Prophylactic Treatment with Factor VIII in Hemophilia
Patricia A. Stoeck
Howard J. Weinstein
Prophylaxis Versus Episodic Treatment to Prevent Joint Disease in Boys With Severe Hemophilia
Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. N Engl J Med. 2007;357(6):535–544
Background
Hemophilia A, which can cause life-threatening bleeding, affects over 300,000 people worldwide. Hemarthrosis is the most common bleeding complication, comprising 70% to 80% of bleeding episodes.1 With the development of recombinant products mitigating infection risk, some providers began using factor VIII (FVIII) prophylactically to prevent hemophilic arthropathy, rather than only during bleeding complications, as prevention has tremendous potential to improve quality of life and life expectancy. The timing, dosing, and duration of preventive therapy, however, were based on empiric guidelines, rather than objective studies, and cost remained a barrier.
Objectives
To determine if prophylactic FVIII infusions were superior to episodic FVIII for joint hemorrhage in preventing joint damage in boys with severe hemophilia A.
Methods
Single-blind, randomized, multicenter trial from 1996 to 2005.
Patients
65 males ages <30 months with severe hemophilia A (FVIII level ≤2 U/dL). Select exclusion criteria: >2 joint hemorrhages into each index joint (ankle, knee, and elbow), abnormal joints on examination or imaging, presence of FVIII inhibitor, abnormal platelet counts.