing Director, Nancy Potthast have “officially” joined forces as the International ITP Alliance — the intercontinental partnership of ITP patient support organizations committed to education, awareness and establishing a global voice for immune thrombocytopenia patients. As each representative shared details relating to their organization’s size, communication vehicles and means of support, the gateways and barriers existing within geographic borders became clearly evident — there are substantial differences in patient needs based on their country of origin. Amidst the vast contrasts in healthcare funding and patient support, however, there emerged critical common ground — patients overwhelmingly agree that the patient voice is intensely deficient regardless of geographic location. There are four short years left as we round out a decade of ITP awareness. The time has come to share resources, learn from each other’s trials and Pictured L to R at the ITP Support Association Charity Event in Putney, London are Caroline Kruse (Executive Director, PDSA), Barbara Taylor (Novartis), Nancy Potthast (Marketing Director, PDSA), Rosemary Elston, John Flemming, MD (Novartis), and Derek Elston (Board Member ITP Support Association) successes, advance research, focus on patient-centered care and treatments, and create a global voice for immune thrombocytopenia patients. For more information about the International ITP Alliance, photos, global news, and plans for ITP Awareness in 2017 visit www.GlobalITP.org. PDSA Launches First ITP Natural History Study Registry CONTINUED FROM PAGE 1 and Drug Administration (FDA), and a committee of leading hematologists, ITP patients, and caregivers. Registries can provide health care professionals and researchers with firsthand information about people with certain conditions like ITP and other platelet disorders, individually and as a disease population, to increase our understanding of that condition over time. Rare diseases like ITP have posed unique challenges to researchers and drug developers because of small patient populations, lack of data, clinical endpoints that are often unclear, and enrollment and retention challenges for clinical trials. NORD’s Natural History Studies project empowers patients and families to help eliminate some of the uncertainty in rare disease research, making way for progress. Natural history studies are longitudinal studies that aim to fill research gaps, which help medical researchers better understand how diseases progress over time. They yield vital information essential to clinical trial design, such as biomarkers, demographics, important clinical symptoms, genetic and environmental variables, and patient perspectives. Your participation in our secure, confidential HIPAA-compliant registry is likely to increase what we know about ITP and other platelet disorders. It will help healthcare professionals improve treatment, and allow researchers to design better studies on a particular condition, including development and testing of new treatments. The more patients who are registered the more data researchers will have to further their work, which can accelerate research into new therapies for ITP and potentially a cure one day. More information on the patient registry will be coming soon. 8 The Platelet News www.pdsa.org Walk / Run News VISIT WWW.ITPWALK.ORG TO FIND A LOCATION NEAR YOU! Cranberry Township, PA Pump It Up For Platelets! Event Date: August 7, 2016 • Total Raised: $5,627.65 Read Cayden’s ITP Journey: http://bit.ly/2fRGiGI Superheroes unite in the fight in support of ITP Survivor, Cayden Tomczak (pictured front center with his sister and event organizer, Lanni Mozzoni) Team Cayden strikes a pose in support of their little superhero, Cayden Tomczak. Cayden’s Mom Kristen (back row, second from left) knows first-hand that real superheroes often live in the hearts of small children fighting big battles. Her little ITP Warrior beat ITP. Cranberry Township, PA event organizer, Lanni Mozzoni takes a break from event set up to pose with her brother and ITP Survivor, Cayden Tomczak. Rancho Cucamonga, CA Pump It Up For Platelets! Event Date: September 10, 2016 • Total Raised: $3,340 Read Barbara’s ITP Journey: http://bit.ly/2eXTTLv ITP Warrior, Freddy Tinajero, Jr. (center) takes a break from the festivities for a picture with his parents, Maria and Freddy, Sr.; ITP Warrior Freddy (left) walks with one of his fans, younger brother Jose (right). Team Freddy with their impressive showing in support of their young ITP Warrior, Freddy Tinajero, Jr. Thank you to our National 2016 Walk / Run Sponsors Silver Sponsor Silver Sponsor Bronze Sponsor Platelet Disorder Support Association phone 1-87-PLATELET 9 Chicago, IL Pump It Up For Platelets! Event Date: September 17, 2016 Total Raised: $15,039.31 Read Trish’s ITP Journey: http://bit.ly/2froG3V It’s a wrap! Chicago event organizers and volunteers gather in celebration of another successful event. ITP Warrior Caden Slad (center with purple hair) lets loose with team members after a job well-done. Team Caden raised over $1,600 in support of PDSA programs and research; And they’re off! Chicago participants hit the trail for the third annual Chicago, IL Pump It Up For Platelets! event at Half Day Forest Preserve in Vernon Hills, IL. Orange, CA Pump It Up For Platelets! Event Date: September 18, 2016 Total Raised: $7,446 Read the Journey of Four ITP Warriors, a Mom and their fight against ITP: http://bit.ly/2eTBaNT Orange, CA event organizers (left to right) ITP Warrior Cathy Aldama, ITP Mom Leilani DeCastro, ITP Warrior Melissa Hilsabeck and ITP Warrior Kelly Torres with little ITP Warrior, Joey DeCastro (front center) Bridging the geographic gap. Because they are spread all across the country, the Ugly Potatoes set up a virtual team in support of ITP Warrior Aandrea Hays (center) and raised over $1,100 The Cal State Fullerton Titans baseball team shines their support in honor of little ITP Warrior Joey DeCastro as part of Team Ikaika Sandgap, KY Pump It Up For Platelets! Event Date: September 30, 2016 Total Raised: $1,555 Read Anthony’s ITP Journey: http://bit.ly/2fRLZ7s The Sandgap, KY group of participants was small and mighty (like little ITP Warrior Anthony) raising $1,555 for PDSA programs and research! Event Organizer and ITP Mom, Amanda Johnson with her little ITP Warrior Anthony. (CONTINUED ON THE NEXT PAGE) 10 The Platelet News www.pdsa.org Outstanding Teen Session at ITP Conference 2016 By Sam Kalina For many teenagers with ITP, finding people of similar age who understand the adversity of a chronic blood disorder is a challenge. At the annual ITP conferences, the teen session is a chance for teenagers and young adults to meet, connect, and form lasting friendships. This year’s 2016 conference offered not just a single teen session, but an entire separate track with multiple special sessions dedicated to the teen/young adult age group. Leading the sessions were Dr. Michael Tarantino and Dr. Michele Lambert. On the first night of the conference, all the teens gathered for a casual meet and greet, and each participant was given a fun, blank “heroes of science” T-shirt to personalize and color with fabric markers. During this time, the teens were able to discuss a topic given to them by Dr. Tarantino that would be the The teen group session at ITP Conference 2016 segue into the next day’s session: what is the biggest controlled, and issues that could affect problem in ITP? For some, their biggest the study results. With the combined problem was bruising. For others, it was brainpower of all the teens, as well as frequent hospital visits. But as a group, Dr. Tarantino, and Dr. Lambert, a very the teens decided to progress further realistic study was created. with the issue of fatigue. In the next session, Dr. Tarantino and Dr. Lambert assisted the teens in creating a pseudo-study in order to solve the issue of fatigue in ITP patients’ lives. Some of the discussion included ideas on how to measure fatigue, how frequently it should be measured, variables to be Overall, the teen sessions were wonderful environments for teens/young adults to learn about the scientific process, meet and befriend others with ITP, and have fun. The ITP Conference 2016 teen sessions were great successes, and next year’s will only be better! Walk/Run News CONTINUED FROM PAGE 9 Cleveland, OH Pump It Up For Platelets! Event Date: October 22, 2016 Total Raised: $6,263.00 Read Caroline’s ITP Journey: http://bit.ly/2eyi5VW The 42 degree rainy weather didn’t put a damper on the first Cleveland, OH Pump It Up for Platelets! event. PDSA Executive Director Caroline Kruse with her family (L to R), sister Suzanne Kerner, parents Gil and Pat George, ITP Warrior Caroline and husband Ken, and niece Lexi Kerner (front). The Kerner Purple Platelets team raised $1,160! Healing Hailey was the top fundraising team, raising $1,310 for PDSA programs and research. 