This prospective, multicenter, single‐arm, open‐label phase 3 study aimed to evaluate the efficacy and safety of IqYmune in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Patients received one induction dose of 2 g/kg and then seven maintenance doses of 1 g/kg at 3‐week intervals. The primary endpoint was the responder rate at the end of study (EOS), defined as an improvement of ≥1 point on the adjusted inflammatory neuropathy cause and treatment (INCAT) disability scale. The responder rate was compared with the responder rate of a historical placebo group (33.3%). Secondary endpoints included changes from baseline to EOS of adjusted INCAT disability score, grip strength, Medical Research Council (MRC) sum score, Rasch‐modified MRC sum score, Rasch‐built overall disability scale score and the clinical global impression. Forty‐two patients, including 23 Ig‐naïve and 19 Ig‐pre‐treated, were included in the efficacy set. The overall response rate at EOS was 76.2% (95% confidence interval [60.5%‐87.9%]). The superiority of IqYmune compared to the historical placebo control was demonstrated (P < .0001). The responder rate was numerically higher in Ig‐pre‐treated than in Ig‐naïve patients but confidence intervals were overlapping (84.2% [60.4%‐96.6%] vs 69.6% [47.1%‐86.8%]). All secondary endpoints confirmed this conclusion. The median time to response was 15 weeks [8.9‐19.1 weeks]. A total of 156 adverse events including five serious were considered related to IqYmune, 87.2% were mild. Neither hemolysis nor signs of renal or hepatic impairment were observed. These results demonstrate that IqYmune is an effective and well‐tolerated treatment in patients with CIDP.

中文翻译：

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IqYmune 在慢性炎症性脱髓鞘性多发性神经根神经病患者初始和维持治疗中的国际多中心有效性和安全性研究：PRISM 研究。

这项前瞻性、多中心、单臂、开放标签的 3 期研究旨在评估 IqYmune 在慢性炎症性脱髓鞘性多发性神经根神经病 (CIDP) 患者中的疗效和安全性。患者接受 1 次 2 g/kg 的诱导剂量，然后每 3 周接受 7 次 1 g/kg 的维持剂量。主要终点是研究结束时的反应率 (EOS)，定义为调整后炎症性神经病变病因和治疗 (INCAT) 残疾量表的改善≥1 分。将反应率与历史安慰剂组的反应率 (33.3%) 进行比较。次要终点包括调整后的 INCAT 残疾评分从基线到 EOS 的变化、握力、医学研究委员会 (MRC) 总分、Rasch 修正 MRC 总分、Rasch 建立的整体残疾量表评分和临床总体印象。42 名患者，包括 23 名 Ig 初治患者和 19 名 Ig 预处理患者，被纳入疗效组。EOS 的总体反应率为 76.2%（95% 置信区间 [60.5%-87.9%]）。证明了 IqYmune 与历史安慰剂对照相比的优越性（P  < .0001)。Ig 预处理患者的应答率在数值上高于 Ig 初治患者，但置信区间重叠（84.2% [60.4%-96.6%] vs 69.6% [47.1%-86.8%]）。所有次要终点都证实了这一结论。中位反应时间为 15 周 [8.9-19.1 周]。共有 156 起不良事件，其中 5 起严重被认为与 IqYmune 相关，87.2% 是轻微的。未观察到溶血或肾或肝损害迹象。这些结果表明 IqYmune 是一种治疗 CIDP 患者的有效且耐受性良好的治疗方法。