Management of previously untreated patients with severe haemophilia A preferentially treated with recombinant factor VIII products: Two French centres' real-life experience

Authors: Drillaud, N., Babuty, A., Rugeri, L., Fouassier, M., Lienhart, A., Béné, MC., Lefranc, C., Ternisien, C., Chamouard, V., Sigaud, M., Pennetier, M., Trossaërt, M., and Meunier, S.

Publication: Haemophilia; September 2020

Affiliations: Haemophilia Treatment Center University Hospital, Nantes, France; Haemophilia Treatment Center University Hospital, Lyon, France.

Abstract: Severe haemophilia A, defined by Factor VIII activity levels < 1 IU/dL, requires prophylactic supplementation. In our centres, prophylaxis is initiated when children start walking. The two main sources of FVIII are plasma derived (pdFVIII) and recombinant (rFVIII). Replacement therapy can be complicated by the appearance of anti‐FVIII antibodies (inhibitors), occurring in 19%‐45%1 of previously untreated patients (PUPs).

Low‐titre inhibitors (<5 Bethesda Units [BU]/mL) may be transient and allow to continue treatment with a higher dose of FVIII, while high‐titre inhibitors (≥5 BU/mL) impose the use of bypassing agents or emicizumab. Their eradication may require a program of immune tolerance induction (ITI).