The HBO Max medical drama THE PITT, winner of five Emmy Awards including Outstanding Drama Series, continues to spotlight complex and emotionally charged emergency cases. In Season 2, Episode 3, the show follows a busy Fourth of July shift at Pittsburgh Trauma Medical Center—an episode that includes an especially meaningful storyline: the diagnosis of Immune Thrombocytopenia (ITP) in a young patient.
Paris, December 23, 2025 – The European Commission has approved Wayrilz (rilzabrutinib), a novel, oral, reversible, Bruton’s tyrosine kinase (BTK) inhibitor, as a new treatment for immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. This follows the positive opinion by the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP).
- From sanofi.com press release
A non-profit organization that operates in pediatric hospitals is bringing “healing through happiness” to children battling serious illnesses in medical centres, officials say.
Campfire Circle delivers camp-inspired programs to pediatric hospitals across Ontario, including London Health Sciences Centre’s Children’s Hospital, where the organization served more than 500 children over the past year, said the charity’s director of programs and impact, Jenny Edmonds.
- From The London Free Press

We’re proud to announce that PDSA President and CEO Caroline Kruse was honored with a TORCH Award for her outstanding leadership and dedication to the rare disease community. Presented at Sanofi’s headquarters in Boston, this award recognizes individuals who go above and beyond to make a meaningful impact—and Caroline’s tireless advocacy for patients with immune thrombocytopenia (ITP) and other platelet disorders continues to inspire and elevate patient voices and the lived experience.
Caroline accepted the award alongside fellow changemakers, PDSA staff Brenda Foster and Jody Shy, Medical Advisor James Bussel, MD, and her husband Ken, also an ITP patient, in a moment that highlighted the power of community and collaboration. Her presence at the ceremony was not just symbolic—it was deeply personal. As someone who has walked the path of ITP herself, Caroline brings a unique perspective to her leadership: one rooted in empathy, resilience, and a profound understanding of what it means to live with a rare disease.
Her journey with ITP has shaped every facet of her work at PDSA, from championing patient-centered research to building bridges between clinicians, researchers, and the patient community. Caroline’s leadership is defined not only by strategic vision, but by a heartfelt commitment to ensuring that no patient feels alone, unheard, or unseen.
You can watch Caroline’s brief interview below, where she shares her personal ITP experience and reflects on how it has informed her approach to advocacy, leadership, and building a more inclusive future for all those affected by platelet disorders.
The UCLA Health patient was diagnosed with a platelet condition that puts him at high risk of bruising and bleeding
"Sei Nagashima was an ebullient 4-year-old enjoying his first day of preschool in Porter Ranch when a plastic swing hit him lightly on his left eye. By the time his parents arrived to pick him up, the scratch had become a bruise that soon blossomed into a black eye."
- From uclahealth.org. Written by Martha Groves. Photo courtesy of Taku Nagashima.
"After a 19-year-old man developed "a strange rash on his arms and legs" and noticed blood in his urine, he learned that a platelet disorder was preventing his blood from clotting—and tests revealed a surprising trigger."
- From advisory.com. Lisa Sanders writes for the New York Times Magazine.
"For the past year I religiously text my husband, Shea, every Friday afternoon. There’s nothing unusual in that, but the weekly message contains just a number, no words. Triple digits and we’re happy; single digits and it’s time to get family in to mind the children and pack a bag for hospital.
The number I text is my platelet count. I am one of approximately 1,000 people in Ireland with ITP (immune thrombocytopenia), a rare blood disorder in which the immune system mistakenly attacks its own platelets. It can occur in pregnancy, follow a virus, vaccination or certain medications, but for most people the cause is unknown."
-By Karen Tomkins for The Irish Times. Image courtesy of Karen Tomkins and The Irish Times.
The blood drives are especially important because Daniel, as well as his father, relies on blood products every two weeks to survive.
“The importance of donating blood is critical in saving lives. Daniel would not have survived if it wasn’t for blood and plasma donors,” his mother, Dawn, told TAPinto Bordentown in November. “We started Daniel’s Fight because he coded waiting for blood. They did not have a match at the time. So now we hold blood drives every 3 to 4 months.”
- By Kristin Antonello. Photo courtesy "Daniel's Fight"
"For decades, McMaster University in Hamilton, Ont., has been a hub for research on blood and its diseases — known as hematology — but in recent weeks it has taken on an even more prominent role in the field: working to identify the rare blood-clotting syndrome linked to certain COVID-19 vaccines.
The lab, a small space on the third floor of the university hospital, is the only one in Canada with the equipment and expertise to test for the syndrome, known as vaccine-induced prothrombotic immune thrombocytopenia, or VIPIT."
