Department of Immunology, Children's Memorial Health Institute, Warsaw, Poland.
Department of Pediatrics, Faculty of Medical Sciences in Zabrze, Medical University of Silesia in Katowice, Zabrze, Poland.
Expert Rev Clin Immunol. 2023 Jul-Dec;19(10):1281-1291. doi: 10.1080/1744666X.2023.2240514. Epub 2023 Jul 25.
Subcutaneous administration of immunoglobulins is associated with fewer systemic adverse events and easier infusion compared to intravenous administration. Ig20Gly is a 20% immunoglobulin formulation effective and safe in patients with primary immune deficiency diseases (PIDDs). Real-world data are scarce, therefore our study aimed to examine the real-life treatment regimen and clinical outcomes of Ig20Gly in Polish children with PIDDs.
We retrospectively analyzed the medical documentation of 75 pediatric patients aged 0-17 years (mean 9.9) who received Ig20Gly (Cuvitru®; Baxalta US, Inc.; part of Takeda, MA, U.S.A.).
The median exposure to treatment of the study population was 22.3 months. At the end of the study, 59 (78.7%) were still on Ig20Gly. The median monthly dose was 0.40 g/kg. The median treatment interval was 7.7 days. Most patients (96%) used one infusion site. The median infusion rate increased with patient age. The median IgG level in the study population, 8.0 g/L, was stable. There was one case of serious bacterial infection.
This is the largest, long-term real-world study to date on the treatment patterns of Ig20Gly in pediatric patients with PIDDs. The results of this study support the feasibility and tolerability of Ig20Gly usage in PIDD patients across the pediatric age spectrum.
The trial is registered at ClinicalTrials.gov (NCT04636502).
与静脉注射相比，皮下注射免疫球蛋白与较少的全身不良反应和更容易输注相关。Ig20Gly 是一种 20%的免疫球蛋白制剂，在原发性免疫缺陷病（PIDD）患者中有效且安全。真实世界的数据很少，因此我们的研究旨在检查 Ig20Gly 在波兰 PID 儿童中的真实治疗方案和临床结果。
我们回顾性分析了 75 名 0-17 岁（平均 9.9 岁）接受 Ig20Gly（Cuvitru®；Baxalta US，Inc.；隶属于 Takeda，MA，美国）治疗的儿科患者的病历。
研究人群的中位治疗暴露时间为 22.3 个月。研究结束时，59 名（78.7%）仍在接受 Ig20Gly 治疗。中位月剂量为 0.40 g/kg。中位治疗间隔为 7.7 天。大多数患者（96%）使用一个输注部位。输注率随患者年龄的增加而增加。研究人群的中位 IgG 水平为 8.0 g/L，保持稳定。有 1 例严重细菌感染。
这是迄今为止关于 Ig20Gly 在 PID 儿科患者中的治疗模式的最大、长期的真实世界研究。该研究结果支持 Ig20Gly 在儿科 PID 患者中的使用具有可行性和耐受性。
该试验在 ClinicalTrials.gov 上注册（NCT04636502）。
