CAR-T Meeting 2026 | The potential role of CAR T-cell therapy in acquired hemophilia A

So, hemophilia is a complex coagulopathy, and it’s a disease that is mostly a genetic disease. So, it’s passed on in an X-linked recessive manner, and that’s a disease where currently I don’t see any role of CAR T-cell therapy. In contrast, there’s also acquired hemophilia A, which is an autoimmune disease. It’s a very rare disease, usually just affecting about 600 patients annually in Germany. And worldwide, we see about one to two cases per million citizens per year. So it’s a rare autoimmune disease affecting the elderly. And the problem about acquired hemophilia is that it causes severe bleedings, and these bleeding episodes are extremely difficult to control. And one way to control this obviously is to give clotting factors like recombinant factor seven. But in order to break the disease, you have to do something to stop the immune system from overreacting. And in the past, or I would also say currently, the standard is to combine rituximab in combination with steroids and cyclophosphamide. And in the majority of patients, you see a response. Now, unfortunately, there are patients with no response to standard immunosuppressive therapy, and that’s where there could be a role for CAR T-cell therapy. 

And in our center, we had a patient when all immunosuppressive therapies that are currently employed in acquired hemophilia A did not work, and the patient continued to bleed. And we tried CAR T-cell treatment in this patient when we were standing with our backs to the wall. And fortunately, the patient responded. He had a complete remission of the disease, meaning that factor VIII levels increased and the inhibitor was not detectable anymore. The patient was asymptomatic. And that’s something that was a huge, huge success. Unfortunately, the patient relapsed after several months of doing extremely well. But he responded then to one course of teclistamab. So in conclusion, I would say the indication could be for patients who don’t respond to standard immunosuppressive therapies with acquired hemophilia A. 

So currently, I would say the treatment is still experimental, meaning that it’s not standard of care. However, I do see a role for the therapy if standard immunosuppressive therapy fails. And with that, I mean a patient who is not responding, for instance, to rituximab, cyclophosphamide, and steroids, or one other kind of course of immunosuppressive therapy. And I think in that setting, it is possible to think about CAR T-cell therapy sooner rather than later, because we need to keep in mind it’s a life-threatening disease and treatment with coagulation factors is also very expensive. So in the future, I would wish that we had the opportunity to think about CAR T-cell treatment in such a patient once standard immunosuppressive therapy fails.

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