Cyclophosphamide vs rituximab for eradicating inhibitors in acquired hemophilia A: A randomized trial in 108 patients
H. Lévesque, Jean‐François Viallard, Estelle Houivet, Bernard Bonnotte, Sophie Voisin, Véronique Le Cam‐Duchez, F. Maillot, M. Lambert, É. Liozon, B. Hervier, Olivier Fain, Benoît Guillet, J. Schmidt, L.E. Luca, Mikaël Ebbo, Nicole Ferreira-Maldent, Antoine Babuty, L. Sailler, P. Duffau, Virginie Barbay, Sylvain Audia, Jacques Bénichou, J. Graveleau, Y. Benhamou
Abstract
BACKGROUND: Acquired hemophilia A (AHA) is a rare autoimmune disorder due to autoantibodies against Factor VIII, with a high mortality risk. Treatments aim to control bleeding and eradicate antibodies by immunosuppression. International recommendations rely on registers and international expert panels. METHODS: rituximab once weekly for 4 weeks. The primary endpoint was complete remission over 18 months. Secondary endpoints included time to achieve complete remission, relapse occurrence, mortality, infections and bleeding, and severe adverse events. RESULTS: ), significantly more remissions were observed with cyclophosphamide (22 patients, 78.6 %) than with rituximab (12 patients, 48.0 %; p = 0.02). Relapse rates, deaths, severe infections, and bleeding were similar in the 2 groups. In patients with severe infection, cumulative doses of steroids were significantly higher than in patients without infection (p = 0.03). CONCLUSION: Cyclophosphamide and rituximab showed similar efficacy and safety. As first line, cyclophosphamide seems preferable, especially in poor prognosis patients, as administered orally and less expensive. FUNDING: French Ministry of Health. CLINICALTRIALS: gov number: NCT01808911.