Real-world safety and effectiveness of rurioctocog alfa pegol in 338 patients with hemophilia A in South Korea: A postmarketing surveillance study

Abstract

Introduction

Rurioctocog alfa pegol is a recombinant coagulation factor VIII (FVIII) with an extended half-life versus the parent product. Limited real-world data exist on the treatment of hemophilia A with rurioctocog alfa pegol in South Korea. This postmarketing surveillance study assessed its safety and effectiveness in a real-world setting, according to Korean regulatory requirements.

Methods

In this prospective, multicenter study (NCT03824522), data were collected from medical records of patients, including children, with hemophilia A receiving rurioctocog alfa pegol as standard clinical practice in South Korea. Safety (adverse events [AEs], adverse drug reactions [ADRs], and unexpected AEs/ADRs) and hemostatic effectiveness were analyzed.

Results

In total, 338 patients were included (mean age [range], 25.0 [1.0–61.0] years; children <12 years, n = 54 [16 %]; all had a history of FVIII treatment). AEs occurred in 20 (5.9 %) patients (unexpected AEs, n = 18 [5.3 %]; ADRs, n = 4 [1.2 %]; unexpected ADRs, n = 2 [0.6 %]; serious AEs, n = 4 [1.2 %]; serious ADRs, n = 0 [0.0 %]). Among children, AEs occurred in 1 (50 %) and 6 (11.5 %) patients aged <2 and ≥2 to <12 years, respectively. No new safety signals were observed. No bleeding events occurred in 239 (76.1 %) patients on prophylaxis. Hemostatic effectiveness was rated by physicians as excellent/good in most patients. Bleeding events were managed with 1–2 infusions. Treatment effectiveness was maintained in children during the study period. No patients developed inhibitor antibodies against rurioctocog alfa pegol.

Conclusion

Rurioctocog alfa pegol was effective in children and adults, with no new safety signals; most patients on prophylaxis experienced no bleeds during the study.