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Observational Study
. 2025 Feb;114(2):248-257.
doi: 10.1111/ejh.14309. Epub 2024 Oct 21.

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

Johannes Oldenburg  1 , Charles Hay  2 , Flora Peyvandi  3   4 , Annarita Tagliaferri  5 , Pål Andrè Holme  6 , María Teresa Álvarez-Román  7 , Christine Biron-Andréani  8 , Håkan Malmström  9 , Linda Bystrická  10 , Stefan Lethagen  10   11 ; A‐SURE Study Group
Collaborators, Affiliations
Observational Study

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

Johannes Oldenburg et al. Eur J Haematol. 2025 Feb.

Abstract

Objectives: 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.

Methods: 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.

Results: 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 < 0.0001); and mean annualized factor consumption 243 024.2 versus 288 718.6 International Units (difference of 45 694.5; p = 0.0003). rFVIIIFc was well tolerated, with no inhibitor development.

Conclusions: rFVIIIFc has superior prophylactic effectiveness versus SHL FVIII, providing higher bleed protection with fewer injections and lower factor consumption.

Keywords: factor VIII; hemophilia A; propensity score; prospective study.

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Conflict of interest statement

J.O. declares grants or contracts from Bayer, Biotest, CSL Behring, Octapharma, Pfizer, Sobi, and Takeda paid to the Medical Faculty University of Bonn; payment or honoraria from Bayer, Biogen Idec, Biomarin, Biotest, CSL Behring, Chugai, Freeline, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Spark Therapeutics, Sobi, and Takeda; and support for attending meetings/travel from Bayer, Biogen Idec, Biomarin, Biotest, CSL Behring, Chugai, Freeline, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Spark Therapeutics, Sobi, and Takeda; has participated in data safety monitoring/advisory boards for Bayer, Biogen Idec, Biomarin, Biotest, CSL Behring, Chugai, Freeline, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Spark Therapeutics, Sobi, and Takeda; leadership or fiduciary role in other board, society, committee or advocacy, paid or unpaid for the GTH, and foundation “Hämotherapie Forschung”; and receipt of equipment materials, drugs, medical writing, gifts, or other services from Bayer to the Medical Faculty University of Bonn. C.H. has received consulting fees from Sobi, CSL Behring, and Roche, declares payment or honoraria from Roche, Sobi, and Biotest; support for attending meetings and/or travel for Roche, CSL Behring, and Sobi; and leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid as Director of the UK Haemophilia Database. F.P. declares honoraria from Grifols, Roche, and Sobi, as well as participation in data safety monitoring/advisory boards for Biomarin, Roche, Sobi, and Takeda. A.T. has received consulting fees from Bayer and support for attending meetings from Bayer and Sobi. P.A.H. declares grants or contracts from Bayer, Pfizer, Roche, and Sobi paid to their institution; payment or honoraria for lectures and advisory boards from Bayer, BMS, CSL Behring, NovoNordisk, Octapharma, Pfizer, Roche, Sobi, and Takeda; support for attending meetings and/or travel from Bayer, CSL Behring, NovoNordisk, Octapharma, Pfizer, Roche, Sobi, and Takeda; and leadership or fiduciary role in other board, society, committee or advocacy, paid or unpaid as a member of the executive committee of ACHIEVE group and ADVANCE group. M.T.Á.‐R. has participated as a speaker and in advisory boards and sponsored symposia, with Amgen, CSL Behring, Novartis, NovoNordisk, Octapharma, Pfizer, Roche, Sobi, and Takeda. C.B.‐A. has received consulting fees from CSL Behring, Takeda, and LFB and declares payment or honoraria for LFB. S.L. is a shareholder of stock or stock options of Sobi. S.L., H.M., and L.B. are employees of Sobi.

Figures

FIGURE 1
FIGURE 1
A‐SURE non‐interventional study design. †Switches in prophylaxis were allowed during this period. EOS, end of study; FVIII, factor VIII; rFVIIIFc, recombinant factor VIII Fc fusion protein; SHL, standard half‐life.
FIGURE 2
FIGURE 2
Patient disposition flow chart. FVIII, factor VIII; rFVIIIFc, recombinant factor VIII Fc fusion protein; SHL, standard half‐life.
See this image and copyright information in PMC

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