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Open Access 01-09-2021 | Hemophilia | Original Research

Matching-Adjusted Indirect Comparison of Efficacy and Consumption of rVIII-SingleChain Versus Two Recombinant FVIII Products Used for Prophylactic Treatment of Adults/Adolescents with Severe Haemophilia A

Authors: Santiago Bonanad, Ramiro Núñez, Jose Luis Poveda, Karin Kurnik, Georg Goldmann, Valeska Andreozzi, Björn Vandewalle, Sandra Santos

Published in: Advances in Therapy | Issue 9/2021

Abstract

Introduction

Given the relatively small number of patients with haemophilia A, head-to-head comparisons between recombinant FVIII (rFVIII) products are difficult to conduct. This study compared the efficacy and consumption of rVIII-SingleChain (lonoctocog alfa, AFSTYLA^®) with rAHF-PFM (octocog alfa, Advate^®) and rFVIIIFc (efmoroctocog alfa, Elocta^®), for the prophylaxis and treatment of bleeding episodes in previously treated adolescents/adults with severe haemophilia A, through a matching-adjusted indirect comparison (MAIC).

Methods

A systematic literature review identified published clinical trials for rAHF-PFM and rFVIIIFc. Individual patient data for rVIII-SingleChain were used to match baseline patient characteristics to those from published trials, using an approach similar to propensity score weighting. After matching, annualized bleeding rates (ABR), percentage of patients with zero bleeds, and rFVIII consumption were compared across trial populations.

Results

Published data were identified from two rAHF-PFM trials and one rFVIIIFc trial. rVIII-SingleChain had similar ABR (risk ratio [RR]: 0.74 [0.16; 3.48]; RR: 1.18 [0.85; 1.65]) and percentage of patients with zero bleeds (odds ratio [OR]: 1.34 [0.56; 3.22]; OR: 0.78 [0.47; 1.31]) versus rAHF-PFM and rFVIIIFc, respectively. Annual rVIII-SingleChain consumption was significantly lower than rAHF-PFM (mean difference: − 1507.66 IU/kg/year [− 2011.71; − 1003.61]) and equivalent to rFVIIIFc (RR: 0.96 [0.62; 1.49]).

Conclusion

Although limited to published information for comparator trials, these results suggest 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 ABR and percentage of patients with zero bleeds, attesting to the long-acting nature of rVIII-SingleChain.

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Title: Matching-Adjusted Indirect Comparison of Efficacy and Consumption of rVIII-SingleChain Versus Two Recombinant FVIII Products Used for Prophylactic Treatment of Adults/Adolescents with Severe Haemophilia A
Authors: Santiago Bonanad
Ramiro Núñez
Jose Luis Poveda
Karin Kurnik
Georg Goldmann
Valeska Andreozzi
Björn Vandewalle
Sandra Santos
Publication date: 01-09-2021
Publisher: Springer Healthcare
Keyword: Hemophilia
Published in: Advances in Therapy / Issue 9/2021
Print ISSN: 0741-238X
Electronic ISSN: 1865-8652
DOI: https://doi.org/10.1007/s12325-021-01853-0

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