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

Abstract

AbstractObjectivesWe aimed to indirectly compare the efficacy of personalized prophylaxis with simoctocog alfa (Nuwiq®) versus three extended half‐life (EHL) recombinant FVIII (rFVIII) concentrates.MethodsTreatment 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.ResultsA 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%.ConclusionsIndirect 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.