Cai Huacong, Wang Jishi, Yan Zhenyu, Zhou Hu, Li Zhenyu, Yang Feng'e, Guo Pengxiang, Gao Da, Jin Jie, Zeng Yun, Wang Shujie
Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Affiliated Hospital of Guizhou Medical University, Guiyang, China.
Clin Exp Med. 2025 May 12;25(1):153. doi: 10.1007/s10238-025-01658-3.
A novel, highly purified 10% intravenous immunoglobulin (IVIG) formulation was evaluated for both therapeutic efficacy and safety profile in adult patients diagnosed with persistent or chronic primary immune thrombocytopenia (ITP). This phase III, multicenter, open-label, single-arm clinical trial enrolled Chinese adult patients diagnosed with persistent or chronic ITP presenting with baseline platelet counts below 30 × 10/L. Participants received intravenous administration of 10% IVIG at a standardized dosage of 1 g/kg/day for two consecutive days. The primary efficacy endpoint was defined as the proportion of subjects achieving both a platelet count elevation to ≥ 30 × 10/L and a minimum two-fold increase from baseline values within a 7-day post-treatment observation period following the first dose administration. Seventy-two patients were enrolled and sixty patients completed the study. 52 (72.2%; 95% CI: 60.4, 82.1) patients achieved platelet count ≥ 30 × 10/L and experienced a ≥ twofold increase from baseline within 7 days, and 52 (72.2%; 95% CI: 60.4, 82.1) patients achieved complete response (CR) or response (R) within 7 days. 64 patients (88.9%; 95% CI: 79.3, 95.1) achieved platelet count ≥ 50 × 10/L within 7 days with a median time of 3 days. 71 patients completed the ITP bleeding scale assessment after 7 days, showing a decrease of 0.6 ± 1.07 from baseline. A total of 66 patients (91.7%) reported treatment-emergent adverse events (TEAEs) during the study, and 37 patients (51.4%) reported adverse drug reactions (ADRs). The most prevalent ADRs with an incidence exceeding 5% included headache (n = 12, 16.7%), fever (n = 10, 13.9%), decreased white blood cell count (n = 5, 6.9%), and nausea (n = 5, 6.9%). The therapeutic regimen of 10% IVIG administered at a dosage of 1 g/kg/day for two consecutive days demonstrated both favorable safety profiles and clinical efficacy. These robust findings provide substantial evidence supporting the clinical application of this novel 10% IVIG formulation in the management of adult patients with ITP.
对一种新型、高度纯化的10%静脉注射免疫球蛋白（IVIG）制剂进行了评估，以确定其在诊断为持续性或慢性原发性免疫性血小板减少症（ITP）的成年患者中的治疗效果和安全性。这项III期、多中心、开放标签、单臂临床试验纳入了诊断为持续性或慢性ITP且基线血小板计数低于30×10⁹/L的中国成年患者。参与者连续两天接受标准化剂量为1 g/kg/天的10% IVIG静脉注射。主要疗效终点定义为在首次给药后的7天治疗后观察期内，血小板计数升高至≥30×10⁹/L且较基线值至少增加两倍的受试者比例。72名患者入组，60名患者完成了研究。52名（72.2%；95%置信区间：60.4，82.1）患者在7天内血小板计数≥30×10⁹/L且较基线值增加≥两倍，52名（72.2%；95%置信区间：60.4，82.1）患者在7天内达到完全缓解（CR）或缓解（R）。64名患者（88.9%；95%置信区间：79.3，95.1）在7天内血小板计数≥50×10⁹/L，中位时间为3天。71名患者在7天后完成了ITP出血量表评估，显示较基线值下降了0.6±1.07。共有66名患者（91.7%）在研究期间报告了治疗中出现的不良事件（TEAE），37名患者（51.4%）报告了药物不良反应（ADR）。发生率超过5%的最常见ADR包括头痛（n = 12，16.7%）、发热（n = 10，13.9%）、白细胞计数减少（n = 5，6.9%）和恶心（n = 5，6.9%）。连续两天以1 g/kg/天的剂量给予10% IVIG的治疗方案显示出良好的安全性和临床疗效。这些有力的研究结果为这种新型10% IVIG制剂在成年ITP患者管理中的临床应用提供了充分的证据。