29 family members and friends rallied to support seven-year old ITP Warrior Hailey Leffel! The Johnson Family from Warren, Michigan drove about three hours to attend the event and support little 3-year-old ITP Warrior Ava The Brecksville/Broadview Heights High School National Honor Society members who volunteered as part of their community service enjoyed sporting purple to show support for PDSA and the six ITP Warriors of this year’s event! Platelet Disorder Support Association phone 1-87-PLATELET 11 ITP in Adults: Q&A ITP CONFERENCE 2016 • PANEL WITH DRS. DONALD ARNOLD, CRAIG KESSLER, JAMES BUSSEL, HOWARD LIEBMAN, TERRY GERNSHEIMER, JOHN SEMPLE, AND DAVID KUTER By Carol Hoxie During this Saturday afternoon session patients had an opportunity to ask questions of the doctors’ panel on a wide range of topics. Q: How often should bone marrow biopsies be done in ITP patients? A: Dr. Kuter: Never. I haven’t done one in a bonafide ITP patient in about 30 years. It is not part of the guidelines for evaluating any ITP patient. There is no need for it if you get a rise in platelets from steroids. Dr. Kessler: If someone has regular runof-the-mill ITP, then it’s not needed, but if you’ve been treated for ITP a while, are not responding to treatment, and have physical exam findings not in keeping with ITP (enlarged spleen or anemia, for example), it might be justified to do bone marrow testing. Dr. Arnold: Some studies included bone marrow tests on patients who were on the TPO treatments Nplate® (romiplostim) or Promacta®/Revolade® (eltrombopag). The studies found no significant differences in bone marrow that was of concern. They concluded there was no need for surveillance bone marrows in patients on TPO agents. Q: I understand CD4+ Treg cells can reduce the tolerance to infection in their absence. When there is reduced number of Tregs can it result in a loss of tolerance to the bacteria in food? My hematologist plans to test me for celiac disease. A: Dr. Semple: I don’t think so. If anything, you’d be more hyperresponsive to the bacteria. I don’t think the low Tregs that you see in active autoimmunity will set you up for increased bacterial infections.” Q: Is there any likelihood ITP could evolve into leukemia or lymphoma? A: Dr. Kessler: There’s no direct link between ITP and development of underlying malignancy. Lymphoproliferative disorders can be affected by autoimmune problems. If you have a patient who presents with ITP and has some other physical abnormalities then it’s fair game to look for some other secondary stimulus. However, ITP itself does not lead to malignancy. My hematologist thinks my ITP is triggered by multiple viruses in my body. I was tested and found positive for Epstein-Barr virus (EBV), CMV, herpes, and a couple others. Can I eliminate those viruses from my body? I had black toxic mold syndrome where this all started. Doctors are telling me those viruses are triggering my ITP episodes. I’ve been on steroids a long time but off them the past four weeks. A: Dr. Kuter: To verify someone is virally infected two documented tests of antibody are needed: 1) Evidence you had the virus and have now cleared it, and 2) the actual viral load. If you have an active virus in circulation that could possibly be a trigger for ITP, a high viral load could affect a person’s immune status. Dr. Liebman: In Chronic Fatigue Syndrome there is a subset of people who can’t handle Herpes viruses, including EBV and CMV. However, I’ve not heard of a case of ITP associated with this syndrome. You should see a good immunologist for a full work up. Dr. Kuter: Getting off steroids may be key here. It’s important to look at using other agents to raise your platelets instead of chronic steroids. some people who are doing research on rapid screening on a Smartphone for hematocrit (hemoglobin) but I’m not aware of anything for platelet testing. Dr. Kuter: I have a patent on a device that never got picked up by industry. It could be done by measuring serotonin in platelets using spectra photography the way they measure blood sugar. Testing is affected by people taking SSRIs and a lot of other stuff, which is why it didn’t get picked up by industry. Most patients don’t need platelet counts more than about once per month (twice per month in some exceptions). The idea that you need a platelet count every time you get an Nplate shot is nonsense. As to testing frequency, unless you live in a remote area, there’s usually a blood center nearby to get your test done readily. You need frequent counts only if you are changing dosage of your treatment. Dr. Liebman: As to platelet count results, be aware that sometimes blood is drawn one day but platelets are not tested until a day later, which can lead to lower counts and false readings. On your lab results check the time of the draw and the time tested. Dr. Kuter: There are some problems with differences in how labs count the platelets. Some labs, for instance, don’t measure the large platelets, which leads to a lower count. Please be nice to your hematologist — get your labs done early in the morning. When you get your counts done at 4 p.m., then (by law) your hematologist gets notified by labs running the tests at 4 a.m. letting me know someone has a low count. Q: Is there any fast blood-screening Q: How well does Rituximab work? Is it platelet test that could be done at home or any ongoing research on home platelet tests? A: Dr. Gernsheimer: I’m working with safe to use it every 2 – 3 years? Could it cause CVID (common variable immune disease)? What are the long-term side Q: Do viruses in the body affect ITP? (CONTINUED ON PAGE 24) 12 www.pdsa.org The Platelet News Purple Power Spreads New ITP Awareness! In fall 2016 Sport Purple for Platelets Day, held September 30th, once again raised public awareness of ITP and platelets across the US and around the world. Many of this year’s participants sent PDSA their great ‘sport purple’ photos (shown here). Adults, teens, and kids wore bracelets, tee shirts, and sported purple attire, fingernails, and hair. Even pets wearing purple joined in the fun. Schools, offices, and sports teams wore their purple shirts. Some ate purple cookies and cupcakes! Whatever activities or events you took part in to help PDSA raise ITP awareness this fall, we heartily thank you for joining in this year’s effort. Platelet Disorder Support Association phone 1-87-PLATELET 13 Dear Caroline, I wanted to share with you that we had a great deal of fun at Rigel Sporting Purple for Platelets!! Our employees are very pumped up to be part of the effort and I wanted to compliment you and your team for doing this event. It binds the entire ITP community together. I am glad we were able to be part of it. Best regards, Raul Rodriguez, President & CEO, Rigel Pharmaceuticals, Inc. 14 The Platelet News www.pdsa.org PDSA’s ITP Poke-R Club Abstract Chosen as One of the Top Posters at NORD Summit By Alexandra Kruse At this year’s NORD Rare Diseases and Orphan Products Breakthrough Summit, participants were invited to submit a scientific poster abstract on “Life-Transforming Treatments: Innovative Research, Medical Education Advancement, and Patient Community Building.” We felt that PDSA’s ITP Poke-R Club (L – R) PDSA’s Alexandra Kruse and Nancy Potthast pose with for Kids with ITP fit the their winning ITP Poke-R Club poster at the October 17-18 NORD criteria and we knew Rare Diseases and Orphan Products Breakthrough Summit in that the Poke-R Club Arlington, VA was a unique and Sixty-seven percent of participants uplifting program for children battling enjoyed everything about the program, ITP, but we needed more information to including BUZZY, the distraction cards, assess the real impact it has on our the prizes, and how it eased their pediatric community. anxiety and helped them to feel less alone in their ITP journey. Overall, the We created a survey and sent it to Poke-R Club program received an participants in the Poke-R-Club. We average rating of 9/10 by participants. then analyzed their responses to questions regarding demographic Needle phobia significantly decreased information, needle phobia and anxiety after enrollment in the Poke-R Club, before and after introduction to the demonstrating that PDSA has Poke-R Club, their favorite and least accomplished its goal in minimizing favorite parts about the program, and the anxiety correlated with treatments, how they felt overall about the draws, and pokes, although we also program. A majority of respondents found that Buzzy doesn’t completely claimed the best part of the Poke-R eliminate pain from venipuncture. The Club program was the BUZZY device or program has a significant positive that it made the child feel special. impact on pediatric patients with ITP, and is most effective for children and preteens who have chronic ITP. Maddie F., who has ITP, happily displays the award and prize she won in the Poke-R Club Not only was PDSA selected to present “ITP Poke-R Club: A Clinical Support Program Bringing Comfort and Support to Kids Coping with Immune Thrombocytopenia” at the Summit, but our poster abstract was chosen as one of the top three abstracts out of 40 posters (CONTINUED ON THE NEXT PAGE) In Appreciation The following individuals were active members of the CIRCLE OF HOPE between July 1 and September 30, 2016. Circle of Hope members donate or