Treatment of acquired hemophilia A, a balancing act: results from a 27-year Dutch cohort study

Sarah J. Schep*, Wobke E. M. van Dijk, Erik A. M. Beckers, Karina Meijer, Michiel Coppens, Jeroen Eikenboom, Frank W. G. Leebeek, Lize F. D. van Vulpen, Kathelijn F. Fischer, Roger E. G. Schutgens, Dutch Society of Haemophilia Treaters

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review


Acquired hemophilia A (AHA) is a severe auto-immune bleeding disorder. Treatment of AHA is burdensome and optimal management is still unresolved. Therefore a retrospective nationwide multi-center cohort study (1992-2018) was performed to evaluate clinical presentation and treatment efficacy and safety of AHA in the Netherlands. Multivariate logistic and Cox regression analysis was used to study independent associations between patient characteristics and clinical outcomes. A total of 143 patients (median age 73 years; 52.4% male) were included with a median follow-up of 16.8 months (IQR 3.6-41.5 months). First-line immunosuppressive treatment was mostly steroid monotherapy (67.6%), steroids/cyclophosphamide (11.9%) and steroids/rituximab (11.9%), with success rates of 35.2%, 80.0% and 66.7% respectively,P<.05. Eventually 75% of patients achieved complete remission (CR). A high anti-FVIII antibody titer, severe bleeding and steroid monotherapy were associated with lower CR rates. Infections, the most important adverse event, occurred significantly more often with steroid combination therapy compared to steroids alone (38.7% vs 10.6%;P= .001). Overall mortality was 38.2%, mostly due to infections (19.2%) compared to 7.7% fatal bleeds. Advanced age, underlying malignancy and ICU admission were predictors for mortality. This study showed that AHA is characterized by significant disease-related and treatment-related morbidity and mortality. A high anti-FVIII titer, severe bleeding and steroid monotherapy were associated with a lower CR rate. The efficacy of steroid combination therapies however, was overshadowed by higher infection rates and infections represented the most important cause of death. The challenging and delicate balance between treatment effectivity and safety requires ongoing monitoring of AHA and further identification of prognostic markers.

Original languageEnglish
Pages (from-to)51-59
Number of pages9
JournalAmerican Journal of Hematology
Issue number1
Early online date10 Oct 2020
Publication statusPublished - Jan 2021



Cite this