Supplementary Material for: RESULTS OF LONG-TERM THERAPY WITH A BIOSIMILAR OF ECULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

Introduction. The study aimed to assess the safety, immunogenicity, and efficacy of long-term therapy with biosimilar of eculizumab (Elizaria®) in PNH patients. Methods. 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. Results. Throughout the study, the levels of LDH, 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 two cases of adverse reactions reported in two patients (6.6%): elevated aspartate aminotransferase (3.3%) and alopecia (3.3%). Immunogenicity analysis showed no significant differences in the frequency of anti-drug antibody detection compared to baseline (p > 0.05). Conclusion. The study findings confirm the long-term efficacy and safety of biosimilar in patients with PNH.

引言。本研究旨在评估eculizumab（Elizaria®）生物类似物长期治疗在阵发性睡眠性血红蛋白尿（PNH）患者中的安全性、免疫原性和疗效。方法。研究纳入了30名已完成先前临床试验的PNH患者。其中，25名患者继续接受生物类似物产品治疗，5名患者从原研产品Soliris转换为生物类似物。最长随访时间为104周，在此期间，研究产品以标准剂量接受了52次给药。结果。在整个研究过程中，与基线相比，无论先前治疗方案如何，乳酸脱氢酶（LDH）、血红蛋白、网织红细胞和PNH克隆水平均保持稳定（p > 0.05）。在不同访问期间，慢性肾脏病患者的数量以及接受供体红细胞和血小板输血的患者数量均无显著差异（p > 0.05）。在两名患者中报告了两种不良事件（6.6%）：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，抗药物抗体检测的频率无显著差异（p > 0.05）。结论。研究结果表明，生物类似物在PNH患者中的长期疗效和安全性得到证实。