Top 10 Bleeding Disorders News stories of 2025
Readers were especially interested in stories about ITP, Doptelet
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Bleeding Disorders News brought you daily coverage of the latest clinical research and scientific breakthroughs related to bleeding disorders in 2025.
Here are the year’s top 10 most-read stories, each with a brief description.
No. 10 — FDA approves Doptelet, Doptelet Sprinkle for pediatric ITP
In July, the U.S. Food and Drug Administration (FDA) approved Doptelet (avatrombopag), available as a tablet and an oral granule called Doptelet Sprinkle, for children ages 1 and older with hard-to-treat chronic immune thrombocytopenia (ITP). Doptelet is designed to counteract ITP-causing immune attacks on platelets, the cell fragments that help blood clot, by stimulating the bone marrow to produce more platelets. Its approval was based on data from a Phase 3b clinical study, which showed that Doptelet boosted platelet counts at least twice in nearly all children with chronic ITP who hadn’t responded to prior treatments.
No. 9 — Enrollment ongoing in efgartigimod trial for adult ITP
The Phase 3 ADVANCE-NEXT trial is evaluating efgartigimod in approximately 63 adults with primary ITP lasting more than one year who have not responded to at least one ITP treatment. Its enrollment is ongoing at sites in the U.S., Europe, and China. Efgartigimod, designed to reduce self-reactive antibodies that lower platelet counts, is approved for adults with ITP in Japan, where it’s sold as Vyvgart. Argenx, the therapy’s developer, already markets Vyvgart Hytrulo, a subcutaneous (under-the-skin) formulation of efgartigimod, in the U.S. and elsewhere for other autoimmune conditions. Top-line ADVANCE-NEXT results are expected in the second half of this year.
No. 8 — High blood clot risk found in ITP patients
People with ITP have an increased risk of developing blood clots that block circulation, according to an analysis of multiple studies. Researchers also found greater rates of clotting events, or thrombosis, in individuals living in the U.S. and Europe compared to those living in Asia. Older age, other underlying medical conditions, and the use of thrombopoietin receptor agonists, treatments designed to increase platelet production, also increased the risk of blood clots in ITP. These findings were based on a pooled analysis of 20 published studies that included more than 100,000 participants.
No. 7 — Age impacts quality of life of children with VWD: Survey
Children with von Willebrand disease (VWD) have a lower quality of life, but the areas most affected vary by age, according to a French survey. VWD occurs when the body lacks sufficient von Willebrand factor, a protein needed for blood clotting. The study included 117 children younger than 18. Young children, ages 4-7, were primarily limited in physical activity, while older children struggled more with independence. Children 8 and older generally reported good physical and social well-being, though physical scores were lower for those with type 3 VWD, the most severe form of the disease. Across all types, independence, treatment burden, friendships, and participation in sports were common challenges.
No. 6 — Scholarship open for students affected by bleeding disorders
Applications are open for the 2026 Eric Dostie Memorial College Scholarships, awarded to 10 full-time college students whose lives have been directly affected by von Willebrand disease or hemophilia. Run by Nufactor, the program is open to U.S. citizens living in the U.S. who are enrolled full-time at an accredited two- or four-year college. Interested participants should complete an online application and a 400-word essay explaining what motivates them, their chosen field, the challenges they’ve faced, and how they plan to give back to their community. The deadline is Feb. 17, 2026, and the winners of the $ 1,000 prize will be announced in May.
No. 5 — Doptelet safe, effective for pediatric ITP with prior treatment
Doptelet was found to be safe and effective in children with treatment-resistant ITP, according to published data from the Phase 3b clinical trial that supported the therapy’s recent U.S. approval. It enrolled 75 children, ages 1-17, with primary ITP, most of whom had previously received another thrombopoietin receptor agonist and at least three prior lines of treatment. After 12 weeks with either Doptelet or a placebo, 81% of Doptelet recipients had a platelet response, and 28% had a durable response. No children in the placebo group responded. An open-label extension phase is ongoing, with all children receiving Doptelet for up to two years.
No. 4 — New funding supports tAN trial for heavy menstrual bleeding
Spark Biomedical received funding from Wellcome Leap‘s women’s health program to run a clinical trial testing transcutaneous auricular neurostimulation (tAN) for heavy menstrual bleeding in women with VWD and in adolescents with unexplained heavy bleeding. A noninvasive, drug-free method, tAN may improve blood clotting by delivering electrical impulses to specific nerves. The trial will collect data to support future FDA approval. Early studies showed promising results, with a more than 50% drop in blood loss and shorter menstrual periods in users, according to the developer.
No. 3 — Preclinical study IDs potential new way to treat VWD type 1
Scientists have developed a potential treatment for type 1 VWD that may stabilize the faulty protein that causes VWD and increase its activity to improve blood clotting. In VWD type 1, the most common form of the disease, VWF protein levels are lower than normal, but the protein remains present in small amounts. Dubbed KB-V13A12, the novel treatment consists of two fused nanobodies, tiny antibody fragments that link VWF to albumin, a very stable blood protein. In a mouse model of VWD type 1, KB-V13A12 doubled VWF protein levels and significantly reduced bleeding after a single subcutaneous injection.
No. 2 — FDA approves oral rilzabrutinib, now Wayrilz, for ITP
In August, the FDA approved Sanofi‘s rilzabrutinib — now Wayrilz — to treat adults with persistent or chronic ITP who haven’t responded to previous treatments. Wayrilz blocks BTK, an enzyme that supports the function of B-cells and macrophages, immune cells that produce harmful antibodies and destroy platelets in ITP. Its approval is based on data from the Phase 3 LUNA 3 study, which showed that about twice as many Wayrilz-treated patients reached key platelet thresholds compared with those on a placebo. Approximately one-fourth of those on Wayrilz achieved a durable platelet response, whereas none of those on the placebo did, meeting the study’s primary goal. Sanofi will continue testing the therapy in a Phase 3 clinical study, LUNA 4, in ITP patients who don’t respond to first-line treatments.
No. 1 — ITP Awareness Month shines spotlight on rare disorder
In September, patients, caregivers, medical professionals, and other advocates gathered during Immune Thrombocytopenia Awareness Month to raise awareness and support for the rare bleeding disorder. The Platelet Disorder Support Association (PDSA), which created ITP Awareness Month in 2010, led this year’s activities. Patients and supporters joined “Pump It Up For Platelets!” walks and runs, either in person or online, to raise research funds. People were also encouraged to shop the Platelet Store for discounted awareness items and to wear purple for Sport Purple for Platelets Day on Sept. 26. Supporters can also donate, fundraise, or become PDSA members to help the cause.
At Bleeding Disorders News, we hope our stories and reporting throughout 2025 positively impacted the lives of those with bleeding disorders. We look forward to continuing to serve the community in 2026 and to providing ongoing support and information.
We wish all our readers a very happy New Year!
