Choi Eun Jin, Hwang Tai Ju, Choi Yong Mook, Kim Hugh Chul, Yoo Myung Chul, Song Haylee, Badejo Kayode
Department of Pediatrics, Daegu Catholic University Medical Center, Daegu, Korea.
Korea Hemophilia Foundation Clinic, Gwangju, Korea.
Blood Res. 2020 Dec 31;55(4):246-252. doi: 10.5045/br.2020.2020225.
Rixubis (recombinant factor IX, nonacog gamma) is indicated for the control and prevention of bleeding episodes, perioperative management, and routine prophylaxis in hemophilia B patients. This real-world, postmarketing surveillance study aimed to evaluate the safety and effectiveness of Rixubis in adult and pediatric hemophilia B patients in South Korea.
This prospective, observational, multicenter study (clinicaltrials.gov identifier: NCT029 22231) was conducted in hemophilia B patients between April 2015 and April 2019, who were observed for up to 6 months after the initiation of Rixubis treatment. Safety was evaluated based on the number and severity of adverse events (AEs) and serious AEs (SAEs). Hemostatic effectiveness was assessed by physicians and patients by using a four-point scale and rated as excellent, good, fair, or no response based on treatment type.
In all, 58 patients were enrolled from four centers by seven physicians during the study period. The safety and effectiveness analysis sets included 57 and 54 patients, respectively. Overall, 11 AEs were reported in eight patients (14.0%), of which three were SAEs and occurred in three patients (5.3%). All 11 AEs were reported as unexpected and mild in severity, with no anaphylactic reaction, and 10 AEs (90.9%) resolved. The majority of AEs (10) were unrelated to Rixubis. Of the 142 hemostatic effectiveness assessments, 123 (86.6%) were reported as good or excellent.
Rixubis demonstrated an acceptable safety and effectiveness profile in the treatment of bleeding, perioperative management, and prophylaxis in hemophilia B patients in a real-world setting in South Korea.
瑞舒立（重组凝血因子IX，非阿考γ）适用于控制和预防B型血友病患者的出血发作、围手术期管理及常规预防。这项真实世界的上市后监测研究旨在评估瑞舒立在韩国成人和儿童B型血友病患者中的安全性和有效性。
这项前瞻性、观察性、多中心研究（clinicaltrials.gov标识符：NCT02922231）于2015年4月至2019年4月在B型血友病患者中进行，患者在开始瑞舒立治疗后接受长达6个月的观察。根据不良事件（AE）和严重不良事件（SAE）的数量及严重程度评估安全性。由医生和患者使用四点量表评估止血效果，并根据治疗类型评为优秀、良好、一般或无反应。
在研究期间，7名医生从4个中心招募了58例患者。安全性和有效性分析集分别包括57例和54例患者。总体而言，8例患者（14.0%）报告了11例AE，其中3例为SAE，发生在3例患者（5.3%）中。所有11例AE均报告为意外且严重程度为轻度，无过敏反应，10例AE（90.9%）得到缓解。大多数AE（10例）与瑞舒立无关。在142次止血效果评估中，123次（86.6%）报告为良好或优秀。
在韩国的真实世界环境中，瑞舒立在治疗B型血友病患者的出血、围手术期管理和预防方面显示出可接受的安全性和有效性。
