Long-term outcomes in the treatment of acquired hemophilia A: a 16-year single institution prospective cohort experience.

Abstract

: Acquired hemophilia A (AHA) is a rare bleeding disorder caused by autoantibodies against coagulation factor VIII. We conducted a single institution prospective cohort study to assess treatment strategies and long-term outcomes in AHA patients and provide further evidence for effective treatment and relapse timing. A total of 25 patients diagnosed with AHA between 2001 and 2017 at Penn State Hershey Medical Center were prospectively followed. Information was collected on factor VIII activity and inhibitor titer at diagnosis, treatment regimen(s), complete remission, and relapse time. For immunosuppressive therapy (IST), 19 patients were treated initially with prednisone and cyclophosphamide, four were treated with prednisone, one with prednisone and rituximab, and one with prednisone and second-line rituximab. 13/17 (76%) evaluable patients treated with prednisone and cyclophosphamide achieved complete remission. Four patients received rituximab as second-line therapy (inhibitor titers 34, 122, 416, and 768 BU); three achieved complete remission and one died from sepsis. Both evaluable patients receiving initial prednisone alone achieved complete remission. Five relapses occurred from 17 days to 7 years; all were treated with prednisone and cyclophosphamide and achieved complete remission. IST with prednisone and cyclophosphamide is highly effective in achieving and maintaining complete remission, even for relapsed patients. Despite dual IST with prednisone and cyclophosphamide, some patients, particularly with extremely high inhibitor titers, required addition of second-line rituximab to achieve complete remission. This supports rituximab as effective salvage treatment, including for patients with inhibitor titers at least 100-200 BU. Those who experienced relapse often did so years after complete remission, signifying importance of continued monitoring and vigilance.