Compliance with Early Long-Term Prophylaxis Guidelines for Severe Hemophilia A.
J Pediatr
; 234: 212-219.e3, 2021 Jul.
Article
en En
| MEDLINE
| ID: mdl-33676933
ABSTRACT
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. STUDYDESIGN:
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.
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Pautas de la Práctica en Medicina
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Factores de Coagulación Sanguínea
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Adhesión a Directriz
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Hemofilia A
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Artropatías
Tipo de estudio:
Clinical_trials
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Etiology_studies
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Guideline
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Prognostic_studies
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Risk_factors_studies
Límite:
Child, preschool
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Humans
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Infant
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Male
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Newborn
País/Región como asunto:
Europa
Idioma:
En
Revista:
J Pediatr
Año:
2021
Tipo del documento:
Article