Long-Term Safety of Facilitated Subcutaneous Immunoglobulin 10% Treatment in US Clinical Practice in Patients with Primary Immunodeficiency Diseases: Results from a Post-Authorization Safety Study.

IF 7.2 2区 医学 Q1 IMMUNOLOGY
Arye Rubinstein, Mohsen Mabudian, Donald McNeil, Niraj C Patel, Richard L Wasserman, Sudhir Gupta, Paz Carrasco, Jie Chen, Enrique Garcia, Andras Nagy, Leman Yel
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引用次数: 0

Abstract

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.

Abstract Image

在美国临床实践中对原发性免疫缺陷病患者进行 10%促进性皮下免疫球蛋白治疗的长期安全性:授权后安全性研究的结果。
10% 促效皮下免疫球蛋白(fSCIG)是一种免疫球蛋白替代疗法,它利用重组人透明质酸酶(rHuPH20)增强免疫球蛋白的分散和吸收,从而延长了与静脉注射免疫球蛋白类似的治疗间隔(最多每月一次)。这项前瞻性、非干预性、开放标签、多中心、授权后安全性研究(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。年龄≥16岁、处方为和/或已开始接受fSCIG 10%治疗的PID患者被纳入研究。共有 253 名患者入选并纳入研究(完整分析集)。参与者接受 fSCIG 10% 治疗的中位数(四分位数间距)为 10.0(3.5-11.8)个月,大多数患者每 4 周输注一次(54.4% [1197/2201 次输注]),在家输注(62.6% [1395/2230 次输注])。总体而言,98.5%的输液未因不良事件(AEs)而降低输液率、中断或中止。52名患者(20.6%,284例)出现了与治疗相关的非严重不良反应。两名患者(0.8%)各出现了一次与治疗相关的严重不良事件(无菌性脑膜炎和深静脉血栓)。rHuPH20抗体的产生并不常见;14/196名患者(7.1%)的结合抗体检测呈阳性(滴度≥1:160),但未检测到中和抗体。抗 rHuPH20 抗体阳性与治疗相关的严重或非严重急性症状之间没有关系。在美国的临床实践中,PID 患者对长期、反复自行给药 fSCIG 10% 的耐受性良好。
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来源期刊
CiteScore
12.20
自引率
9.90%
发文量
218
审稿时长
2 months
期刊介绍: The Journal of Clinical Immunology publishes impactful papers in the realm of human immunology, delving into the diagnosis, pathogenesis, prognosis, or treatment of human diseases. The journal places particular emphasis on primary immunodeficiencies and related diseases, encompassing inborn errors of immunity in a broad sense, their underlying genotypes, and diverse phenotypes. These phenotypes include infection, malignancy, allergy, auto-inflammation, and autoimmunity. We welcome a broad spectrum of studies in this domain, spanning genetic discovery, clinical description, immunologic assessment, diagnostic approaches, prognosis evaluation, and treatment interventions. Case reports are considered if they are genuinely original and accompanied by a concise review of the relevant medical literature, illustrating how the novel case study advances the field. The instructions to authors provide detailed guidance on the four categories of papers accepted by the journal.
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