Melikyan Anait L, Protsenko Ekaterina A, Salogub Galina N, Bakirov Bulat A, Davydkin Igor L, Kovalik Vladislav V, Gefen Mariia L, Matvienko Yuliia D, Saparova Valeria B, Khokhlov Alexander L, Makarenko Igor E, Drai Roman V
National Research Center for Hematology Moscow Russian Federation.
R&D Center GEROPHARM St Petersburg Russian Federation.
EJHaem. 2025 Jul 24;6(4):e70105. doi: 10.1002/jha2.70105. eCollection 2025 Aug.
This was a randomized multicenter single-blinded active-controlled equivalence Phase III study evaluating the efficacy and safety of the biosimilar of romiplostim (GP40141) compared to the reference drug Nplate in patients with persistent or chronic immune thrombocytopenia (ITP).
The study included 136 adult patients randomized 1:1 to receive either the biosimilar or reference drug for 26 weeks. The primary endpoint was the proportion of patients achieving a platelet response (≥ 50 × 10/L) at Week 11. Key secondary endpoints included proportion of patients with durable platelet response, stable response in treatment-naive patients, incidence of bleeding, and rescue therapy usage.
The proportion of patients with platelet response after 10 weeks of therapy was 78% in the test group and 85% in the comparison group. The absolute difference in proportion between groups was -0.06 (95% CI [-0.196, 0.068] and it falls within the equivalence limits [-0.225, 0.225]). The study demonstrated equivalence in the primary endpoint, with no significant differences between the biosimilar and reference drug groups in durable responses rate, bleeding events, or safety profiles.
The results support the biosimilar romiplostim as an equivalent and comparable treatment option to the reference drug for patients with persistent or chronic ITP.
NCT06497036.
这是一项随机、多中心、单盲、活性对照的等效性III期研究，旨在评估罗米司亭生物类似药（GP40141）与参比药物Nplate相比，用于持续性或慢性免疫性血小板减少症（ITP）患者的疗效和安全性。
该研究纳入136例成年患者，按1:1随机分组，接受生物类似药或参比药物治疗26周。主要终点是第11周时达到血小板反应（≥50×10⁹/L）的患者比例。关键次要终点包括持久血小板反应患者的比例、初治患者的稳定反应、出血发生率和挽救治疗的使用情况。
治疗10周后，试验组血小板反应患者的比例为78%，对照组为85%。两组比例的绝对差值为-0.06（95%CI[-0.196, 0.068]），且落在等效性界限[-0.225, 0.225]内。该研究在主要终点上显示出等效性，生物类似药组和参比药物组在持久反应率、出血事件或安全性方面无显著差异。
结果支持罗米司亭生物类似药作为持续性或慢性ITP患者的一种等效且可比的治疗选择，可替代参比药物。
NCT06497036。