raise $1,000 or more within a calendar year and serve as PDSA’s philanthropic leaders. April – June, 2016 Cathy and Raul Aldama • Emily Auterson • Andrew Avrick • Karen and Adam Avrick • Stuart Avrick • Sydney Avrick • Cindy and Philip Ayliff • Kenda and Brian Bamesberger • Kim and Robert Barber • Amita and Ranjive Bhalla • James Bussel, MD • Brendan Cameron • Leilani de Castro • Barry Deutsch • Rachel and Patrick English • Robert Feiner • Allison and Keith Flowers • Susan Frank • Jan and Steve Gardner • Terri and Michael Goldberg • Wanda Gregory • Jen and Benjamin Grimes • Kim and Rodney Hall • Kristin Henrikson and Jon Brandt • Noelle and Mat Heyman • Melissa Hilsabeck • Madeline and John Hromyak • Kristin Hunt • Karen and David Imig • Amanda Johnson • Anne and Pitkin Johnson • Joan and Richard Jordan • Diane and Robert Joseph • Sharon Kaye • Emily and Kris Kile • Louise Kittel • Alexandra Kruse • Caroline and Ken Kruse • Irene and David Kuter, MD, DPhil • Paula Lagree • David Lihani • Jenn and Andy Lindal/For the Love of Gracie • Barbara Link • Mary Lou Lyons • Kristie Lyons • Eleonore and Russell McCabe • The McGuirl Family • Irma and Daniel Miles • Stephanie and Mike Moran • Lani Mozzoni • Destiny Owen • Jeanne and Dan Pinnell • Nancy and Michael Potthast • Jacqueline and Bruce Prescott • Meredith Prescott • Barbara and Peter Pruitt, Jr. • Shirley and Peter Pruitt, Sr. • Patricia Pulley • Angela Racoosin • Virginia Rennie • Martha Roberts • Steven Rodgers • Karen Rosenbaum • Maria Soledad Ruiz (CONTINUED ON PAGE 17) Platelet Disorder Support Association 15 phone 1-87-PLATELET PDSA Fundraising News 2016 Awareness Champion Team Intensifies in Support of ITP Warriors By Nancy Potthast ITP Warrior, John Camera (center, holding medal) and his Student Council celebrate Sport Purple Day. The starting line-up of awareness champions continues to grow as they inspire, share and make the world #ITPAWARE! Do you or someone you know run, cycle or participate in community fitness events? There’s still time to join our team of Awareness Champions! Contact PDSA’s Programs and Events Manager, Jody Shy at [email protected] for more information and to set up your personal fundraising page and get started today! EVENT ORGANIZER(S) LOCATION AMOUNT Key West Half Marathon Cheri Zimmerman Key West, FL $4,080.19 Maple City Savings Bank Raffle prizes and literature at Sport Purple Day organized by ITP Warrior John Camera and his Student Council. The group raised over $270 for PDSA programs and research. “A tribute to all patients with platelet disorders — our office collected ‘Pennies for Platelets’ during September ITP Awareness Month — we collected $250.00 for ITP awareness!!” – Bleeding and Clotting Disorders Institute, Peoria, IL Hornell, NY $232.00 #FASTRUBY The Tillinghast Family Lincoln, NE $100.00 to-date Lagree Fundraisers Paula Lagree McCormick, SC $100.00 to-date Cleveland Half Marathon Alex Kruse Cleveland, OH $3,165.50 Tri-Beta Bio Honor Society Jennifer Roberts, PhD Romeoville, IL $825.00 Linda’s Angels Nicole Novello Bellmore, NY $245.28 to-date Lugo Fundraiser John Camera Brooklyn, NY $722.00 August Challenge Tammy Fassett Bothell, WA $169.27 to-date Virtual Walk Kristie Lyons Oak Ridge, NJ $655.00 to-date Bravelets Linda Hanson Decatur, GA $40.00 to-date Pennies for Platelets Cade Kleisner Neenah, WI $200.00 Parents Partner for PDSA Kim Barber St. Petersburg, FL $345.00 Clinch’s Hope for a Cure Yvonne Molino Longs, SC $361.88 Pennies for Platelets BCDI Peoria, IL $250.00 For a complete list of fundraising events, visit: http://pdsa.org/contribute/fundraising/fundraising-events.html PDSA is a 501 (c)3 nonprofit corporation. All contributions are tax deductible to the extent of the law. PDSA receives NO federal funding. Poke-R Club Abstract One of the Top Posters at NORD Summit CONTINUED FROM PAGE 14 Staff at the Bleeding Clotting Disorders Institute (BCDI), in Peoria, IL decked out in purple for September ITP Awareness Month (of which 80% were submitted by hospitals, prestigious research institutions, and pharmaceutical companies). We were asked to provide a one page-flyer about the Poke-R Club that was printed in the NORD Summit program and distributed to over 600 rare disease stakeholders. You can find both the ITP Poke-R Club flyer and the poster abstract on the PDSA Web site here: http://pdsa.org/resources/researchprogram.html. To enroll your child in the Poke-R Club contact PDSA Programs and Events Manager Jody Shy at 1-87-PLATELET or [email protected]. 16 The Platelet News www.pdsa.org PDSA Welcomes Alexandra Kruse — Its First Research Coordinator By Carol Hoxie PDSA is pleased to welcome Alexandra Kruse as PDSA’s first Research Coordinator. She is responsible for overseeing and executing all research studies affiliated with PDSA. She designed PDSA’s research framework as part of an award from the Patient-Centered Outcomes Research Institute (PCORI), creating new grant guidelines for clinical, laboratory, and observational research. Alexandra plans to work with patients and clinicians to pilot patient-centered research studies conducted by PDSA. She is also PCOR-science trained as a PCORI ambassador. Her other main responsibility is creating and overseeing PDSA’s new ITP Natural History Study/ Patient Registry. She will construct a clinical variables database for ITP patients in conjunction with the National Organization of Rare Disorders (NORD) and the US Food and Drug Administration (FDA). This new database will help advance research in ITP and further understand the individual and community profiles of ITP patients. Alexandra runs PDSA’s Instagram account (follow us at @pdsa_itp!), live tweeted the conference this past July, and writes several of the scientifically-based articles for the quarterly newsletter The Platelet News and the monthly E-news. Although originally from Cleveland, OH she currently lives in Washington, DC, which she said is truly the epicenter of healthcare policy and research in the United States. Because of her DC location, Alexandra has been able to attend several meetings and conferences as a PDSA representative, such as the Rally for Medical Research Day on Capitol Hill to advocate for NIH funding, the National Organization for Rare Disorders (NORD) Summit, the Patient-Centered Outcomes Research Annual Conference, various research training programs, and several meetings at the National Institutes of Health (NIH). She has her B.A. in Modern Languages and Literatures (Spanish and Arabic) with a Biology minor and Pre-Medicine concentration from Kenyon College, and completed a Post-Baccalaureate Program at Cleveland State University in Pre-Medical Studies. Alexandra also attended a Public Health program with the School for International Training in Buenos Aires, Argentina, where she studied public health policy, urban epidemiology, and health care equity, and researched barriers to healthcare for socioeconomically disadvantaged pregnant women in Argentina. Before joining PDSA, Alexandra worked as a Clinical Research Intern at Weill Cornell Medical Center with PDSA Medical Advisor, Dr. James Bussel. She was responsible for reviewing electronic medical records, interviewing and consenting patients, analyzing data, and providing administrative assistance to research coordinators. Alexandra has authored and collaborated on a number of abstracts through Weill Cornell and presented research at annual conferences for the European Hematology Association (EHA), the American Society of Hematology (ASH), and even at PDSA conferences! When she decided to train for the Cleveland Rite Aid Half Marathon this past spring, Alexandra wanted to use the event to raise awareness for a disease that has impacted every aspect of her life. Most people who know her know that she would never have labeled herself “a runner.” However, when she was training Alexandra thought about all of the people who have touched her life as a result of her involvement with PDSA. She knew the run would Alexandra Kruse, PDSA’s new Research Coordinator, with Dr. James Bussel at the 2016 ITP Conference in Florida definitely be worth it. She finished her half marathon in less than 2 ½ hours (in rain, hail, and 30 degree temperatures!) in the top quartile of half-marathon runners. To keep herself motivated while she ran she carried a list of ITP warriors in her pocket! Now she is training to run half-marathon #2 and to Sport Purple for Platelets in Washington! Alexandra described her relationship to ITP and PDSA this way. She was only seven years old when her mom, PDSA’s Executive Director Caroline Kruse, was diagnosed with Idiopathic Thrombocytopenic Purpura. She couldn’t even pronounce those words, let alone understand them. With her being the oldest sibling, her mom insisted Alexandra needed to be able to explain her mom’s condition in case they ever got into a car accident or she cut herself. As a result, Alexandra’s exposure to medicine began at a young age, often waiting at the hematologist for the results of a CBC or sitting with her mom during lengthy IVIg treatments. Instead of being fearful, she was empowered to learn as much as she could