Back to Search Start Over

Clinical efficacy of simoctocog alfa versus extended half-life recombinant FVIII concentrates in hemophilia A patients undergoing personalized prophylaxis using a matching-adjusted indirect comparison method.

Authors :
Kessler CM
Corrales-Medina FF
Mannucci PM
Jiménez-Yuste V
Tarantino MD
Source :
European journal of haematology [Eur J Haematol] 2023 Nov; Vol. 111 (5), pp. 757-767. Date of Electronic Publication: 2023 Aug 16.
Publication Year :
2023

Abstract

Objectives: We aimed to indirectly compare the efficacy of personalized prophylaxis with simoctocog alfa (Nuwiq®) versus three extended half-life (EHL) recombinant FVIII (rFVIII) concentrates.<br />Methods: Treatment effects were compared using matching-adjusted indirect comparisons after matching individual patient-level baseline characteristics for simoctocog alfa (pharmacokinetic [PK]-guided personalized prophylaxis) against published aggregate personalized prophylaxis data for efmoroctocog alfa, damoctocog alfa pegol, and rurioctocog alfa pegol.<br />Results: A higher percentage (p < .001) of patients with zero bleeds was found with simoctocog alfa compared with efmoroctocog alfa (75% vs. 45%), damoctocog alfa pegol (77% vs. 38%), and rurioctocog alfa pegol (target trough level 1%-3%; 78% vs. 42%). Similar efficacy was found comparing simoctocog alfa against rurioctocog alfa pegol 8%-12% (77% vs. 62%). The mean total annualized bleeding rate was lower (p < .001) with simoctocog alfa than damoctocog alfa pegol (1.5 vs. 4.9). Consistent with approved dosing, the mean FVIII weekly dose was higher (p < .001) for simoctocog alfa than efmoroctocog alfa, damoctocog alfa pegol, or rurioctocog alfa pegol 1%-3%, but lower (p < .001) than rurioctocog alfa pegol 8%-12%.<br />Conclusions: Indirect comparisons demonstrated that PK-guided, personalized prophylaxis with simoctocog alfa can lead to higher zero bleed rates compared with personalized EHL rFVIII concentrate regimens, albeit with higher weekly doses, and a lower percentage of patients treated twice weekly or less.<br /> (© 2023 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd.)

Details

Language :
English
ISSN :
1600-0609
Volume :
111
Issue :
5
Database :
MEDLINE
Journal :
European journal of haematology
Publication Type :
Academic Journal
Accession number :
37587687
Full Text :
https://doi.org/10.1111/ejh.14073