Compliance with Early Long-Term Prophylaxis Guidelines for Severe Hemophilia A.

Objectives To evaluate the applicability and compliance with guidelines for early initiation of long-term prophylaxis in infants with severe hemophilia A and to identify factors associated with guideline compliance. Study design This real-world, prospective, multicenter, population-based FranceCoag study included almost all French boys with severe hemophilia A, born between 2000 and 2009 (i.e. after guideline implementation). Results We included 333 boys in the study cohort. The cumulative incidence of LTP use was 61.2% at three years of age vs. 9.5% in a historical cohort of 39 boys born in 1996 (i.e. before guideline implementation). The guidelines were not applicable in 23.1% of patients due to an early intracranial bleeding or inhibitor development. LTP was delayed in 10.8% of patients. In the multivariate analysis, two variables were significantly associated with “timely LTP” as compared with “delayed LTP”: Hemophilia treating center location in the southern regions of France (OR 23.6, 95% CI 1.9-286.7, p=0.013 vs. Paris Area) and older age at LTP indication (OR 7.2 for each additional year, 95% CI 1.2-43.2, p=0.031) ( Figure 3 ). LTP anticipation was observed in 39.0% of patients. Earlier birth year (OR 0.5, 95%CI 0.3-0.8, p=0.010 for birth years 2005-2009 vs. 2000-2004) and age at first factor replacement (OR 1.9 for each additional year, 95%CI 1.2-3.0, P = .005) were significantly associated with “LTP guideline compliance” vs “LTP anticipation.” Conclusions This study suggests that LTP guidelines are associated with increased LTP use. However, early initiation of LTP remains a challenge.