Kulagin Alexander D, Ptushkin Vadim V, Lukina Elena A, Davydkin Igor L, Korobkin Alexander V, Konstantinova Tatiana S, Komartseva Elena Yu, Minaeva Natalia V, Mitina Tatiana A, Klimova Olesya U, Arshanskaya Evgeniya G, Latyshev Vitalii D, Markova Oksana A, Zuev Eugene V
RM Gorbacheva Research Institute, Pavlov University, Saint Petersburg, Russian Federation.
Moscow City Hematology Center, Botkin Moscow City Clinical Hospital, Moscow, Russian Federation.
Acta Haematol. 2025;148(4):443-451. doi: 10.1159/000542294. Epub 2024 Nov 25.
The study aimed to assess the safety, immunogenicity, and efficacy of long-term therapy with biosimilar of eculizumab (Elizaria®) in paroxysmal nocturnal hemoglobinuria (PNH) patients.
The study included 30 patients with PNH who had completed previous clinical trials. Of these, 25 patients continued receiving the biosimilar product, and 5 patients switched from the originator product Soliris. The maximum duration of follow-up was 104 weeks, during which the investigational product was administered 52 times at a standard dose.
Throughout the study, the levels of lactate dehydrogenase, hemoglobin, reticulocytes, and PNH clone remained stable compared to baseline, regardless of the previous therapy (p > 0.05). There were no significant differences in the number of patients with chronic kidney disease at different visits, as well as in the number of patients who received donor red blood cell and platelet transfusions during the study (p > 0.05). There were 2 cases of adverse reactions reported in 2 patients (6.6%): elevated aspartate aminotransferase (3.3%) and alopecia (3.3%). Immunogenicity analysis showed no significant differences in the frequency of antidrug antibody detection compared to baseline (p > 0.05).
The study findings confirm the long-term efficacy and safety of biosimilar in patients with PNH.
本研究旨在评估依库珠单抗生物类似药（Elizaria®）长期治疗阵发性睡眠性血红蛋白尿（PNH）患者的安全性、免疫原性和疗效。
本研究纳入了30例已完成先前临床试验的PNH患者。其中，25例患者继续接受生物类似药产品治疗，5例患者从原研产品Soliris转换而来。随访的最长持续时间为104周，在此期间，研究产品按标准剂量给药52次。
在整个研究过程中，无论先前的治疗如何，与基线相比，乳酸脱氢酶、血红蛋白、网织红细胞和PNH克隆水平均保持稳定（p>0.05）。不同访视时慢性肾脏病患者数量以及研究期间接受供体红细胞和血小板输注的患者数量均无显著差异（p>0.05）。有2例患者（6.6%）报告了不良反应：天冬氨酸转氨酶升高（3.3%）和脱发（3.3%）。免疫原性分析显示，与基线相比，抗药抗体检测频率无显著差异（p>0.05）。
研究结果证实了生物类似药在PNH患者中的长期疗效和安全性。
