OR05 - Long-term results of patients with acquired haemophilia A in the era of emicizumab
OR05
Long-term results of patients with acquired haemophilia A in the era of emicizumab
M. Erard1,*, T. Vanassche1, P. Verhamme1, C. Lambert2, C. Hermans2, Q. Van Thillo3
1Department of Cardiovascular diseases, University hospitals Leuven, Leuven, 2Haemostasis and Thrombosis unit, division of Adult Haematology, Cliniques universitaires Saint-Luc, Brussels, 3Department of Cardiovascular diseases, Haemophilia centre, University hospitals Leuven, Leuven, Belgium
Introduction: Acquired haemophilia A (AHA) is a rare bleeding disorder caused by inhibitory autoantibodies against endogenous factor VIII (FVIII) for which emicizumab was shown to be effective in preventing bleeding events (Tiede et al. 2024). In the GTH-AHA-EMI study (Schimansky et al. 2025), immunosuppressive therapy (IST) was postponed following initial therapy with emicizumab, which led to fewer infectious complications and improved survival. However, the long-term prospects under emicizumab treatment remain unclear.
Methods: This retrospective Belgian multicentre study (University hospitals Leuven and Cliniques universitaires Saint-Luc) included 34 patients diagnosed with AHA from the availability of emicizumab in January 2020 to June 2025. We collected patient characteristics, laboratory coagulation parameters, the use of haemostatic agents, type of IST, bleeding and thrombotic events.
Results: Patients presented with spontaneous hematoma (n=28), intramuscular bleeding (n=13), haematuria (n=5), and gastrointestinal bleeding (n=9). All but one were treated with emicizumab, this patient was excluded from further analysis. Sixteen patients (47.1%) experienced major bleeding for which recombinant factor VII was required in 9 patients (26.5%). To eradicate the underlying inhibitor, 30 patients (88.2%) received IST including monotherapy with steroids (n=2) or rituximab (n=8), or a combination therapy (n=20). In 23 patients (76.7%), inhibitor eradication was successful allowing emicizumab to be discontinued after a median of 151 days (IQR 81-201) of therapy. In 6 patients (17.6%), emicizumab was continued until the patient’s death. One thrombotic event occurred, and there were no major bleeds after initiation of treatment. Seven patients died due to infection (n=2), underlying disease (n=3) or other unrelated events (n=2). There was no bleed-related mortality.
Discussion/Conclusion: This study reaffirms the efficacy and safety of emicizumab for the prevention of bleeding complications during early AHA management. Interestingly, most patients were able to discontinue emicizumab after a relatively short period of time following successful inhibitor eradication. By combining strong haemostatic efficacy with the possibility to postpone IST, emicizumab may contribute to a safer, more efficient, and shorter overall treatment course in AHA.
Disclosure of Interest: M. Erard: None declared, T. Vanassche Grant/Research support from: Thomas Vanassche is a clinical investigator in industry‐sponsored studies and has received honoraria for consultancy and lectures from Bayer, Daiichi Sankyo, Sanofi, and Amgen., P. Verhamme Grant/Research support from: Peter Verhamme is a clinical investigator in industry‐sponsored studies and has received honoraria for consultancy and lectures from CSL Behring, Roche, CAF‐DCF, and Bayer., C. Lambert Grant/Research support from: Catherine Lambert reports speakers and members of advisory board for Amgen, CSL Behring, LFB, Octapharma, Sanofi, Sobi, Roche., C. Hermans Grant/Research support from: Cedric Hermans has received research support from Bayer, Shire, Pfizer, and Novo Nordisk; CH has obtained consultant fees from Pfizer, Bayer, Shire, Novo Nordisk, CSL Behring, Octapharma, Sobi Biogen, LFB, CAF-DCF, and Roche; CH has been invited to give guest lectures at different conferences by Pfizer, Bayer, Shire, Novo Nordisk, CSL Behring, Octapharma, Sobi Biogen, LFB, CAF-DCF, Roche; and Kedrion. , Q. Van Thillo Grant/Research support from: Quentin Van Thillo has received consultant fees from Pfizer and Roche, and travel support from CSL Behring.