Blood Res  
Subgroup analysis of a phase 2/3 study of rurioctocog alfa pegol in patients with severe hemophilia A: efficacy and safety in previously treated Korean patients
Chur Woo You1, Hee Jo Baek2, Sang Kyu Park3, Young Shil Park4, Ho-Jin Shin5, Werner Engl6, Srilatha Tangada7
Department of Pediatrics, 1Eulji University Hospital, Daejeon, 2Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Gwangju, 3Ulsan University Hospital, Ulsan, 4Kyung Hee University Hospital at Gangdong, Seoul, 5Pusan National University Hospital, Busan, Korea, 6Baxalta Innovations GmbH, Vienna, Austria, 7Baxalta US Inc., Cambridge, MA, USA
Correspondence to: Srilatha Tangada
Baxalta US Inc., 650 East Kendal Street, Cambridge, MA 02142, USA
Email: Srilatha.Tangada@takeda.com
Published online: August 23, 2019.
© The Korean Journal of Hematology. All rights reserved.

Abstract
Background
The efficacy and safety of extended half-life, full-length, pegylated recombinant factor VIII rurioctocog alfa pegol [BAX 855, ADYNOVATE (USA)/ADYNOVI (Europe); Baxalta US Inc., a Takeda company, Lexington, USA] was investigated in previously treated Korean patients with severe hemophilia A (HA).
Methods
A post hoc data analysis from the international, multicenter, phase 2/3 PROLONG-ATE study of rurioctocog alfa pegol in patients with severe HA (NCT01736475) determined annualized bleeding rates (ABRs) and rates of adverse events (AEs) in Korean patients treated in this study.
Results
All 10 enrolled Korean patients receiving rurioctocog alfa pegol (9 prophylaxis, 1 on-demand) completed the study [median (range) age, 28.0 (12-50) yr; weight, 64.8 (45-90) kg; 8 patients had ≥1 target joint at screening]. Median (range) ABR was 1.9 (0.0-14.5) for patients on prophylaxis and 62.2 for patients receiving on-demand treatment. The hemostatic efficacy of rurioctocog alfa pegol was rated “excellent” or “good,” and only single infusions were required per bleeding. ABRs improved in most patients ,compared with prestudy values. No dose adjustments were required for prophylaxis, and the dosing frequency was reduced in 8 patients, compared with their previous prophylaxis regimen. No serious AEs were reported; all 9 nonserious AEs (in 3 patients) were mild in severity and unrelated to the study treatment.
Conclusion
This post hoc analysis of a small group of Korean patients with severe HA indicated that rurioctocog alfa pegol was effective, and no serious AEs were observed. For most patients, the dosing frequency was also reduced compared with that prescribed in their previous regimen.
Keywords: Hemophilia A, Factor VIII, Rurioctocog alfa pegol, BAX 855, Post hoc analysis


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