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
Craig M. Kessler, Fernando F. Corrales‐Medina, Pier Mannuccio Mannucci, Víctor Jiménez‐Yuste, Michael D. Tarantino - Hematology
- General Medicine
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.
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.
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%.
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.