Roger Simon D, Wong Chew Ming, Vejakama Phisitt, Rahardjo Kuspudji Dwitanto, Hsu Bang-Gee, Lee Chang Meng, Wu I-Wen, Lee Chin-Chan, Situmorang Tunggul Diapari, Krisanapan Pajaree, Mazlan Sadanah Aqashiah, Peng Yu-Sen, Sarwono Johanes, De Asis Norman, Solimen Domingo, Sangthawan Pornpen, Chen Jin-Bor, Wang Chia-Liang, Chung Sungjin, Villaflor Agnes Jeans, Yang Chul Woo, Kan Wei-Chih, Yang Yu, Rubio-Bicol Jenny, Yan Lee Yee, Lee Sang Ho, Chiu Yi-Wen, Chen Cheng-Hsu, Na Ki Young, Hassah Wan Hasnul Halimi Wan, Kang Young Sun, Choi Bum-Soon, Aquitania Grace, Na Ki Ryang, Wu Mai-Szu, Ahmad Mohd Kamil, Isidto Rey, Wu Vincent, Leong Goh Bak, Sung Junne-Ming, Noppakun Kajohnsak, Chou Kang-Ju, Abdul Wahab Mohamad Zaimi, Shin Seok Joon, Nugroho Pringgodigdo
Renal Research, Gosford, New South Wales, Australia.
University Malaya Medical Centre, Petaling Jaya, Malaysia.
Nephrology (Carlton). 2025 May;30(5):e70046. doi: 10.1111/nep.70046.
Efepoetin alfa, a novel long-acting erythropoietin (EPO)-hybrid Fc fusion protein, represents a promising erythropoiesis-stimulating agent (ESA) for addressing anaemia in chronic kidney disease (CKD) patients. This Phase 3 trial was to assess the efficacy and tolerability of subcutaneous efepoetin alfa in comparison to subcutaneous methoxy polyethylene glycol-epoetin beta in stage 3 or 4 CKD patients.
A randomised, multicentre, open-label Phase 3 trial enrolled 391 CKD stage 3 or stage 4 patients. Subjects underwent a 20-week correction period followed by an 8-week evaluation period. Responders continued treatment for an extra 24-week extension to evaluate long-term safety, maintenance effectiveness, and the longer treatment interval.
In the efepoetin alfa Q2W (every 2 weeks) group, the response rate was 75.6%; while in the methoxy polyethylene glycol-epoetin beta Q2W group, the response rate was 69.3%. The difference in the response rate was 6.3% with 95% CI (confidence interval) -3.1% to 15.5%. The lower limit of the 95% CI was above the prespecified non-inferiority margin of -9.0%. Adverse event rates were comparable between the treatment groups.
Efepoetin alfa demonstrated non-inferiority to methoxy polyethylene glycol-epoetin beta in correcting anaemia and maintaining haemoglobin (Hb) levels among stage 3 and 4 CKD patients. Moreover, the safety profile of efepoetin alfa was comparable to methoxy polyethylene glycol-epoetin beta.
阿法依泊汀是一种新型长效促红细胞生成素（EPO）-杂交Fc融合蛋白，是一种有前景的促红细胞生成剂（ESA），用于治疗慢性肾脏病（CKD）患者的贫血。这项3期试验旨在评估皮下注射阿法依泊汀与皮下注射甲氧基聚乙二醇化促红细胞生成素β相比，在3期或4期CKD患者中的疗效和耐受性。
一项随机、多中心、开放标签的3期试验纳入了391例3期或4期CKD患者。受试者先经历20周的校正期，随后是8周的评估期。有反应者继续接受额外24周的延长治疗，以评估长期安全性、维持有效性以及更长的治疗间隔。
在阿法依泊汀每2周一次（Q2W）组中，反应率为75.6%；而在甲氧基聚乙二醇化促红细胞生成素β每2周一次组中，反应率为69.3%。反应率差异为6.3%，95%置信区间（CI）为-3.1%至15.5%。95%CI的下限高于预先设定的非劣效性界值-9.0%。治疗组之间的不良事件发生率相当。
在纠正3期和4期CKD患者贫血及维持血红蛋白（Hb）水平方面，阿法依泊汀显示出不劣于甲氧基聚乙二醇化促红细胞生成素β。此外，阿法依泊汀的安全性与甲氧基聚乙二醇化促红细胞生成素β相当。
