Hospital for Children and Adolescents, Hospital St. Georg, Academic Teaching Hospital of the University of Leipzig, Leipzig, Germany.
ImmunoDeficiencyCenter Leipzig (IDCL), Hospital St. Georg, Academic Teaching Hospital of the University of Leipzig, Leipzig, Germany.
Adv Ther. 2023 Dec;40(12):5168-5187. doi: 10.1007/s12325-023-02649-0. Epub 2023 Sep 26.
The CORE study aimed to provide a detailed understanding of real-world immune globulin subcutaneous (human) 20% solution (Ig20Gly) utilization in patients with primary immunodeficiency diseases (PIDs) in Germany and Switzerland.
Patients with PIDs receiving a stable dose of any subcutaneous immunoglobulin for ≥ 3 months before enrollment were eligible for this multicenter (n = 5), phase 4, non-interventional, prospective, longitudinal cohort study. Besides baseline demographics and clinical characteristics, Ig20Gly utilization and safety data, and patient-reported outcomes (Life Quality Index/Treatment Satisfaction Questionnaire for Medication) were collected at baseline, 6 and 12 months. Statistical analysis was descriptive.
Overall, 36 patients provided data at baseline [69.4% female; mean age: 41.6 years (7-78 years)]. Totals of 23 and 26 patients attended 6- and 12-month visits, respectively; 16 attended all three visits. One patient withdrew consent before 6-month follow-up. Median maximum infusion rates of Ig20Gly at baseline, 6 months, and 12 months were 26.7, 24.5, and 40.0 mL/h, respectively (10-60 mL/h). Infusion and dosing parameters remained consistent across time points: patients used a median of two infusion sites, primarily the abdomen, and all patients used an infusion pump; all but one infused at home and most self-administered Ig20Gly (80.8-83.3%) at once-weekly intervals (69.2-73.9%). During follow-up, 10 adverse events were reported: none were rated serious, while 2 were considered probably related to Ig20Gly. Total patient-reported outcome scores remained high throughout the study.
The CORE study provides real-world evidence of the flexibility, feasibility, safety, and tolerability of Ig20Gly infusions, at mostly weekly intervals, over 1 year in patients with PIDs.
German Clinical Trials Register, DRKS00014562. Registered April 9, 2018, https://drks.de/search/en/trial/DRKS00014562.
CORE 研究旨在深入了解德国和瑞士原发性免疫缺陷病（PID）患者中免疫球蛋白皮下（人）20%溶液（Ig20Gly）的实际应用情况。
这项多中心（n=5）、4 期、非干预性、前瞻性、纵向队列研究纳入了在入组前至少稳定接受任何皮下免疫球蛋白治疗≥3 个月的 PID 患者。除了基线人口统计学和临床特征外，还在基线、6 个月和 12 个月时收集 Ig20Gly 的使用情况和安全性数据以及患者报告的结局（生活质量指数/药物治疗满意度问卷）。统计分析为描述性。
总体而言，36 名患者在基线时提供了数据（69.4%为女性；平均年龄：41.6 岁（7-78 岁））。分别有 23 名和 26 名患者参加了 6 个月和 12 个月的随访，16 名患者参加了所有 3 次随访。1 名患者在 6 个月随访前撤回了同意。基线、6 个月和 12 个月时 Ig20Gly 的最大输注率中位数分别为 26.7、24.5 和 40.0 mL/h（10-60 mL/h）。输注和给药参数在各时间点保持一致：患者使用中位数为 2 个输注部位，主要是腹部，所有患者均使用输注泵；除 1 名患者外，其余患者均在家中输注，大多数患者（80.8-83.3%）以每周 1 次的间隔自行给药。在随访期间，报告了 10 起不良事件：均非严重事件，其中 2 起被认为可能与 Ig20Gly 有关。整个研究过程中，患者报告的总结局评分一直很高。
CORE 研究提供了真实世界的证据，证明了 Ig20Gly 输注在 PID 患者中具有灵活性、可行性、安全性和耐受性，可在 1 年以上的时间内以每周大部分时间的间隔进行。
德国临床试验注册处，DRKS00014562。于 2018 年 4 月 9 日注册，https://drks.de/search/en/trial/DRKS00014562。
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