A study published in Haemophilia demonstrated it is possible to successfully transition children with hemophilia A from standard half-life coagulation factor VIII to extended half-life products.
To date, only a few studies have looked at the real-world clinical use of extended half-life coagulation factors in children with hemophilia.
Here, a team of researchers led by Helen Pergantou from the Haemophilia Centre/Haemostasis and Thrombosis Unit at “Aghia Sophia” Children’s Hospital in Athens, Greece, retrospectively examined real-life experience with the use of extended half-life factor VIII products in children with hemophilia A and compared this to the clinical experience with standard half-life products.
The researchers analyzed 23 children with severe hemophilia A, aged 3 to 6 years. The children were treated with extended half-life factor VIII concentrates for a median of 78 weeks.
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After switching to extended half-life products from standard half-life products, the median dosing interval increased from 2.3 days to 3.5 days. Moreover, the mean trough levels of factor VIII increased from 2.3% to 4.1% with extended half-life products.
Importantly, the mean annual bleeding and joint bleeding rates decreased following treatment with extended half-life products. However, after the switch, the actual factor VIII consumption was significantly increased. There was no inhibitor development following the switch to extended half-life products.
The researchers concluded that prophylaxis with extended half-life products is safe in children with hemophilia A. They added that children with severe hemophilia A being treated prophylactically with standard half-life factor VIII products “could be considered for switching to an [extended half-life] factor so as to have fewer injections, better compliance, better bleeding outcomes and lower bleeding rates.”
Dettoraki A, Michalopoulou A, Mazarakis M, et al. Clinical application of extended half-life factor VIII in children with severe haemophilia A. Haemophilia. Published online May 3, 2022. doi:10.1111/hae.14576