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Article

Bleeding phenotype according to factor level in 825 children with nonsevere hemophilia: data from the PedNet cohort.

Journal of thrombosis and haemostasis : JTH - 1 Sept 2024

de Kovel Marloes S, Escuriola-Ettingshausen Carmen, Königs Christoph, Ranta Susanna, Fischer Kathelijn

Abstract excerpt

BACKGROUND: Information on bleeding phenotype in nonsevere hemophilia may be used to determine target factor levels for prophylaxis or gene therapy in severe hemophilia. OBJECTIVES: To assess the association between endogenous factor level and bleeding phenotype in children with nonsevere (factor [F]VIII/FIX activity 1%-25%) hemophilia A (HA) and B without prophylaxis. METHODS: Data on annualized bleeding rate...

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PMID: 38866249
DOI: 10.1016/j.jtha.2024.05.030

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