Why carry out the study?

Prophylaxis with recombinant factor VIII (rFVIII) replacement therapy is the recommended and most widely accepted treatment strategy to reduce bleeding risks in patients with severe haemophilia A.

Standard-acting rFVIII products like rAHF-PFM have a short half-life leading to the need for injection intervals of 2–3 days to maintain sufficiently high factor activity levels to reduce the risk of bleeding.

Long-acting rFVIII products, such as rVIII-SingleChain and rFVIIIFc, have been designed with improved pharmacokinetic properties to enable longer intervals between injections; these long-acting rFVIII products allow patients to maintain, or even decrease, their bleeding rates, whilst reducing the injection burden.

This study used matching-adjusted indirect comparison (MAIC) to compare the efficacy and consumption of rVIII-SingleChain versus rAHF-PFM and rFVIIIFc.

What was learned from the study?

Prophylactic treatment with rVIII-SingleChain has comparable efficacy to rAHF-PFM but significantly lower consumption. Comparable efficacy and consumption were established when rVIII-SingleChain was compared to rFVIIIFc.

This indirect treatment comparison suggests that with an annualized rFVIII consumption comparable to rFVIIIFc, but significantly lower than rAHF-PFM, routine prophylaxis with rVIII-SingleChain is able to maintain a similar annualized bleeding rate and percentage of patients with zero bleeds, attesting to the long-acting nature of rVIII-SingleChain.