Long term outcome of patients with acquired haemophilia – A monocentre interim analysis of 82 patients

BackgroundAcquired haemophilia (AH) is a rare condition leading to life threatening bleedings with a mortality ranging between 7.9 and 22%. Due to the low incidence of AH, randomized studies are not available, but observational studies with a long term follow up are of high interest.MethodsOur haemophilia centre has documented since 1994 the treatment of 82 patients with AH, suffering from severe and moderate AH. Patient's clinical data, treatment schedules and long term outcomes were analyzed.ResultsIn 73% of patients the first manifestation of AH was a severe life threatening bleeding. These patients were successfully treated via a multimodal immunomodulating regime (Bonn Protocol) with an overall response rate of 93% after a median treatment time of 16 d (95% CI: 13–18.9 d). Solid cancer, lymphoma, surgery and an adjacent autoimmune disease were the main “associated conditions” of AH (AHSAC). In patients with less severe AH, conventional immunosuppressive treatment was successful in 11 patients after a median of 3.9 months (range 1–12), 5 patients failed and were treated successfully second line via the Bonn protocol.In both treatment groups no bleeding associated fatalities occurred.Four patients required an additional treatment of acute bleedings with bypassing agents leading to fatal thrombotic events.ConclusionOur data show that an optimal treatment schedule in AH should be adapted to the patient's individual risk profile considering the severity of bleeding and comorbidities. Idiopathic AH predisposes to severe AH requiring a more intensive treatment compared to AHSAC. In the latter, the so called “bystander immunological phenomena” induced by the primary disorder might have an important impact on the inhibitor development. Therefore the differentiation between idiopathic AH and AHSAC should be considered for a treatment decision.