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

초록

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.

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