- From cbc.ca. Image copyright and courtesy of McMaster University.
Sobi™, a North American affiliate of the international biopharmaceutical company Swedish Orphan Biovitrum AB (publ) (Sobi®), dedicated to rare diseases, in partnership with the Platelet Disorder Support Association (www.pdsa.org PDSA), a leading immune thrombocytopenia (ITP) advocacy organization in North America, today announced the launch of florio®ITP, an app for patients living with ITP. florio ITP is a digital diary mobile app developed by Florio GmbH, a Sobi company, in collaboration with ITP patients and healthcare professionals. The florio ITP app is intended to help support patients with ITP with a simple tool that allows them to organize, track and record their health and treatment information. It is available for free download for iOS (Apple) and Android mobile phone users.
- From Sobi Press Release
The Platelet Disorder Support Association is proud to announce that its strong financial health and ongoing accountability and transparency have earned a 100/100 rating from Charity Navigator’s new Encompass Rating System. This score designates PDSA as an official “Give with Confidence” charity, indicating that our organization is using its donations effectively based on Charity Navigator’s criteria. Charity Navigator is America’s largest and mostutilized independent charity evaluator.
"Gregory Michael, MD, a 56-year-old ob/gyn in private practice in Florida, died from complications of immune thrombocytopenia (ITP), according to a Facebook post written by his wife, Heidi Neckelmann.
Public health officials at the state and federal level are continuing to investigate Michael's case in relation to the COVID-19 injection, but vaccine experts and hematologists have emphasized that this case is extremely rare, and the coincidental timing of ITP and vaccination doesn't demonstrate causality."
- Amanda D'Ambrosio, Staff Writer, MedPage Today. Photo courtesy of the family. From Facebook.
"ITP's link to COVID gained a media spotlight earlier this month after the Miami obstetrician, Gregory Michael, MD, developed ITP days after getting the Pfizer COVID vaccine. In early January, after 2 weeks in the ICU, Michael died of a hemorrhagic stroke caused by the low platelet count.
Pfizer said in a statement that they are "actively investigating" the case, "but we don't believe at this time that there is any direct connection to the vaccine." Other experts have said the timing, particularly in a relatively young and healthy man, means a link to the vaccine is possible or even likely, but final results won't be known until the US Centers for Disease Control and Prevention finishes its investigation."
- By Donavyn Coffey, Medscape.com
"Anti-vaccine groups are exploiting the suffering and death of people who happen to fall ill after receiving a COVID-19 shot, threatening to undermine the largest vaccination campaign in U.S. history.
In some cases, anti-vaccine activists are fabricating stories of deaths that never occurred."
- By Liz Szabo | Kaiser Health News/Tribune Content Agency
"Kayleigh was diagnosed with hypothyroidism and then hyperthyroidism, which are prevalent for Down syndrome individuals. She also had sleep apnea and immune thrombocytopenia — or ITP — a condition in which the blood doesn’t clot normally.
When Kayleigh reached about 215 pounds, doctors gave Williamson the grim warning that things would not end well for her daughter.
“It dawned on me I didn't want my daughter laying in a hospital bed and me apologizing for her for something I could have prevented with a lifestyle change,” said Williamson.
The mother and daughter made health their main mission. The two joined Weight Watchers, and Williamson, who had just finished her first half marathon, encouraged Kayleigh to start running with her."
- By Travis Recek, Spectrum News 1. Photo courtesy Spectrum News 1.
Rigel Pharmaceuticals, Inc. and Medison Pharma today announced that Health Canada has approved the new drug submission (NDS) for TAVALISSE® (fostamatinib disodium hexahydrate) for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to other treatments.
"This approval of TAVALISSE provides ITP patients and physicians in Canada with a new oral treatment option, the only therapy to address the underlying platelet destruction that causes ITP," said Raul Rodriguez, Rigel's president and CEO. "With Medison as our collaborative partner, we believe TAVALISSE is well positioned for commercial success in the Canadian market."
In October 2019, Rigel entered into exclusive license agreements with Medison to commercialize TAVALISSE in Canada and Israel. With the approval from Health Canada, Medison intends to launch TAVALISSE in Canada in Q1 2021. In Israel, a decision on the new drug application (NDA) is anticipated during Q2 2021.
- From Rigel Press Release
COVID-19 continues to present a prolonged, global public health emergency, but PPTA remains steadfastly committed to its mission to promote the availability of — and access to — safe and effective plasma protein therapies for all patients. PPTA member companies operate more than 1,000 Source plasma donation centers in the U.S. and four countries in the European Union and manufacture plasma-derived therapies that treat patients with serious, rare, and often genetic diseases and disorders, including: primary immune deficiencies, bleeding disorders, Alpha-1 Antitrypsin deficiency, hereditary angioedema, and certain neurological conditions. These conditions are often only treated with therapies produced from plasma.
