Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia
| Autor: | Amy D. Shapiro, George R. Buchanan, Gregory Thomas, Alexis A. Thompson, Harlan Austin, Michael Recht, Michele R. Hacker, Kevin McRedmond, Sharon Funk, Alison Matsunaga, Donna DiMichele, Diane J. Nugent, J. David Ingram, Desiree Medeiros, Thomas C. Abshire, Marilyn J. Manco-Johnson, Ray F. Kilcoyne, Linda J. Jacobson, W. Keith Hoots, Alan R. Cohen, Leonard A. Valentino, Brenda Riske, Shirley Bleak, J. Michael Soucie, Bruce L. Evatt, Deborah L Brown, Prasad Mathew, M L Manco-Johnson, Cindy A. Leissinger |
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| Rok vydání: | 2007 |
| Předmět: |
Male
Pediatrics medicine.medical_specialty Hemorrhage Hemophilia A Drug Administration Schedule Hemarthrosis Arthropathy medicine Coagulopathy Humans Child Infusions Intravenous Emicizumab Factor VIII business.industry Infant General Medicine Turoctocog alfa medicine.disease Confidence interval Treatment Outcome El Niño Child Preschool Relative risk Joint Diseases business Follow-Up Studies |
| Zdroj: | New England Journal of Medicine. 357:535-544 |
| ISSN: | 1533-4406 0028-4793 |
| DOI: | 10.1056/nejmoa067659 |
| Popis: | Effective ways to prevent arthropathy in severe hemophilia are unknown.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).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 (P0.001 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.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 [ClinicalTrials.gov].). |
| Databáze: | OpenAIRE |
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