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Observational Study
. 2019 May 30;9(5):e028012.
doi: 10.1136/bmjopen-2018-028012.

Design of a prospective observational study on the effectiveness and real-world usage of recombinant factor VIII Fc (rFVIIIFc) compared with conventional products in haemophilia A: the A-SURE study

Johannes Oldenburg  1 , Charles R M Hay  2 , Víctor Jiménez-Yuste  3 , Flora Peyvandi  4   5 , Jean-François Schved  6 , Johan Szamosi  7 , Bent Winding  8 , Stefan Lethagen  9   10
Affiliations
Observational Study

Design of a prospective observational study on the effectiveness and real-world usage of recombinant factor VIII Fc (rFVIIIFc) compared with conventional products in haemophilia A: the A-SURE study

Johannes Oldenburg et al. BMJ Open. .

Abstract

Introduction: Haemophilia A is a rare bleeding disorder caused by coagulation factor VIII (FVIII) deficiency. This is treated with factor VIII, conventionally using products with a half-life of 8-12 hours typically administered every 2-3 days. Recombinant FVIII Fc (rFVIIIFc) represents a new generation of products with an extended half-life allowing higher FVIII levels and longer dosing interval. The efficacy and safety of rFVIIIFc have been established in clinical studies and several years of postmarketing use. However, there remains a need to compare treatment outcome with conventional products in routine clinical use.

Methods and analysis: A-SURE is an ongoing, non-interventional European study with the primary objective to compare the clinical effectiveness of rFVIIIFc with conventional factor products used for haemophilia A prophylaxis. Data covering a 24-month prospective period and a 12-month retrospective period will be collected. Three primary endpoints: bleeding rate, injection frequency and factor consumption will be used to evaluate treatment outcomes. Enrolment of 175 patients on rFVIIIFc and 175 on conventional products is planned. All eligible patients from participating centres will be invited to participate. Visits and treatments follow routine clinical practice. Bias will be reduced by patient matching for age at baseline and the last weekly prophylaxis dose of a conventional product prior to baseline. Propensity scores will be calculated based on prognostic factors and potential confounders assessed at baseline and adjusted for in the estimation of the treatment effect.

Ethics and dissemination: Study approval was obtained by local independent ethics committees and/or authorities, and informed consent from patients or their legal representative is a requirement for participation. Names of ethical committees and approval numbers are provided as supplementary information. The study results will be submitted for publication in a peer-reviewed scientific journal and presented at scientific conferences.

Trial registration number: NCT02976753, Pre-results.

Keywords: haemophilia A; non-interventional study; recombinant factor VIII Fc.

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Conflict of interest statement

Competing interests: JO received grant/research support from Bayer, Biotest, CSL Behring, Novo Nordisk, Octapharma and Shire. Personal fees has been received for travel support, participation in advisory boards and participating in symposia as chair or speaker from Bayer, Biotest, CSL Behring, Novo Nordisk, Octapharma, Shire, Chugai, Grifols, Pfizer, Roche and Sobi. CRMH involved in clinical trials or observational research with Sobi, Bayer, Novo, Roche, Alnylam, Biomarin, Pfizer and Shire; speaker’s bureaus for Shire, Biotest, Sobi, Pfizer and Roche. VJ-Y received grant/research support from Bayer, Biotest, Grifols, Novo Nordisk, Octapharma, Pfizer, Shire and Sobi; Speaker Bureau with Bayer, Biogen, Novo Nordisk, Pfizer, Roche, Shire and Sobi. FP received honoraria for participating as a speaker with Bayer, Grifols, Sobi, Shire, F. Hoffmann-La Roche, Alnylam; received consulting fees from Kedrion and LFB; has been the member of scientific advisory boards of F. Hoffmann-La Roche and Shire. J-FS received grants from Sobi and Behring. JS, BW and SL are the employees of Sobi and holders of Sobi shares.

Figures

Figure 1
Figure 1
A-SURE study design. A-SURE is a 24-month prospective observational study also including the collection of 12 months prebaseline data. The prophylactic factor treatment will be prescribed according to usual clinical practice and patients prescribed recombinant factor VIII Fc (rFVIIIFc) will be enrolled in the rFVIIIFc treatment arm and matched with patients with similar baseline characteristics receiving conventional coagulation factor VIII (FVIII) therapy.
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