Late-breaking phase 1 data presented at EHA 2026 suggest BCMA-directed CAR-T therapy may induce rapid platelet recovery, durable drug-free remissions, and broad immune reprogramming in refractory immune thrombocytopenia
“CAR-T is not only for malignant disease, it may offer a new therapeutic roadmap for chronic autoimmune hematologic disorders like ITP”
touchHEMATOLOGY coverage from EHA 2026
Abstract LB5003: Shu J, Xu M, Xie W, et al. BCMA CAR-T CELL THERAPY INDUCES COMPREHENSIVE IMMUNE RESETTING AND DURABLE REMISSION IN REFRACTORY PRIMARY IMMUNE THROMBOCYTOPENIA. Presented at: EHA 2026, Stockholm, Sweden. June 11–14th, 2026.
New late-breaking data presented at the European Hematology Association (EHA) 2026 Congress provide the first clinical evidence supporting BCMA-targeted CAR-T therapy in refractory primary immune thrombocytopenia (ITP). In the phase 1 study (NCT06519565), investigators evaluated BCMA CAR-T in heavily pretreated, autoantibody-positive patients with severe thrombocytopenia (<30×10^9/L). Among four evaluable patients, three achieved complete response (CR) within 14 days and maintained drug-free remission for 6–14 months, while a fourth achieved delayed CR at day 28 before relapsing at month 9. CAR-T expansion peaked at day 14 and persisted for 3 months. Treatment was well tolerated, with only grade 1–2 cytokine release syndrome (CRS), and no ICANS, grade ≥3 ICAHT, or infections. Beyond efficacy, translational analyses suggested BCMA CAR-T induced a broader “immune reset,” characterized by B-cell reprogramming, suppression of pathogenic anti-platelet antibodies, and remodeling of inflammatory and cytotoxic T-cell compartments.
Speaking exclusively with touchHEMATOLOGY, principle investigator Prof Heng Mei (Wuhan Union Hospital, China) discusses the rationale, findings, and future implications of this strategy.
What was the rationale for exploring BCMA CAR-T in refractory ITP?
ITP is one of the most common hematologic autoimmune diseases, and around one-third of patients become refractory or relapse. These patients often have chronic disease and require multiple therapies over time, including corticosteroids, IVIG, rituximab, and TPO-receptor agonists, which can significantly affect quality of life. Our group has worked with CAR-T for more than 10 years, initially in hematologic malignancies such as leukemia, lymphoma, and myeloma, and later in autoimmune diseases including lupus. We believed there was a rationale to extend this to ITP. Previously, there had only been two reported cases of CD19 CAR-T in ITP, one in secondary ITP and one in primary ITP, but the remission duration was short, only around three to four months. BCMA is a better target in this setting because it directly targets late-stage B cells and plasma cells, which are the main producers of pathogenic autoantibodies.
You describe this as an “immune reset.” What does that mean in practice?
For autoimmune disease, the goal is not only to reduce antibodies, but to fundamentally reset the immune environment. That is very important. In our study, single-cell analyses showed that BCMA CAR-T did not only reduce B cells and plasma cells, but also reshaped the wider immune system, including T cells and even the myeloid compartment. This is very different from simply depleting cells. That may explain why long-term remission can persist even after CAR-T cells are no longer detectable — because the immune system has been rebuilt into a more stable state.
Three patients achieved complete response within 14 days. Were you surprised by the speed of platelet recovery?
Yes, the speed was faster than we expected. With conventional oral therapies in ITP, responses often take around 28 days, and many patients achieve only partial responses. Here, three out of four patients achieved strong complete responses within just 14 days. What is especially encouraging is not only the speed, but the durability. These remissions have remained stable and drug-free, which is highly significant.
Do you have any insight into why one patient relapsed?
I think T-cell quality is very important. In the relapsed patient, the T-cell population appeared more exhausted, and we observed a high frequency of terminal effector T cells. That may have limited the quality or persistence of the CAR-T response. By contrast, the other three patients had better preserved naïve T-cell populations, which may have contributed to better outcomes.
What are the next steps for this programme?
This is only the beginning. This is a small cohort, and we need to enroll more patients. We are continuing dose exploration, particularly comparing 100×10^6 and 200×10^6 cells, because toxicity may be somewhat higher at the higher dose. We also plan multicenter studies to validate these findings and potentially randomized studies against standard therapies. In the future, we are also interested in dual-target CAR-T strategies and real-world evaluation.
What is the main message for the hematology community?
CAR-T is not only for malignant diseases. It may also have an important role in autoimmune diseases, especially chronic hematologic immune disorders like ITP. We should pay more attention to these diseases and explore new strategies such as immunotherapy and cellular therapy. I believe this can provide a new roadmap for patients with refractory disease.
This content has been developed independently by Touch Medical Media for touchHEMATOLOGY. Views expressed are the speaker’s own and do not necessarily reflect the views of Touch Medical Media. This article was created by the editorial team utilizing AI as an editorial tool (ChatGPT 5.5 [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.
Cite: BCMA CAR-T shows early promise for immune resetting in refractory primary ITP. touchHEMATOLOGY. 1st July 2026.
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