Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan.
Department of Pediatrics, Kurume University Hospital, Fukuoka, Japan.
Int J Hematol. 2021 Jun;113(6):921-929. doi: 10.1007/s12185-021-03106-w. Epub 2021 Mar 18.
Studies investigating the safety of IgPro10 (Privigen, CSL Behring, King of Prussia, PA, USA) in Japanese patients with primary immunodeficiency (PID) are lacking. This study evaluated safety and tolerability of IgPro10 in Japanese patients with PID. In this prospective, open-label, single-arm, registrational study for Japan, IgPro10 was administered intravenously at pre-study doses of 138-556 mg/kg body weight per 3-/4-weekly dosing cycle for up to 4 months. Frequency and intensity of adverse events (AEs), their relationship to IgPro10 and AE rate per infusion (AERI) were evaluated. Of 11 enrolled patients, 10 completed the study. The median (range) total duration of exposure was 16.14 (4.1-16.3) weeks. Eight patients reported 19 AEs, none severe (based on maximum severity), giving an AERI of 0.442. One AE was deemed related to IgPro10 treatment. Three patients experienced temporally associated AEs. No serious AEs or deaths were reported. Nine patients (90%) who completed the study tolerated flow rates of ≥ 8 mg/kg/min; 5 tolerated 12 mg/kg/min (7.2 mL/kg/h), translating into a threefold decrease in mean infusion time. IgPro10 was well tolerated at a flow rate of up to 12 mg/kg/min. Safety and tolerability findings were consistent with previously reported studies in non-Japanese patients with PID.
在原发性免疫缺陷 (PID) 日本患者中，有关 IgPro10（Privigen，CSL Behring，King of Prussia，PA，USA）安全性的研究尚缺乏。本研究评估了 IgPro10 在 PID 日本患者中的安全性和耐受性。在这项针对日本的前瞻性、开放标签、单臂、注册研究中，IgPro10 按研究前剂量在每 3/4 周治疗周期内静脉输注 138-556mg/kg 体重，最长达 4 个月。评估了不良事件 (AE) 的频率和强度、它们与 IgPro10 的关系以及每输注 (AERI) 的 AE 发生率。在纳入的 11 名患者中，有 10 名完成了研究。暴露的中位（范围）总持续时间为 16.14（4.1-16.3）周。8 名患者报告了 19 起 AE，均不严重（基于最大严重程度），AERI 为 0.442。1 起 AE 被认为与 IgPro10 治疗有关。3 名患者经历了与时间相关的 AE。未报告严重 AE 或死亡。完成研究的 9 名患者（90%）能够耐受≥8mg/kg/min 的流速；5 名患者耐受 12mg/kg/min（7.2mL/kg/h），这意味着平均输注时间缩短了三倍。IgPro10 在高达 12mg/kg/min 的流速下具有良好的耐受性。安全性和耐受性发现与以前在非日本 PID 患者中报告的研究一致。