about autoimmune disease and bleeding disorders. Alexandra volunteered at ITP support group meetings and several PDSA conferences running mics during sessions, preparing conference materials, and helping with the Kids (CONTINUED ON THE NEXT PAGE) Platelet Disorder Support Association In Memory We received contributions from July 1, 2016 to September 30, 2016 in memory of: Talen Chao Gregory Chin Barbara Crawford Pamela Yukihiro In Honor We received contributions from July 1, 2016 to September 30, 2016 in honor of: Jessica Cohen Guests of Rebecca Antas’ Wedding Susan Frank Mark Jacobson David L. Johnson Barb Link Easton Parson Hayley Shimanek Cade Slad If you are considering an honorarium donation, please contact PDSA, 8751 Brecksville Road, Ste. 150, Cleveland, OH 44141 or [email protected] or call 1-87-platelet (1-877-528-3538) toll free. In Appreciation CONTINUED FROM PAGE 14 • Trish and Paul Santaromana • John Semple, PhD • Kimberly and Ronald Sewald • Hayley and Nathan Shimanek • Jody and Jon Shy • Jennifer and Aaron Slad • Michael Tarantino, MD • The Tillinghast Family • Kristen and Steve Tomczak • Kelly Torres • Lois Umhoefer • Stephanie Zane, Esq. • Cheri and Derek Zimmerman • Joyce and Dale Zimmerman If you have any questions about the CIRCLE OF HOPE, please contact PDSA at (877) 528-3538 or visit www.pdsa.org/hope to learn more. phone 1-87-PLATELET 17 PDSA Develops New Patient-Centered Research Guidelines for ITP Research Proposals By Alexandra Kruse Part of PDSA’s mission statement is “enhancing the lives of people with immune thrombocytopenia (ITP) and other platelet disorders through research.” When we received an opportunity to update our research protocol with the help of an award from the PatientCentered Outcomes Research Institute (PCORI), we knew our goal of working to engage the needs of ITP patients would remain our first priority when developing new investigative guidelines. At the 2016 ITP conference, the newlyestablished “PDSA Patient Research Panel” comprised of PDSA medical advisors Dr. James Bussel and Dr. Michael Tarantino, and five ITP patients and caregivers convened to discuss how research supported by PDSA should be conducted with the patient voice specifically in mind. The panel decided that research must be focused on clinically-oriented, observational, and laboratory research proposals related to primary ITP. Research methods must include evidence of consultation and/or collaboration with ITP patients. Funding should be allocated to hypothesis-driven research projects showing patient benefit, according to PCORI. Benefits could include “disease incidence, prevalence, burden, causes, prevention and management; gaps in evidence; potential for new evidence to improve health, well-being, and quality of care [such as patient qualitative studies to clarify symptoms or other quality of life issues of living with ITP]; patient needs, outcomes, and preferences; [and] relevance to patients and clinicians in making informed health decisions [about treating ITP].” Applications from investigators will be evaluated by PDSA’s Patient Research Panel. The PDSA Research Coordinator will screen proposals for proper adherence to our newly updated guidelines, and PDSA Medical Advisors will review for feasibility and relevance to ITP. After receiving updated proposals, grants will be awarded based on selection by the patient panel. Proposals will be evaluated based on importance, relevance, investigation plan, and interest to the ITP patient community. PDSA is especially grateful to our Patient Research Panel for their opinions in the creation of this new program. With their cooperation, we will be accepting grant applications starting this month to fund new and exciting ITP research, selected with the ITP community’s needs in mind. You can find the full text of PDSA’s Research Guidelines and donate to the research fund on PDSA Web site’s Research Program page https://www.pdsa.org/ resources/research-program.html PDSA Welcomes Alexandra Kruse — Its First Research Coordinator CONTINUED FROM PAGE 16 Camp. She gained insight into the lives of patients with rare and chronic diseases. From listening to heart-wrenching ITP patient stories, she learned the importance of patient advocacy, a correct diagnosis, the opportunity to see an expert in the field, and ensuring proper treatment. Although Alexandra loves research, she is drawn to the personal connection that comes with listening to the patient story, and she plans to apply to medical school this spring. 18 The Platelet News www.pdsa.org PDSA Local Support Groups PDSA has support groups across the United States and Canada and new groups continue to form. Listed below are existing groups with meeting dates. No group listed in your area? Consider forming a group and becoming a facilitator. We’ll provide you with a New Facilitator Kit to help you get a group started. Just contact Jody Shy at [email protected] or call toll free at 1-877-528-3538. For information on meeting locations, dates, and times please contact the group facilitator. ITP PARENTS TELECONFERENCE GROUP ATLANTA, GEORGIA RALEIGH, NORTH CAROLINA Facilitator: Jay Charness, [email protected] or (303) 731-7731 2016 Meetings: December 7 2017 Meetings: February 1, April 4, June 7, August 1, October 4, December 12 Facilitators: Joan Coppolino, [email protected] or (770) 924-2258 Facilitator: Amy Prokopowicz, [email protected] or (919) 280-0872 SOUTHEAST, GEORGIA CINCINNATI, OHIO Facilitator: Patricia Johnson, [email protected] or (229) 247-0699 Facilitators: Mary and Mark Griffith, [email protected] or (513) 922-4255 Currently looking for a Facilitator CHICAGO, ILLINOIS CLEVELAND, OHIO OTTAWA, ONTARIO - CANADA Facilitator: Trish Santaromana, [email protected] or (630) 292-1485, http://chicagoitpsupport.org Facilitators: Barbara Hise, fourstrings@ ameritech.net or (216) 310-3926 and Caroline Kruse, [email protected] or (440) 526-0629 PEORIA, ILLINOIS DAYTON, OHIO CANADIAN TELECONFERENCE GROUP Facilitator: Livia Chyurlia and Vanessa Clermont, [email protected] or (613) 612-9689 2017 Meetings: January 17, March 21 WATERLOO-WELLINGTON, ONTARIO CANADA Facilitator: Dale Paynter, [email protected] or (519) 651-2352 PHOENIX, ARIZONA Facilitator: Bunnie Stevenson, [email protected] or (480) 451-7661 ROGERS, ARKANSAS Facilitator: James Martin, [email protected] or (479) 270-7760 LOS ANGELES (SIMI VALLEY), CALIFORNIA Facilitators: Nina and Stephanie Schussman, [email protected] or (818) 370-8877 ORANGE COUNTY, CALIFORNIA Facilitators: Melissa Hilsabeck & Leilani de Castro, [email protected] or (714) 598-7102 PATTERSON, CALIFORNIA Facilitator: Carla Nelson, [email protected] or (209) 892-8813 SACRAMENTO, CALIFORNIA Facilitators: Dawn and John Phillips, [email protected] or (916) 607-1699 2016 Meetings: December 29 2017 Meetings: February 18 SAN DIEGO, CALIFORNIA Facilitator: Shanna Beard, [email protected] or (390) 692-5337 Facilitator: Tina Garland, [email protected] or (937) 643-1705 BOSTON, MASSACHUSETTS POWHATAN POINT, OHIO – SOUTHEASTERN OH/WV Facilitator: Michael Westfort, michael@platelet health.org or (508) 366-1073 or (508) 930-0309 DETROIT, MICHIGAN Facilitators: Sharon Cisco and Linda Galka, [email protected] or (586) 783-8014 ST. PAUL, MINNESOTA Facilitator: Joe Winter, [email protected] or (651) 792-5277 OMAHA, NEBRASKA Facilitator: Heidi Green, [email protected] or (402) 498-3826 CENTRAL/NORTH NEW JERSEY Facilitator: Linda McGuirl, [email protected] or (908) 764-1819 NORTH NEW JERSEY Facilitators: Dianne Danielle, [email protected] or (201) 265-8875 and Lanie Gastman, [email protected] or (201) 592-1181 Facilitator: Camie Sims, [email protected] or (304) 816-2608 POCONOS, PENNSYLVANIA Facilitator: John Catalano, [email protected] or (917) 892-4264 RHODE ISLAND Facilitators: Lisa & Steve Sack, [email protected] or (401) 884-5711 CHATTANOOGA, TENNESSEE Facilitator: Sharon Putnam, [email protected] or (423) 991-6450 NASHVILLE, TENNESSEE Facilitator: Charity Hasty Backs, [email protected] or (615) 473-3372 AMARILLO, TEXAS (PANHANDLE) Facilitator: Robin Abshire, [email protected] or (337) 296-7052 DALLAS FORT WORTH, TEXAS SOUTH NEW JERSEY Facilitator: Michael Vitale, [email protected] CAPITAL DISTRICT (ALBANY AREA), NEW YORK Facilitators: Linda and Kris Dorasami, [email protected] or Linda (817) 7272351 or Kris (817) 727-2361 and Marsha Inman (817) 249-5391, [email protected] Facilitator: Susan Pounder, [email protected] or (858) 217-6587 Currently looking for a new facilitator SALT LAKE CITY, UTAH LONG ISLAND, NEW YORK WASHINGTON, D.C. Facilitator: Claudia Montuori, [email protected] or (718) 725-2645 Facilitator: Cory Bushman, [email protected] or (801) 404-4580 Facilitator: Cindy Ayliff, [email protected] or (703) 849-0054 TRIANGLE, NORTH CAROLINA Facilitator: Barbara Pruitt, [email protected]; (305) 409-6887 Facilitators: Donna Goldstein, ds.goldstein@ mindspring.com and Emily Goldstein, [email protected] or (919) 942-4082 NORTH FLORIDA CHARLOTTE, NORTH CAROLINA Facilitator: Irene Xynides-Rincon, xynidesi@ bellsouth.net; (904)710-9055 or (904) 823-9055 Facilitator: Carol Mullis, carolhmullis@ windstream.net or (704) 