摘要：Hemophilia A is a rare X-linked disease that occurs as a result of a defect in the FVIII-encoding gene. The reduction or absence of plasma FVIII compromises the coagulation cascade, resulting in frequent bleeds, especially in joints or soft tissues. Currently, replacement therapy with coagulation factor concentrates is the gold standard for the treatment of FVIII deficiency. Herein, we report a case series of five hemophilia A patients treated with an extended half-life recombinant human coagulation factor, FVIII-Fc fusion protein (efmoroctocog alfa). The prophylactic regimen for each patient was individualized based on their pharmacokinetic profile. Compared to previous prophylactic treatments, most patients received a reduced weekly dose of concentrate, all underwent a reduced frequency of administration, the annualized bleeding rates (ABR) and hemophilia joint health scores (HJHS) were stable or improved. The half-life of efmoroctocog alfa and the 72-hour trough levels were higher than those observed in the A-LONG Phase III trial. In conclusion, all patients reported clinical improvements and general subjective wellbeing in the absence of significant safety concerns after switching to efmoroctocog alfa.
