Superior Prophylactic Effectiveness of a Recombinant FVIIIFc Over Standard Half-Life FVIII in Hemophilia A: A-SURE Study

• Published in: European journal of haematology 2024-10
• Main Authors: Oldenburg, Johannes, Hay, Charles, Peyvandi, Flora, Tagliaferri, Annarita, Holme, Pål Andrè, Álvarez-Román, María Teresa, Biron-Andréani, Christine, Malmström, Håkan, Bystrická, Linda, Lethagen, Stefan
• Format: Article
• Language: English
• ISSN: 0902-4441; 1600-0609; 1600-0609
• DOI: 10.1111/ejh.14309

The 24-month, prospective, non-interventional, European multicenter A-SURE study evaluated the real-world effectiveness of prophylaxis using an extended half-life recombinant factor VIII (FVIII) Fc fusion protein, efmoroctocog alfa (hereinafter rFVIIIFc), compared with prophylaxis using standard half-life (SHL) FVIII products in patients with hemophilia A. Primary endpoints were annualized bleeding rate (ABR), annualized injection frequency, and annualized factor consumption. A comparative study design unique for an observational hemophilia study was implemented to reduce potential confounding in effectiveness estimates, wherein each patient prescribed rFVIIIFc was matched with one receiving SHL FVIII. Propensity scores were used for adjustment in statistical analyses. Outcomes for all primary endpoints were significantly better in the rFVIIIFc group (n = 184) compared with the SHL FVIII group (n = 170): mean ABR 1.5 versus 2.3 (difference of -0.8; p = 0.0147); mean annualized injection frequency 114.4 versus 169.2 (difference of -54.8; p