Potential role of prophylactic treatment for prevention of joint disease in hemophilia A

摘要

Evaluation of: Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N. Engl. J. Med. 357, 535–544 (2007). There has been significant debate about the potential role of prophylactic treatment for prevention of joint disease in hemophilia. In this study, Manco-Johnson and colleagues report the results of a randomized study that assessed the benefit of recombinant factor VIII for the prophylactic treatment of boys with severe hemophilia A. Prophylaxis, that is, intravenous injection of clotting factor concentrate in anticipation of bleeding, was compared with on-demand, or episodic, treatment, defined as the administration of factor VIII whenever signs of joint bleeding appeared. After a mean follow-up of 49 months, 93% of subjects in the prophylaxis group showed no joint damage on magnetic resonance imaging as compared with 55% in the episodic-therapy group. The number of factor VIII units administered per patient was approximately 350,000 in the prophylaxis group (0.63 joint bleeds per year) and 113,000 in the episodic-therapy group (4.89 joint bleeds per year). This study concludes that prophylaxis with recombinant factor VIII can prevent joint damage and decrease joint bleeds in boys with hemophilia.