843-2734 MIAMI, FLORIDA SEATTLE, WASHINGTON Facilitators: Tammy Fassett, [email protected] or (206) 465-3451 and Taylor White, [email protected] or (253) 250-5873 MILWAUKEE, WISCONSIN Facilitators: Kim Everett, [email protected] or (920) 755-8430 and Trudy Sensat, [email protected] or (414) 202-6291 Platelet Disorder Support Association phone 1-87-PLATELET 19 News from Our Support Groups Our support groups are the grassroots that help us BUILD STRENGTH THROUGH SUPPORT and ADVANCE ADVOCACY THROUGH EDUCATION. When you share your personal stories with others, you give hope to those who may have felt all alone in dealing with their ITP. We hope you will find comfort in knowing that whether you are part of a meeting to listen, talk or both that you are not alone. We encourage you to make your medical providers, family and friends aware of our numerous resources that help make the World of ITP a more manageable place to be. It’s this strong network that will continue to create awareness, educate, and advocate. Here are some inspirational stories and news we’ve received from our support groups recently. To share your group’s news, please contact Nancy Potthast at [email protected] Atlanta, GA Support Group Meeting Facilitator: Joan Coppolino [email protected] or 707-924-2258 The group warmly welcomed a new attendee to their August 27th meeting and discussed updates from ITP Conference 2016 in Orlando, FL. Central/North NJ Support Group Meeting Facilitator: Linda McGuirl, [email protected] or 908-764-1819 The group welcomed four new members to their September 20th meeting with 13 in attendance. The engaged group reviewed ITP Conference 2016. Attendees shared their experiences with ITP, discussed the need to be one’s own advocate, and reviewed personal experiences with treatments (side effects, successes and failures). They celebrated what lifts their spirits — meditation, energy, healing, daily exercise, good nutrition — and agreed to reflect on five things to be grateful for upon awakening each day. welcomed 15 attendees (familiar and new) who shared their journeys in living life with ITP! It was a wonderful afternoon with the first PDSA ITP Network Meeting at the historic Runnymede Library in Bloor West Village. The meeting offered great discussions about treatments and what is working, not working and wishing their doctors had the resources they have through groups like PDSA. The group also wants all its American friends to come up and see them sometime! Sacramento, CA Support Group Meeting Facilitators: Dawn and John Phillips [email protected] or 916-607-1699 Facilitators Dawn and John welcomed two new families to the group at the October 18th meeting in Sacramento. The newcomers (one newly diagnosed, Waterloo-Wellington/Greater Toronto Area, Ontario – Canada Support Group Meeting Facilitator: Dale Paynter [email protected] or 519-651-2352 The Waterloo-Wellington group expanded their area, held their October 16th meeting in Toronto, and Members of the Sacramento, CA Support Group sport purple at a game with ITP Warrior Josh Phegley (catcher for the Oakland A’s) in July. ITP Warrior and Sacramento group co-facilitator John Phillips is pictured (center, left) with Josh Phegley (center, right). the other an ITP veteran) found comfort in hearing the journeys of others, were relieved to uncover resolution to unanswered questions, and are grateful to have new sources of support through the group and PDSA. Chicago, IL Support Group Facilitator: Trish Santaromana [email protected] or 630-292-1485 Happy Anniversary to all who have shared their ride on the ITP Rollercoaster in Chicagoland for the past nine years! Parents ITP Teleconference Support Group Facilitator: Jay Charness [email protected] or 303-731-7731 The ITP parents had a great teleconference call on Aug. 3rd with several families joining in on the call. The first half hour was dedicated to the kids giving them time to share their ITP and life experiences. Parents on the call had an outstanding question and answer session with ICON Pediatric Hematologist Robert Klaassen, MD. Questions and answers from the call are printed in this issue of The Platelet News. Following the Q&A session, parents discussed many other topics of concern for their children with ITP. The next teleconference call is scheduled for Dec. 7th, 7 pm EST. 20 The Platelet News www.pdsa.org Parents Get Answers During Q&A with ICON Pediatrician Dr. Robert Klaassen By Carol Hoxie During PDSA’s Aug. 3rd ITP Parents’ Teleconference Support Group call parents asked their questions of ICON pediatric hematologist, Dr. Robert Klaassen. He is Professor of Pediatrics in the Division of Pediatric Hematology/ Oncology at the University of Ottawa in Ontario, Canada. Dr. Klaassen has been on staff at the Children’s Hospital of Eastern Ontario (CHEO) since 1998. His current area of research focuses on quality of life of patients with hematologic disorders. His clinical expertise is in benign hematology including bleeding disorders. Dr. Klaassen is a Site Investigator for ICON (ITP Consortium of North America), a collaborative research effort dedicated to improving the understanding, treatment, and quality of life of pediatric ITP patients. ICON is pleased to partner with and work in collaboration with PDSA. Dr. Robert Klaassen, Pediatric Hematologist from the University of Ottawa in Ontario, Canada answered questions during the August 3rd Parents’ Teleconference Call Here are the parents’ questions and Dr. Klaassen’s answers. Q: Our child’s migraines are triggered Q: Are there long-term effects of the when her platelets are low. We have a 30 year-old house and recently got an ionizer that kills mold. Is mold something that triggers ITP? A: Infection is a trigger for ITP. It’s important to do anything you can to avoid infection, as it will drop platelet counts and might exacerbate ITP. platelet growth drugs? A: These treatments have been used for ITP about 7-8 years in addition to the years of clinical trials. Romiplostim (Nplate®) is injected, eltrombopag (Promacta®) is a pill. These work equally well and seem quite safe. We are getting more data as time goes on. Promacta was approved for use in children in 2015. Q: Is there a chance for remission from ITP after being on Nplate? A: About 5 – 10% of patients on Nplate or Promacta have gone into remission for a while after treatment was ended. The question is: Was it the drug that led to the remission or was it from the natural history of the disease? In children a majority of ITP patients go into remission. Q: What other ITP treatments are you and ICON working on? Q: If one of these two drugs doesn’t Q: On IVIg our child’s platelet counts A: The ICON study has 26 centers. work well or has bad side effects, is it worth switching to the other one? A: Just because someone gets side effects with Promacta doesn’t mean they will have the same effect with Nplate. It’s worth a try. were up and down and then was treated with Dapsone. First, the count was 48,000, then fell to 30,000 and recently to 20,000. Our hematologist is thinking of trying Promacta. A: Dapsone works in some ITP patients. Some patients respond, some don’t. Again, was it the Dapsone doing something, or was it the natural history of the child’s ITP? As to treatments, Rituxan® (the most common one used) is similar to IVIg but is directed at one type of cells in the immune system (B cells that make antibodies). Rituxan works in about 2/3 of patients with initial response. About 1/3 get a longer response time. As to side effects we see some infusion reactions, blood pressure changes, and hives. The downside is higher risk of infection after treatment. Risk is lower if we give Rituxan and no other drugs at the same time. There is also research on drugs that stimulate platelet-making cells (megakaryocytes), whereby pieces bud off to make platelets. These growth drugs are very effective, working in about 80% who get them. However, they only work as long as you take them and are very expensive. Q: Our child bleeds at lot at an 8,000 count. We try to treat when the count is down to 10,000. A: I have some patients who at 5,000 platelet counts don’t bleed. However, if a child bleeds at a particular level, they should be treated. Q: What about possible head injuries at low counts? A: Studies have shown about a 1-2% rate of bleeds in the head. There’s about a 99% chance of not having a head bleed. In a study in Switzerland with 3,000 kids, they only had three bleeds (a toddler, an elementary school age child, and a teen). Q: What is your general consultation about ITP children playing standard school sports and what is a safe platelet level? A: We did a survey of US doctors published in Pediatric Blood and Cancer. We got lots of different responses. On any given day you can’t know exactly what your child’s count is. It could be or down. Try to get a picture of what your child’s trend is. Do avoid contact (CONTINUED ON THE NEXT PAGE) Platelet Disorder Support Association What Our Friends Are Saying on Facebook PDSA has a Facebook page that provides a direct online forum for comments and sharing news and information about ITP, treatments, and the ups and downs of having this platelet disorder. If you LOVE PDSA, then be sure to: www.facebook.com/plateletdisorder Note: The views expressed are personal experiences and are not endorsed by PDSA. Here are some recent comments we’ve received on our Facebook page: Comments on fatigue: “The fatigue is like nothing I’ve ever experienced in my life. I have had weeks where I could only muster the strength to roll over in bed. This disease is a dirty rotten scoundrel! It’s 100% a symptom. All autoimmune diseases have fatigue as a symptom; your body is hell-bent on destroying itself. It is exhausting!” – Tara C. “I was diagnosed 15 years ago…fatigue is real. Sadly, for years I was told it was all in my head.” – Shannon K. “Fatigue is really horrible because my brain is telling me to get up and go but my body just won’t oblige. I was told by one of my consultants that ITP doesn’t cause fatigue. What does he know — obviously not a lot — he should try having it!” – Stephanie S. “Every single time I tell the nurse that my husband is tired/exhausted she’ll say, ‘His hemoglobin is fine.’ Ugh!” – Jill F. phone 1-87-PLATELET 21 Parents Get Answers During Q&A with ICON Pediatrician CONTINUED FROM PAGE 20 sports, so no rugby, football or ice hockey. These all have head injury risk. Stay away from sports that involve slamming into others as part of the game. With basketball there is some banging into others and bruising. Soccer could be okay as long as there’s no ‘heading’ and leg injuries are not usually life-threatening. In general, let your child have as normal a life as possible. Don’t put them in a bubble. Have them wear helmets for sports and use common sense. Q: What about fatigue in kids with ITP? I notice a difference in my daughter. She’s more active at 70,000 but her endurance is off at 20,000. It’s like she hits a wall. A: In our study, we looked at fatigue. We found that the group got fatigued when counts were low. It might seem counterintuitive but the best antidote for fatigue is some activity — keep moving. Don’t just let the kids sit on the couch and watch TV or play video games. It’s important that kids get outside and get some exercise. Q: In terms of splenectomy, what are your personal parameters for your patients? A: For ITP patients, the younger you are the greater the risk as removing the spleen increases risk for infections. If kids are over age 5, the risk is lower. As kids get older they have fewer fevers and infection concerns. We wait until kids have had ITP at least a year, as many kids go into remission by then. For kids who have Evans syndrome (red cell breakdown) and ITP, we avoid splenectomy. For kids who are age 6 or older and have ITP more than a year, it is a possible solution. We found from a quality of life (QOL) viewpoint, it can be a dramatic change for the ITP child and their family. If splenectomy works and you see them much later, life is much better for them. 22 The Platelet News www.pdsa.org Advocacy Alert NEWS OF PDSA ADVOCACY ON BEHALF OF PATIENTS WITH ITP AND OTHER PLATELET DISORDERS NORD 2016 Rare Diseases & Orphan Products Breakthrough Summit By Caroline Kruse PDSA Executive Director Caroline Kruse, Marketing Director Nancy Potthast, and Research Coordinator Alexandra Kruse were three of more than 600 members of the rare disease community who came together for the National Organization of Rare Diseases (NORD) annual conference, October 17 and 18 in Arlington, Virginia. The “Rare Diseases & Orphan Products Breakthrough Summit” was a two-day event focused on fueling collaboration to advance progress for patients. It featured expert speakers from multiple stakeholder groups, including patients and patient groups, FDA, NIH, industry, researchers, academia, and venture capitalists, among others. This year, two themes emerged as necessary for continued progress: collaboration and involving the patient in every step along the way. Speakers discussed national election implications, federal and state policy issues, collaboration nationally and internationally, barriers to access, and new drug development. There were also networking opportunities on how to drive progress for people with rare disease. Keynote speakers included: FDA Commissioner Dr. Robert Califf spoke of the obligation of public health agencies to foster collaboration with patient advocates. Calling this an era of remarkable progress, he announced more than 20 new grants, totaling $23 million, to fund rare disease research in the coming years. Dr. Califf praised NORD and its Summit for marshaling the strengths of the rare disease community, saying, “This organization brings together so many different experts across the different spectrums and most importantly the patients and caregivers who provide the best resources in providing expertise.” Dr. Janet Woodcock, Director, Center for Drug Evaluation and Research (CDER), FDA, stated there is no better time for patient involvement in the rare disease drug development process. She discussed the importance of natural history studies and called for greater collaboration between researchers through consortia. Dr. Peter Marks, Director, Center for Biologics Evaluation and Research (CBER), FDA, said we are on the threshold of the first gene therapy approval. He discussed CBER’s goal to accelerate progress and get important therapies for rare and orphan diseases to patients. His reviewers get tremendous satisfaction and help by meeting with patients and advocacy groups to understand what they face with their diseases. Here are highlights from the NORD Breakthrough Summit: Focus on Pediatric Diseases – Noting that the process of achieving pediatric labeling needs to move more quickly, Lynne Yao, MD, FDA, said, “It takes a long time, currently 9 to 10 years on average, to get from adult approval to pediatric labeling,” adding there are now 637 products labeled with pediatric-specific information. Potential Advances through Genetic Innovation – There is an explosion of gene studies with approximately 530 new Investigational New Drug applications. Collaborations Across Borders – The panel endorsed patient-driven registries to collect natural history data and NORD President and CEO Peter L. Saltonstall with PDSA Executive Director Caroline Kruse at the NORD Members Membership Meeting suggested a global depository system, similar to those used for HIV and vaccine treatments, to expand access and care across the globe. The Crucial Role of Data – All members of the panel, representing medical professionals, academics, drug developers and FDA, agreed this is an exciting time in drug development. “We are sort of entering the era of patient-empowered medicine,” said Matt Might. Driving Progress through Policy – Paul Melmeyer and Tim Boyd of NORD’s Policy team said upcoming legislation will affect the rare disease community, including the important vote for 21st Century Cures during the 3-week lame duck session, and looked ahead to 2017 issues. Strategies to Address Patient Challenges – Before a packed room, speakers tackled a series of practical issues that patients face. During an emotional session, undiagnosed individuals spoke of the stress and pain of not getting answers. (CONTINUED ON THE NEXT PAGE) Platelet Disorder Support Association phone 1-87-PLATELET 23 ITP in Kids: Q&A ITP CONFERENCE 2016 • DR. MICHELE LAMBERT AND DR. MICHAEL TARANTINO By Alexandra Kruse Q: My son has tried every treatment, and is now keeping his platelet count up on Promacta® and dapsone. Can he expect remission? A: Dr. Tarantino: It’s very common to see a real remission with both TPO agents (Nplate® and Promacta®). The Bleeding and Clotting Disorders Institute was part of the original Promacta trial (REPEAT), and there was at least one patient who had a sustained elevation in their platelet count without Promacta after the study ended. At the time we thought it was a fluke, but every year we hear about more cases of patients whose counts remain elevated. Both Amgen and Novartis, the pharmaceutical companies who make the TPO agents, are interested in the mechanism of why TPO induces remission and identifying those patients who are likely to achieve remission. Q: Our daughter’s platelet count has been at 100,000 for a year on Promacta. Last week her count was 25,000. Have you ever seen that after a year on a drug the patient’s platelet count plummets? A: Dr. Lambert: Yes, we would first look to determine whether there is something else occurring immunologically based on your CBC, or whether there isn’t something other than the ITP causing issues, but sometimes there is just a loose response. Dr. Tarantino: Because medications are biomolecules, there is a small chance that they will be recognized by the immune system after time and the patient will develop antigens against the medicines. However, a study done in Europe found that switching a patient from Promacta to Nplate has an 80% success rate in re-elevating platelet count, and switching from Nplate to Promacta has a 50% success rate. Q: Can you explain dapsone? A: Dr. Tarantino: Dapsone is a less frequently used immunomodulating drug that historically was used as