Abstract
Retrospective and prospective studies unambiguously show that prophylactic treatment of severe hemophilia A or B should be started as primary prophylaxis at 1–2 years’ of age and ideally before the first joint bleed. The dose and dose frequency should be individually tailored depending on the goal of treatment, venous access and the bleeding phenotype. The circumstances during the first exposures of factor VIII concentrates in hemophilia A may have an impact on the risk of developing inhibitors. Enhanced half-life products, in particular in hemophilia B, will facilitate treatment in patients with difficult venous access but also in achieving a higher trough level. Evidence accumulate that prophylactic treatment is beneficial also in adults and in patients with inhibitors.
Highlights
According to a joint statement made by the World Health Organization (WHO) and the World Federation of Hemophilia (WFH), initiating prophylactic treatment at an early age is considered to be the optimal form of therapy for a child with severe hemophilia [1,2,3]
According to a suggested definition from the Scientific and Standardization Committee (SSC) of the International Society on Thrombosis and Haemostasis (ISTH) [4], primary prophylaxis is a continuous therapy starting after the first joint bleed and before the age of 3 years
Initiating treatment at an early age is the optimal form of therapy for a child with hemophilia A or B
Summary
According to a joint statement made by the World Health Organization (WHO) and the World Federation of Hemophilia (WFH), initiating prophylactic treatment at an early age is considered to be the optimal form of therapy for a child with severe hemophilia [1,2,3]. According to a suggested definition from the Scientific and Standardization Committee (SSC) of the International Society on Thrombosis and Haemostasis (ISTH) [4], primary prophylaxis is a continuous therapy starting after the first joint bleed and before the age of 3 years. Primary prophylaxis can be a continuous treatment started before the age of 3 years in a patient without any previous joint bleed (i.e. initiated based solely on age).
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