Home > Cardiology > ISTH 2020 > Haemophilia and Rare Bleeding Disorders > Recombinant factor VIII safe and effective in PUPs A-LONG study

Recombinant factor VIII safe and effective in PUPs A-LONG study

Presented By
Dr Christoph Königs, University Hospital Frankfurt, Germany
Conference
ISTH 2020
Trial
Phase 3, PUPs A-LONG
In the first prospective study of an extended half-life recombinant factor VIII (rFVIII) treatment for previously untreated patients with severe haemophilia A, rFVIII was well tolerated. Promisingly, the high-titre inhibitor rate was lower than that seen in the literature [1]. The development of alloantibody inhibitors that interfere with prophylaxis and treatment is the largest safety concern in infants with severe haemophilia A, as it occurs in up to 37% of the infant population. Recombinant FVIII products offer a technological solution by reducing the risk of formation of alloantibody inhibitors against FVIII, as well as prolonging the half-life of the protein. The open-label, single-arm, multicentre, phase 3 PUPs A-LONG study investigated the safety and efficacy of rFV...


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