a thirdline agent. The success rate in children with chronic ITP is mostly anecdotal with a 50% response rate, and usually takes a while from beginning of treatment to platelet elevation. Q: For those who have a good response to TPO, how high do you let their platelet counts go and where do you start to get concerned? Where do you get nervous and where do you draw the line for levels that are too high and that it could cause a clot? A: Dr. Tarantino: Initial assessment has shown that there isn’t a correlation between platelet count and an event of thrombosis in Promacta. In those who experienced clotting in an adult clinical trial, 80% had another prior risk factor for clotting that was unrelated, such as a fractured ankle and the use of estrogenbased oral contraceptives. Thrombotic events did not occur in the children’s Promacta trial. The goal of TPO agents is to achieve a hemostatic platelet count, above 50,000. Your hematologist should wean the dose after a platelet count of 200,000 and stop administering treatment if the platelet count is consistently above 400,000. NORD 2016 Rare Diseases & Orphan Products Breakthrough Summit Predicting the Pipeline in 2017 – NIH, FDA and industry representatives spoke about rising trends in orphan drug designations and the positive outlook for continued high activity. Orphan drugs tend to be innovative (based on data showing what percentage are first- Q: We have read some reports on low-carb diets leading to remission. What’s your opinion? A: Dr. Lambert: It’s important to maintain a healthy and balanced diet as well as consistently exercising. Many ITP patients have fatigue and are prone to depression, so it is important to maintain as much of a healthy lifestyle as possible in order to be the healthiest person they can be. Diet and exercise definitely play into ITP as well as stress, which can affect your platelet counts. Q: How often do you recommend treatment with IVIg and how often should we take our son in for a CBC when he is playing a sport? A: Dr. Tarantino: Use altered doses of IVIg at altered frequencies to keep the platelet count above requisite level so that your son can participate in his activity. IVIg treatment is done in a secondary mode, during the sport season, but then we will wean them off of the medication once the season is over as long as their counts are in a safe range. You should monitor counts fairly frequently, every other week, but this is dependent on bleeding level, activity and severity. Above 100,000 is the threshold for unrestricted physical activity. Dr. Lambert: However, we do sympathize in telling a patient that they are not allowed to participate in an activity that they love and is a huge part of their life so we as pediatricians integrate that into our discussion as well. CONTINUED FROM PAGE 22 in-class) and there seems to be growing willingness among developers to expand into new conditions. 24 The Platelet News www.pdsa.org ITP in Adults: Q&A CONTINUED FROM PAGE 11 effects of getting rituximab? A: Dr. Kessler: We know from lymphoma and lymphocytic leukemia patients using rituximab that knocking out B cells can downregulate (decreases the immune response of) certain T cells. In some patients we do see where long-term rituximab can lead to immune deficiency syndrome in adults as well as children. Q: If you use rituximab once and it doesn’t work, is it something you’d recommend trying again a second time? A: Dr. Kessler: All of us, I think, have different philosophies. I think it’s important to know what doses of rituximab are given. I’ve seen a series of patients that had received rituximab at 100 mg/m², which had been popularized by an article in the UK. They had indicated that that dose was adequate for the treatment of ITP. Most of us on this panel don’t think that is an adequate dosing regimen for ITP. Most of us use the traditional dose that is recommended for lymphoproliferative malignancies. As far as failure on one series and redosing, my own theory is I would go on to something else. But I don’t think there’s anything wrong with redosing but it has to be justified. Dr. Kuter: Who do you declare a ‘rituximab failure’? One of the problems is the typical oncologist will treat the ITP patient with three doses at the typical oncology dose level and if it hasn’t worked yet, declare it a failure. As Dr. Kessler has shown usually you see the true immune response and the rise in platelet counts after weeks three and four through maybe 10. I would probably make sure a whole course (4 weeks) is given and wait maybe a month or two before deciding if it hasn’t worked. Then if it is a failure, I would probably not go back and use it again. The doctors on the panel discussed dosages of rituximab they give to treat ITP. Drs. Kuter, Gernsheimer, and Liebman all give 375 mg/m². Dr. Liebman combines that dose with dexamethasone. Dr. Kessler has seen when some patients who also had another autoimmune disorder (such as lupus) were given the higher lupus dose (400 mg/m²) they responded well. Q: Treatments like rituximab or splenectomy depend on reducing antiplatelet antibodies. Has anyone noticed if they are less effective in ITP patients who are antibody negative? A: Dr. Arnold: A number of years ago it was thought antiplatelet antibodies could be a biomarker for ITP and then that turned out to be a not-so-great test. There were some refinements to the test but it still hasn’t turned out to be a great biomarker. In small studies we had of patients who had gotten rituximab we looked at platelet antibodies before and after their treatments. Only about 40% of patients had a detectable antibody by very specific, sensitive techniques. When they got rituximab, their antibody levels dropped more than the placebo group but levels didn’t respond with how well they would do. Rituximab can reduce antibody levels. However, reduction in antibody levels did not seem to correlate with improvement in outcomes. Dr. Semple: The data Dr. Arnold mentioned is from mouse studies. With mice you can make ITP with one set of antibodies or another and can see the difference in those antibodies. Most ITP patients have multiple types of antibodies. You’re not going to be able to get patients with just one type of antibody. We know that certain antiplatelet antibodies are good at getting platelets destroyed and others are good at shutting them off (inhibiting platelet function). So which is worse? Decreasing the count or causing patients to have more bleeds? Q: What is the long-term outlook for ITP patients? Is remission likely? A: Dr. Arnold: There’s an interesting concept about treating ITP of ‘hit hard, hit early’ to bring patients to remission. That’s worth studying some more. The argument can be made the other way — ITP antibodies can accumulate over time but can also disappear over time. It doesn’t mean the ITP is getting worse or better, it’s just evolving. And some patients just get better on their own, so there’s a burnout rate of the disease as well. Dr. Bussel: I agree it kind of depends on the patient and we don’t always know which patient is which. Studies have shown treating with rituximab very early or with high dose dexamethasone are curative. Our own studies showed if you treat within a year you can cure some people with ITP. I think maybe certain treatments look worse over time because some patients get better over time and are no longer being treated. Dr. Kuter: A doctor in Europe who followed 187 ITP patients over five years found 82 percent of the patients who didn’t have splenectomy had a remission and came off therapy after five years. Her findings show that ITP is a highly variable disease. Dr. Bussel: Right, though I wouldn’t want the audience to think that 82% of adults are likely to get better in five years based on the best of our experience here in the U.S. Dr. Kuter: On the contrary, I would like the audience to hear that. It gives them optimism that there’s a light at the end of the tunnel. Q: What about switching between the two TPO agents? A: Dr. Kuter: We looked at patients on romiplostim and eltrombopag, not doing well on their respective treatment. When they were switched to the other agent, 83% of switched patients responded well. Switching is worth a try. 26 The Platelet News www.pdsa.org Living with ITP – Patients Share Their Stories ITP CONFERENCE 2016 PANEL: DALE PAYNTER, KATIE MELOY, YVONNE MOLINO, AND KEITH ORI By Carol Hoxie Each ITP conference features a “Patient Stories” session, where several members share their ITP journey with the audience. Hearing their fellow ITP patients’ experiences helps audience members with ITP realize they are not alone. They learn which treatments worked, which didn’t, and coping strategies. This year’s international panel included speakers from the US, Canada, and the UK. First was Katie Meloy, a 21-year-old from York, UK, diagnosed with ITP in January 2015. After diagnosis she felt her hopes and dreams were being taken away. Life would never be normal. With counts as low as 2,000 and as high as 160,000, she experienced the “emotional roller coaster” that is ITP. When put on steroids Katie suffered sleep