RESUMO
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 (ie, after guideline implementation). RESULTS: We included 333 boys in the study cohort. The cumulative incidence of long-term prophylaxis use was 61.2% at 3 years of age vs 9.5% in a historical cohort of 39 boys born in 1996 (ie, before guideline implementation). The guidelines were not applicable in 23.1% of patients due to an early intracranial bleeding or inhibitor development. Long-term prophylaxis was delayed in 10.8% of patients. In the multivariate analysis, 2 variables were significantly associated with "timely long-term prophylaxis" as compared with "delayed long-term prophylaxis": hemophilia treating center location in the southern regions of France (OR 23.6, 95% CI 1.9-286.7, P = .013 vs Paris area) and older age at long-term prophylaxis indication (OR 7.2 for each additional year, 95% CI 1.2-43.2, P = .031). Long-term prophylaxis anticipation was observed in 39.0% of patients. Earlier birth year (OR 0.5, 95% CI 0.3-0.8, P = .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 "long-term prophylaxis guideline compliance" vs "long-term prophylaxis anticipation." CONCLUSIONS: This study suggests that long-term prophylaxis guidelines are associated with increased long-term prophylaxis use. However, early initiation of long-term prophylaxis remains a challenge.
Assuntos
Fatores de Coagulação Sanguínea/administração & dosagem , Fidelidade a Diretrizes/estatística & dados numéricos , Hemofilia A/complicações , Artropatias/prevenção & controle , Padrões de Prática Médica/estatística & dados numéricos , Fatores de Coagulação Sanguínea/uso terapêutico , Pré-Escolar , Esquema de Medicação , França , Humanos , Lactente , Recém-Nascido , Artropatias/etiologia , Estimativa de Kaplan-Meier , Modelos Logísticos , Masculino , Guias de Prática Clínica como Assunto , Padrões de Prática Médica/normas , Índice de Gravidade de DoençaRESUMO
OBJECTIVES: To evaluate the impact of the 2002 guidelines on the current status of prophylaxis in French children with severe hemophilia A or B. STUDY DESIGN: Clinical information was captured, in a prospective way, using FranceCoag Network. We retrospectively studied 291 patients with severe (<1 IU/dL) hemophilia A and B, with no history of inhibitors. RESULTS: Our results demonstrate that the availability of national medical guidelines has improved clinical practice in France. In the past decade, the proportion of children with severe hemophilia undergoing prophylaxis has shown a significant 2- to 3-fold increase: â¼80% of these children>3 years of age are now receiving prophylaxis. In severe hemophilia A and B, the age at which prophylaxis commences has significantly decreased: 4.0 and 6.1 years for the period 1996-1999 as opposed to 1.8 and 1.4 years for the period 2004-2007 (P=.0001). CONCLUSIONS: Long-term clinical and physical evaluations of patients will be necessary to establish the benefits of this increase in prophylactic treatment on the prevention of hemophilic arthropathy.