There is an urgent need for plasma donations.
- From PPTA Statement
"Laura Hamilton was diagnosed with a bleeding disorder after finding huge bruises.
The 38-year-old TV presenter – who has kids Rocco, five, and Tahlia, four, with husband Alex – was suddenly covered in huge bruises after welcoming her second child, which led to a terrifying diagnosis.
She told the Daily Mirror newspaper: "It was terrifying. I had been on quite a strict diet. I'd cut out sugar and carbs and I was the slimmest I'd been. I was exercising loads and felt in really good shape.
"When Tahlia was about seven months old, I was due to drive to Portugal to film a fitness app, but a few days before I was due to go I started noticing all this bruising coming out on my legs."
Eventually, her mother-in-law suggested she should see a doctor, because she suspected the bruising could be caused by a blood disorder."
- From thelist.co.uk. Photo courtesy laurahamiltonofficial.com.
PPTA has seen plasma donations fall this year. This decline is caused by the ongoing COVID-19 pandemic and the uncertainty felt by plasma donors. Declines in donations have the potential to restrict patients’ access to plasma derived therapies, or PDTs. Amy Efantis, PPTA, President & CEO stated that “Given the length of time to manufacture PDTs, without the needed raw material, it may become challenging at some point to supply enough PDTs to meet rare disease patients clinical needs.” Standard plasma collection remains critically important to support the needs of individuals with rare and chronic diseases. It is encouraging to see an increased awareness about the role convalescent plasma can play as a potential treatment for COVID-19 and we hope this message has a similar effect for standard plasma collection which is needed for patients with rare and chronic diseases every day.
- From PPTA press release
"A cheap and widely available drug can help save the lives of patients seriously ill with coronavirus.
The low-dose steroid treatment dexamethasone is a major breakthrough in the fight against the deadly virus, UK experts say.
The drug is part of the world's biggest trial testing existing treatments to see if they also work for coronavirus.
It cut the risk of death by a third for patients on ventilators. For those on oxygen, it cut deaths by a fifth."
- By Michelle Roberts - Health editor, BBC News online
"According to the Platelet Disorder Support Association, there is no added risk for patients with low platelets to be infected by the virus.
There is also no evidence that platelet production would decrease.
On the group’s website, medical experts say, 'There is no literature to support the virus would suppress platelet production any more than influenza, but some patients seem to be more sensitive to systemic viral infections than others in general.'”
- By Karen Zatkulak for News13 WLOS
Combination treatment with thrombopoietin receptor agonists (TPO-RA), immunosuppressants, and intravenous immunoglobulin G (IVIG) appears to be efficacious in the management of patients with severe refractory immune thrombocytopenia (ITP), according to a study published in the British Journal of Haematology.
Researchers evaluated the combination of ciclosporin A and mycophenolate mofetil (CSA/MMF; to inhibit T-cell activation), romiplostim or eltrombopag (to stimulate platelet production), and IVIG (to inhibit autoantibody-mediated platelet destruction) as a way to target 3 different mechanisms of ITP pathology.
- Article by Lauren Dembeck, PhD for HematologyAdvisor.com. Photo courtesy of HematologyAdvisor.com.
COVID-19 didn’t stop a birthday celebration for one special teen in Kalispell on Tuesday.
Cars and fire trucks lined up at Woodland Park and headed over to Nathan Brewer’s house for a birthday parade.
“Today, Kalispell came together to show my 16-year-old son their love and support,” Nathan Brewer’s mother, Nicole Cooper Christensen, said.
Brewer has a rare blood disorder and his treatment recently failed.
- By Vanessa Perez, NBC Montana. Photo by NBC Montana.
Osaka, JAPAN, and King of Prussia, PA, USA
Partnership brings together world-leading plasma companies to focus on developing and delivering a hyperimmune immunoglobulin in the global fight against COVID-19
April 6, 2020
Biotest, BPL, LFB, and Octapharma have joined an alliance formed by CSL Behring and Takeda Pharmaceutical Company Limited to develop a potential plasma-derived therapy for treating COVID-19. The alliance will begin immediately with the investigational development of one, unbranded anti-SARS-CoV-2 polyclonal hyperimmune immunoglobulin medicine with the potential to treat individuals with serious complications from COVID-19.
“Unprecedented times call for bold moves,” said Julie Kim, President of Plasma-Derived Therapies Business Unit, Takeda. “We collectively agree that by collaborating and bringing industry resources together, we could accelerate bringing a potential therapy to market as well as increase the potential supply. We invite companies and institutions focusing on plasma to support or join our alliance.”
