ISTH 2023 | Emicizumab prophylaxis instead of immunosuppressive therapy in patients with acquired hemophilia A

We presented the results of an investigator-initiated clinical trial that was performed in 14 hemophilia treatment centers in Austria and Germany. We enrolled patients with acquired hemophilia A who were not previously treated with immunosuppressive therapy and did not receive immunosuppressive therapy throughout the study. Instead, our patients received bleed prophylaxis with emicizumab and we found that emicizumab confers excellent protection against bleeding while leaving out immunosuppressive therapy resulted in fewer side effects that we usually see with this kind of treatment. Well, current clinical practice in acquired hemophilia is to treat the bleeding with hemostatic treatment. But then the underlying antibodies against coagulation factors with immunosuppression immediately. So it’s an autoimmune disease treated by immunosuppression. However, patients at the start of their disease are very sick. They are often elderly, fragile, so immediate immunosuppression is harmful to them. And in the past we saw lots of infections. So now with emicizumab, we have a bridge. We can protect patients from bleeding while they are on emicizumab, they can recover and then take the risks of immunosuppressive therapy later on when they have arrived at a better health status. I think it is a good thing that as a community we do investigator-initiated studies that are motivated by burning questions and unmet needs for our patients. So I’m very happy that we received money from the manufacturer of the drug but could design a study on our own here for the purpose of improving the care for our patients.