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 You; Hee Jo Baek; Sang Kyu Park; Young Shil Park; Ho-Jin Shin; Werner Engl; Srilatha Tangada

doi:10.5045/br.2019.54.3.198

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Original Article

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Blood Res 2019; 54(3):

Published online September 30, 2019

https://doi.org/10.5045/br.2019.54.3.198

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

Author Info. +

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, a Takeda Company, Vienna, Austria, ⁷Baxalta US Inc., a Takeda Company, Cambridge, MA, USA

Correspondence to : Srilatha Tangada, M.D.
Baxalta US Inc., a Takeda Company, 650 East Kendall Street, Cambridge, MA 02142, USA
E-mail: Srilatha.Tangada@takeda.com

Received: March 22, 2019; Revised: May 23, 2019; Accepted: July 31, 2019

This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.

Abstract

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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, MA, 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 the patient receiving on-demand treatment. The hemostatic efficacy of rurioctocog alfa pegol was rated “excellent” or “good” and only single infusions were required per bleeding episode. 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 their previous regimen.

Keywords Hemophilia A, Factor VIII, Rurioctocog alfa pegol, BAX 855, Post hoc analysis

Article

Original Article

Blood Res 2019; 54(3): 198-203

Published online September 30, 2019 https://doi.org/10.5045/br.2019.54.3.198

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

Correspondence to:Srilatha Tangada, M.D.
Baxalta US Inc., a Takeda Company, 650 East Kendall Street, Cambridge, MA 02142, USA
E-mail: Srilatha.Tangada@takeda.com

Received: March 22, 2019; Revised: May 23, 2019; Accepted: July 31, 2019

Abstract

Background

Methods

Results

Conclusion

Keywords: Hemophilia A, Factor VIII, Rurioctocog alfa pegol, BAX 855, Post hoc analysis

Abstract

Demographics of Korean patients with severe hemophilia A.

Hemophilia gene mutation was unknown for all 10 Korean study patients..

Abbreviations: NA, not applicable; NR, not reported..

Comparison of on-study annualized bleeding rates (ABRs) with prestudy ABRs in 9 patients with severe hemophilia A receiving prophylaxis with rurioctocog alfa pegol.

Abbreviation: ABR, annualized bleeding rate..

On-study annualized bleeding rate by bleeding site and cause.

Abbreviation: NA, not applicable..

Characteristics of bleeding episodes in patients receiving prophylaxis and on-demand treatment with rurioctocog alfa pegol.

Severity of bleeding episodes was defined as “mild,” little or no pain, little or no change in the range of motion of the affected joint, and mild restriction of mobility and activity; “moderate,” mild or moderate pain, some decrease in range of movement of the affected joint, and moderate decrease in mobility and activity; and “severe,” significant pain, substantial decrease in the range of motion of the affected joint, incapacity, and life-threatening..

Abbreviation: NA, not applicable..