“Leaders lead during uncertainty. There is no question that we are all experiencing the impact of COVID-19,” said Bill Mezzanotte, CSL Behring’s Executive Vice President and Head of Research and Development. “This effort aims to accelerate a reliable, scalable and sustainable option for caregivers to treat patients suffering from the impact of COVID-19. In addition to pooling industry resources, we will also collaborate with government and academic efforts as a single alliance whenever we can, including important activities like clinical trials. This will make it more efficient in these hectic times for these stakeholders as well.”
The collaboration will leverage leading-edge expertise and work that the companies already have underway. Experts from the alliance will begin collaborating across key aspects such as plasma collections, clinical trial development and manufacturing. Further companies and institutions may join the alliance as well.
Developing a hyperimmune will require plasma donation from many individuals who have fully recovered from COVID-19, and whose blood contains antibodies that can fight the novel coronavirus. Once collected, the “convalescent” plasma would then be transported to manufacturing facilities where it undergoes proprietary processing, including effective virus inactivation and removal processes, and then is purified into the product.
Individuals interested in donating plasma can visit this link to find the nearest licensed plasma collection center to their location.
- From press release
Abbey died on Jan. 12 after a sudden and brief battle with idiopathic thrombocytopenic purpura, an autoimmune blood disorder in which the immune system attacks the blood platelets. Because platelets help stop bleeding, it can result in easy bruising, bleeding gums and internal bleeding.
After her death, Abbey’s kidneys went to two people. Her pancreas went to one of the kidney recipients. Her liver has been donated for research.
- By Maggie Gilroy, Binghamton Press & Sun-Bulletin. USA Today Network. Photo courtesy of the family.
Researchers have homed in on the cells that cause an autoimmune disease to arise in the body.
The lack of understanding of the root cause of autoimmune disease means that treatment for autoimmune conditions is mostly symptomatic. Most clinicians aim at reducing the level of immune cell activation and controlling inflammation in other organs such as the joints, kidneys, and skin. None of the current treatments addresses the course of the disease directly.
The problem was with the extreme rarity of the rogue cells in a blood sample. Often, they make up less than 0.25 percent or 1 in 400. The most sophisticated analyses of these cells to date have yielded only an estimated average of the enormous variety of different immune cells in the sample. The current study thus switched to sequencing the genome of each cell in the sample.
- By Dr. Liji Thomas, MD. Image Credit: Dr Ofir Shein-Lumbroso.
A significant number of pediatric patients with immune thrombocytopenia (ITP) have difficulties with fatigue, emotional and behavioral symptoms, and executive functioning, with each having an effect on quality of life (QoL), according to a paper published in the British Journal of Haematology.
Fatigue, minor cognitive impairment, and overall reduction in health-related QoL are concerns in adults with ITP, but no studies have previously measured emotional and behavioral issues among pediatric patients. Therefore, pediatric patients are generally treated with a “watch and wait” approach.
- By Jonathan Goodman, MPhil for Hematology Advisor. Photo courtesty of pixabay.
Doctors diagnosed Bridget Fox, 6, with Immune thrombocytopenia purpura, or ITP, about a year ago during a kindergarten physical. The disorder causes excessive bleeding and bruising.
"She's had eight different blood transfusions. One of them was a platelet transfusion. Then she received medication that has blood products in it," said Tabitha Fox, Bridget's mom.
- By Kelsey Dickeson for KSNB News. Photo courtesy KSNB and Tabitha Fox.
Dova Pharmaceuticals, Inc., a wholly owned subsidiary of Swedish Orphan Biovitrum AB (Sobi™) has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) for avatrombopag for the potential treament of Chemotherapy-Induced Thrombocytopenia (CIT). Enrollment remains ongoing for the phase 3 clinical study for the treatment of patients with CIT.
CIT, a potentially serious complication of chemotherapy, results in low platelet levels and can lead to chemotherapy dose reductions, chemotherapy dose delays, or changes to chemotherapy regimens. For cancer patients receiving chemotherapy with curative intent, alterations in their chemotherapy regimen due to low platelets may compromise their long-term outcomes. Approximately 10 percent of US cancer patients per year experience CIT2 which may require chemotherapy regimen modifications. Currently there are no approved treatments available for CIT in the US or EU.
- From SOBI Press Release

IMPORTANT!
The Platelet Disorder Support Association does not provide medical advice or endorse any medication, vitamins or herbs. The information contained herein is not intended nor implied to be a substitute for professional medical advice and is provided for educational purposes only. Always seek the advice of your physician or other qualified healthcare provider before starting any new treatment, discontinuing an existing treatment and to discuss any questions you may have regarding your unique medical condition.