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Risk factors for inhibitor formation in haemophilia: a prevalent case–control study
Haemophilia, 07/01/09
Ragni MV et al. - Inhibitor formation is associated with high intensity product exposure, CNS bleeding, African-American race and low frequency of missense mutations. The ideal time to initiate prophylaxis to reduce CNS bleeding and inhibitor formation will require prospective studies.
Margaret V. Ragni, 06/30/09
| Hemophilia inhibitor formation is increasingly recognized to be related to early high dose bolus factor treatment, a potent antigenic challenge in very young infants. Because these inhibitors are associated with CNS bleeds, high burden of disease, poor outcomes, and early mortality, prevention is the key. Gene transfer studies in hemophilia mice and dogs suggests that low-level continuous factor VIII or IX may avert inhibitor formation. Whether continuous low level factor VIII or IX in such patients will prevent new inhibitor formation is a critical question, and wil require randomized clinical trials. |
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