Prophylactic Therapy Reduces Joint Damage in Hemophilia
More effective than episodic treatment
WEDNESDAY, Aug. 8 (HealthDay News) -- Among young boys with severe hemophilia, prophylactic treatment with factor VIII considerably reduces the risk of joint damage and hemorrhages compared with episodic treatment, researchers report in the Aug. 9 issue of the New England Journal of Medicine.
Marilyn J. Manco-Johnson, M.D., from the Mountain States Regional Hemophilia and Thrombosis Center in Aurora, Colo., and colleagues randomly assigned 65 boys younger than 30 months with severe hemophilia A to regular prophylactic infusions of recombinant factor VIII, or an enhanced episodic infusion schedule of factor VIII at the time of a joint hemorrhage. Bone or cartilage damage were detected by magnetic resonance imaging.
When the boys were 6 years old, significantly more boys in the prophylactic group had normal index-joint structure (93 percent versus 55 percent). Episodic treatment was associated with a higher risk of joint damage (relative risk 6.1) and significantly higher mean annual numbers of joint and total hemorrhages.
The study "provides renewed and detailed evidence that prophylaxis in severe hemophilia with recombinant factor VIII has clinical benefit despite its huge costs," Goris Roosendaal, M.D., Ph.D., and Floris Lafeber, Ph.D., from University Medical Center Utrecht in the Netherlands, write in an accompanying editorial. They note that the estimated cost of prophylaxis for one patient is $300,000 per year.
Bayer Healthcare donated the factor VIII used in the study.