Albert Einstein College of Medicine and Montefiore Hospital, Bronx, NY, USA.
Allergy Immunology Medical Center, Redlands, CA, USA.
J Clin Immunol. 2024 Aug 19;44(8):181. doi: 10.1007/s10875-024-01769-8.
Facilitated subcutaneous immunoglobulin (fSCIG) 10% is an immunoglobulin replacement therapy that utilizes recombinant human hyaluronidase (rHuPH20) to enhance immunoglobulin dispersion and absorption, allowing for longer treatment intervals similar to intravenous immunoglobulin (up to once monthly). fSCIG 10% is indicated in the USA for treating adults and children aged ≥ 2 years with primary immunodeficiency diseases (PIDs). This prospective, non-interventional, open-label, multicenter, post-authorization safety study (NCT02593188) was conducted in the USA from November 2015 to October 2021 to assess the long-term safety of fSCIG 10% in routine clinical practice. Patients with PIDs aged ≥ 16 years who were prescribed and/or had started fSCIG 10% treatment were enrolled. In total, 253 patients were enrolled and included (full analysis set). Participants received fSCIG 10% treatment for a median (interquartile range) of 10.0 (3.5-11.8) months, with the majority of infusions administered every 4 weeks (54.4% [1197/2201 infusions]) and at home (62.6% [1395/2230 infusions]). Overall, 98.5% of infusions were administered without rate reduction, interruption, or discontinuation due to adverse events (AEs). Treatment-related, non-serious AEs were experienced by 52 patients (20.6%, 284 events). Two patients (0.8%) each experienced one treatment-related serious AE (aseptic meningitis and deep vein thrombosis). Development of antibodies against rHuPH20 was uncommon; 14/196 patients (7.1%) tested positive for binding antibodies (titer ≥ 1:160) with no neutralizing antibodies detected. There was no relationship between anti-rHuPH20 antibody positivity and the occurrence of treatment-related serious or non-serious AEs. Long-term, repeated self-administration of fSCIG 10% was well tolerated in US clinical practice by patients with PIDs.
皮下注射用人免疫球蛋白（fSCIG）10%是一种免疫球蛋白替代疗法，利用重组人透明质酸酶（rHuPH20）来增强免疫球蛋白的分散和吸收，从而允许更长的治疗间隔时间，类似于静脉注射免疫球蛋白（最长可达每月一次）。fSCIG 10%在美国被批准用于治疗 2 岁及以上的原发性免疫缺陷病（PID）成人和儿童。这项前瞻性、非干预性、开放性、多中心、上市后安全性研究（NCT02593188）于 2015 年 11 月至 2021 年 10 月在美国进行，旨在评估 fSCIG 10%在常规临床实践中的长期安全性。纳入符合 PID 诊断标准且年龄≥16 岁、接受过 fSCIG 10%处方和/或开始 fSCIG 10%治疗的患者。共有 253 名患者入组并纳入（全分析集）。参与者接受 fSCIG 10%治疗的中位数（四分位距）为 10.0（3.5-11.8）个月，大多数输注每 4 周进行 1 次（54.4%[1197/2201 次]），且在家中进行（62.6%[1395/2230 次]）。总体而言，由于不良事件（AE），98.5%的输注未进行剂量减少、中断或停止。52 名患者（20.6%，284 次事件）经历了与治疗相关的非严重 AE。两名患者（0.8%）各经历了 1 次与治疗相关的严重 AE（无菌性脑膜炎和深静脉血栓形成）。抗 rHuPH20 抗体的产生不常见；196 名患者中的 14 名（7.1%）检测到结合抗体（滴度≥1：160）阳性，但未检测到中和抗体。抗 rHuPH20 抗体阳性与治疗相关严重或非严重 AE 的发生之间无相关性。在 PID 患者的美国临床实践中，fSCIG 10%的长期、重复自我给药耐受性良好。
