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Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia.

Authors :
Manco-Johnson, Marilyn J.
Abshire, Thomas C.
Shapiro, Amy D.
Riske, Brenda
Hacker, Michele R.
Kilcoyne, Ray
Ingram, J. David
Manco-Johnson, Michael L.
Funk, Sharon
Jacobson, Linda
Valentino, Leonard A.
Hoots, W. Keith
Buchanan, George R.
DiMichele, Donna
Recht, Michael
Brown, Deborah
Leissinger, Cindy
Bleak, Shirley
Cohen, Alan
Mathew, Prasad
Source :
New England Journal of Medicine. 8/9/2007, Vol. 357 Issue 6, p535-544. 10p. 3 Diagrams.
Publication Year :
2007

Abstract

Background: Effective ways to prevent arthropathy in severe hemophilia are unknown. Methods: We randomly assigned young boys with severe hemophilia A to regular infusions of recombinant factor VIII (prophylaxis) or to an enhanced episodic infusion schedule of at least three doses totaling a minimum of 80 IU of factor VIII per kilogram of body weight at the time of a joint hemorrhage. The primary outcome was the incidence of bone or cartilage damage as detected in index joints (ankles, knees, and elbows) by radiography or magnetic resonance imaging (MRI). Results: Sixty-five boys younger than 30 months of age were randomly assigned to prophylaxis (32 boys) or enhanced episodic therapy (33 boys). When the boys reached 6 years of age, 93% of those in the prophylaxis group and 55% of those in the episodic-therapy group were considered to have normal index-joint structure on MRI (P=0.006). The relative risk of MRI-detected joint damage with episodic therapy as compared with prophylaxis was 6.1 (95% confidence interval, 1.5 to 24.4). The mean annual numbers of joint and total hemorrhages were higher at study exit in the episodic-therapy group than in the prophylaxis group (P<0.001 for both comparisons). High titers of inhibitors of factor VIII developed in two boys who received prophylaxis; three boys in the episodic-therapy group had a life-threatening hemorrhage. Hospitalizations and infections associated with central-catheter placement did not differ significantly between the two groups. Conclusions: Prophylaxis with recombinant factor VIII can prevent joint damage and decrease the frequency of joint and other hemorrhages in young boys with severe hemophilia A. (ClinicalTrials.gov number, NCT00207597.) N Engl J Med 2007;357:535-44. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
00284793
Volume :
357
Issue :
6
Database :
Academic Search Index
Journal :
New England Journal of Medicine
Publication Type :
Academic Journal
Accession number :
26115090
Full Text :
https://doi.org/10.1056/NEJMoa067659