Ma Jingyao, Zhang Xiaoli, Zhao Libo, Wu Xiaoyan, Yao Yanhua, Liu Wei, Wang Xiaohuan, Ju Xiuli, Shi Xiaodong, Sun Lirong, Zheng Lili, Liu Shu, Qian Jun, Wu Runhui
Department of Hematology, National Center for Children's Health, Beijing Children's Hospital, Capital Medical University, Beijing, China.
Department of Pediatrics, The First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.
Br J Haematol. 2024 Dec;205(6):2403-2413. doi: 10.1111/bjh.19761. Epub 2024 Sep 12.
The efficacy and safety of recombinant human thrombopoietin (rhTPO) in children and adolescent patients with chronic primary immune thrombocytopenia (ITP) remains unclear. A multicentre, randomized, double-blind, placebo-controlled phase III trial was performed. Patients aged 6-17 years, diagnosed with ITP and resistant or relapsed to corticosteroid treatment were included. For the trial, part 1 was exploratory and part 2 was the main analysis, with part 1 determining whether part 2 was stratified by age. Patients in part 1 were treated with rhTPO (the 6- to 11-/12- to 17-year-old groups; 1:1). Patients in part 2 were randomized (3:1) to receive either rhTPO treatment or placebo. Patients received rhTPO or placebo at a dose of 300 U/kg once daily for up to 14 days. A total of 68 patients were included [part 1 (12 patients), part 2 (56 patients)]. The total response rate (TRR) in part 1 was 50.0% (95% CI: 21.09%-78.91%). For part 2, the TRR was 58.5% (95% CI: 42.11%-73.68%) and 13.3% (95% CI: 1.66%-40.46%) in the rhTPO and placebo groups (FAS) respectively. The difference in TRR between the rhTPO group and placebo group was 45.2% (95% CI: 22.33%-68.08%) and 44.6% (95% CI: 21.27%-67.85%) on the FAS and per-protocol set (PPS), respectively, which indicates the superiority of rhTPO treatment.
重组人血小板生成素（rhTPO）在儿童和青少年慢性原发性免疫性血小板减少症（ITP）患者中的疗效和安全性尚不清楚。开展了一项多中心、随机、双盲、安慰剂对照的III期试验。纳入年龄在6至17岁、诊断为ITP且对皮质类固醇治疗耐药或复发的患者。对于该试验，第1部分为探索性试验，第2部分为主要分析，第1部分确定第2部分是否按年龄分层。第1部分的患者接受rhTPO治疗（6至11/12至17岁组；1:1）。第2部分的患者随机分组（3:1）接受rhTPO治疗或安慰剂。患者接受rhTPO或安慰剂，剂量为300 U/kg，每日一次，最多14天。共纳入68例患者[第1部分（12例患者），第2部分（56例患者）]。第1部分的总缓解率（TRR）为50.0%（95%CI：21.09%-78.91%）。对于第2部分，rhTPO组和安慰剂组（全分析集）的TRR分别为58.5%（95%CI：42.11%-73.68%）和13.3%（95%CI：1.66%-40.46%）。rhTPO组和安慰剂组在全分析集和符合方案集（PPS）上TRR的差异分别为45.2%（95%CI：22.33%-68.08%）和44.6%（95%CI：21.27%-67.85%），这表明rhTPO治疗具有优越性。
