Bleeding before prophylaxis in severe hemophilia: paradigm shift over two decades

Abstract

Bleeding before prophylaxis in severe hemophilia: paradigm shift over two decades Prophylaxis for hemophilia is the scheduled infusion of the missing clotting factor with pre-specified dose, with the intention of preventing bleeds and subsequent hemophilic arthropathy. It is the treatment of choice for patients with severe hemophilia A in countries with available resources.

Long‐term effects of joint bleeding before starting prophylaxis in severe haemophilia

“…Prophylaxis was defined as the regular administration of FVIII/IX at least once a week . Over time prophylaxis was started earlier and has intensified . At the Van Creveldkliniek, generally one joint bleed is allowed for before starting prophylaxis.…”

“…The main objective of prophylaxis with clotting factor VIII/IX (FVIII/FIX) in children with severe haemophilia (FVIII/FIX < 0.01 IU mL −1 ) is prevention of joint bleeds and subsequent haemophilic arthropathy . Early initiation of prophylaxis proved to be critical for effective prevention of arthropathy and consequently prophylaxis has been started at an increasingly younger age and after fewer (joint) bleeds . However, the optimum time for starting prophylaxis in young children with severe haemophilia has not yet been established.…”

When and how to start prophylaxis in boys with severe hemophilia without inhibitors: communication from the SSC of the ISTH

“…During the past few decades, early initiation and an increase in prophylactic dose resulted in fewer bleeds and improved joint status [3,4]. This trend was also observed in the PedNet cohort of 919 boys with severe hemophilia A, where prophylaxis is currently initiated at a median age of 1.3 years (interquartile range [IQR], 0.9-1.9) after a median of one joint bleed, with 70% of patients fulfilling the criteria of primary prophylaxis [5].…”

Large scale studies assessing anti‐factor VIII antibody development in previously untreated haemophilia A: what has been learned, what to believe and how to learn more

“…Whilst this might not be a problem for within‐cohort comparisons, it might inflate the variability of across cohort and pooled analyses. In addition, some studies span a long observation period and very different settings, and therefore include differences in treatment strategies between centres as well as differences in treatment intensity over time (Nijdam et al , ).…”