deprivation, weight gain, and hallucinations. She found Sandi Zancha on PDSA’s Discussion Board and the PDSA Web site, which made her feel less alone. IVIg caused her serious side effects. After treatment with Rituxan® she is in remission, with stable 200.000+ counts. Katie said, “I have ITP — ITP does not have me.” Contacting people across the world through social media she put together an ITP awareness video that can be viewed on YouTube (search for ‘ITP awareness video’). Katie said, “Without ITP I definitely wouldn’t be the person I am today.” Dale Paynter, from Toronto, Ontario, Canada has ITP since he was diagnosed in 2010 after a company physical found an 80,000 platelet count. When it fell to 30,000 he was sent to a hematologist. Bone marrow testing showed healthy platelet production. He tried prednisone This year’s engaging ITP patient panel included: (L to R) Keith Ori, Yvonne Molino, Katie Meloy, and Dale Paynter, moderated by PDSA Executive Director Caroline Kruse (C) but it didn’t help. In 2011 Dale was planning a golf trip to South Carolina; his hematologist recommended IVIg treatment. It raised his count to 350,000. He’s been on IVIg treatments twice per month ever since. After several years Dale was tired of constant IVIg treatments so during fall 2014 he had a splenectomy. Unfortunately it didn’t work. He tried a six-month fostamatinib clinical trial but that didn’t raise his counts. He is back to twice-monthly IVIg. Dale’s PDSA connection began when he attended the 2011 ITP conference in Boston, MA. He has attended every conference since. He runs the WaterlooWellington, Ontario support group. Three years ago he joined PDSA’s Board of Directors. ITP has changed his life. He is more careful around home and he no longer plays ice hockey as he did for many years. Dale considers himself one of the lucky ones, with few petechiae and little bruising. He said, “You don’t have to let ITP take over your life.” Yvonne Molino, of Longs, SC is the parent of a 10-year-old son with ITP and shared his story. Clinch was diagnosed with ITP at age 2. He was hospitalized nine days and received IVIg and WinRho®, but there was no real change in his platelets. When they rose from 1,000 to 3,000 Yvonne was excited until the doctor said, “Well, it’s kind of like whether you have a thousand pennies or three thousand pennies, you’re still kind of broke.” Clinch was covered in bruises and had bleeding mouth blisters. His bone marrow test was normal. They started him on steroids. Doctors said it was probably ‘acute’ ITP and the low counts wouldn’t happen again. Four months later he was in the hospital again. This routine of platelet counts bottoming out and getting treatments lasted until age 5. Between ages 5-7 Clinch had two hospital visits. Yvonne thought he was growing out of it. Around age 7 -8 he was sick again, his counts ranging from 2,000 to 40,000. Then Yvonne found PDSA and got involved. She said, “We realized then this was probably ‘chronic’ ITP.” We tried rituximab for Clinch. It lasted seven months with a normal platelet count. He is doing well on IVIg, rituximab, and immune suppressant. Yvonne shared her insights as an ITP parent: 1) Remember every person with ITP is different; what works for one may not work for another. 2) Try to not limit your child’s activities. Don’t put them in a bubble. ‘No’ all the time creates negativity and may result in them being depressed and angry. 3) Don’t ‘compensate’ for your child’s illness by buying things and spoiling him/ her. Our doctor said it’s not good to spoil as that child has to face real-life in the world. Help them be prepared for that. 4) Get involved for ITP and PDSA. We take part in Sport Purple for Platelets Day and are doing a fundraiser this year. (CONTINUED ON THE NEXT PAGE) Platelet Disorder Support Association phone 1-87-PLATELET 27 Kids Korner By Carol Hoxie Teen’s School Fundraiser Increases ITP Awareness and Raises $200 for PDSA Dear PDSA, My name is Cade Kleisner and I have ITP. I discovered I had ITP when I started to spot bruises and petechiae on my chest and face in January of my 6th grade year. Thanks to my homeroom and ELA teacher Mrs. Schlaak, who had a son with ITP and recognized the signs, I saw my doctor and got the right diagnosis and help I needed. It started out rather rough and after reaching a low 9,000 platelet count, I had my first treatment with IVIg. It worked. After that my platelets declined again and I had to get another treatment. This process continued for quite a while. I also switched to prednisone and dexamethasone treatments. Currently I’m an 8th grader and attend Shattuck Middle School in Neenah, WI. My platelet levels now rest at about 70,000. Thanks to Ben Jones, a close friend of mine, as well as guidance counselor, Mrs. Jamie Lasee, we were able to include the PDSA in the yearly Shattuck Penny Drive where we donate to multiple support groups of different disorders. I also wanted to thank you for supplying me and my family with tons of helpful information. It has definitely made my experience with this blood disorder a heck of a lot easier. PDSA Note: Cade’s fundraiser raised ITP awareness and a $200 donation to PDSA. Thank you! ITP Warrior Donates $150 in Allowance Money to PDSA Hi, My name is Kai and I am 8 years old. I have had ITP since I was 2. I have tried many different treatments, including Promacta®, WinRho®, Nplate®, Cellcept, prednisone, and my typical treatment is Kai, an 8-year-old ITP Warrior saved his allowance for 18 months and donated $150 to PDSA IVIg. I have saved my allowance for 18 months, and I have donated $150.00. Thank you PDSA for all of the hard work that you have done trying to find cures for ITP. PDSA Note: Thank you to Kai for saving up his allowance to give to PDSA to help others with ITP! If you have an interesting story or news about a child/teen with ITP, please send to PDSA at [email protected] or mail to: PDSA, 8751 Brecksville Road, Suite 150, Cleveland, OH 44141. Living with ITP - Patients Share Their Stories CONTINUED FROM PAGE 26 Keith Ori, from Orlando, FL was the last speaker. He buys and flips houses on a reality TV show, Zombie House Flipping. He was fine until 2010 when he experienced fatigue, brain fog and collapsed while playing with his kids. It was one of those “something is really wrong” moments. At the doctor he learned he had a 20,000 platelet count. He received pulse dexamethasone and had a bone marrow test. His hematologist suggested splenectomy, telling him 2/3 of ITP patients get better, although 1/3 don’t and get worse. Keith promptly saw a different hematologist. His count was around 16,000. His doctor said since Keith was Rh+ he could get WinRho®. So he got the infusion. Unfortunately, later at home, he suffered a rare severe hemolytic reaction to WinRho®, a reaction that has caused death in some ITP patients. On prednisone Keith wasn’t much fun to be around. He tried IVIg and Rituxan® in 2011 but it didn’t help. He got the Indium platelet screening test done outside the U.S., which showed his platelets were destroyed mainly in his spleen. He graphed his platelet counts. He noted they rose in the winter and fell in the summer. Keith has seasonal allergies and said in Orlando they use air conditioning a lot in the summer. After adding onto their house in 2007 they experienced trouble with the AC system, including condensation and lots of mold. When he put a UV light in the unit, it killed the mold. After that his platelet counts rose by 2013 to the 100,000 – 150,000 range. Keith asked, “So, after six years of ITP am I cured?” He encouraged the audience to put everything into finding out what might be causing their ITP. NONPROFIT ORG U.S. POSTAGE PAID CLEVELAND, OH PERMIT NO. 2280 8751 BRECKSVILLE ROAD, SUITE 150 CLEVELAND, OH 44141 Postmaster: Send address changes to PDSA 8751 Brecksville Road, Suite 150 Cleveland, OH 44141 Our Mission THE PLATELET DISORDER SUPPORT ASSOCIATION IS DEDICATED TO ENHANCING THE LIVES OF PEOPLE WITH IMMUNE THROMBOCYTOPENIA (ITP) AND OTHER PLATELET DISORDERS THROUGH EDUCATION, ADVOCACY, RESEARCH, AND SUPPORT Don’t Miss a Single Issue! You receive The Platelet News as an exclusive benefit of your annual PDSA membership. When it is time for you to renew your membership, we encourage you to make your gift online. This saves PDSA time and money, puts your gift to work immediately, and helps ensure that you don’t miss a single issue of The Platelet News. To renew your gift online, simply go to http://www.pdsa.org/renew To receive an e-mail reminder when your membership is about to expire, make sure that you have opted-in to receiving e-mail from PDSA. If you are not currently receiving e-mail from PDSA, please send an e-mail to [email protected] to learn more. THANK YOU TO FOR SUPPORTING PUBLICATION OF OUR NEWSLETTER. If renewing your gift online is not convenient, you can call us at (877) 528-3538 to make your gift over the phone, or to request that a gift/pledge card and reply envelope are mailed to you. No matter how you renew your membership gift, we are truly grateful for your support and for all that your philanthropy enables us to do for the ITP community.