Immunopathology Department, National Research Center Institute of Immunology FMBA, 24 Kashirskoe Shosse, Moscow 115522, Russia.
Immunology Department, Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology & Immunology, 1 Samory Mashela Street, Moscow 117997, Russia.
Immunotherapy. 2020 Apr;12(5):299-309. doi: 10.2217/imt-2020-0012. Epub 2020 Mar 26.
To evaluate efficacy and safety of octanorm (16.5% subcutaneous immunoglobulin) in adult patients with primary immunodeficiencies. Primary immunodeficiencies patients (18-70 years) previously treated with intravenous immunoglobulin were included in this Phase III study. Octanorm was administered subcutaneously once weekly over 8 months. End points included infections, adverse events and quality of life. 25 patients (mean age 35.2 years, female 60.0%) were recruited, 24 completed the study. Mean dose of octanorm was 0.11 g/kg/week. No serious bacterial infections occurred. Three patients (12.0%) had an adverse event (mild) assessed as related to octanorm. Both the mental and physical summary 36-item Short Form Health Survey scores were improved. Octanorm is effective, safe and improves quality of life. NCT03988426.
评估 octanorm（16.5%皮下免疫球蛋白）在原发性免疫缺陷成年患者中的疗效和安全性。本 III 期研究纳入了先前接受过静脉免疫球蛋白治疗的原发性免疫缺陷患者（18-70 岁）。octanorm 每周皮下给药一次，持续 8 个月。终点包括感染、不良事件和生活质量。共招募了 25 名患者（平均年龄 35.2 岁，女性 60.0%），24 名患者完成了研究。octanorm 的平均剂量为 0.11 g/kg/周。无严重细菌感染发生。3 名患者（12.0%）发生了不良事件（轻度），评估与 octanorm 相关。精神和身体综合 36 项简短健康调查问卷评分均有改善。octanorm 有效、安全且可提高生活质量。NCT03988426。
