Laisuan Wannada, Pisitkun Prapaporn, Ngamjanyaporn Pintip, Suangtamai Thanitta, Rotjanapan Porpon
Division of Allergy Immunology and Rheumatology, Department of Medicine, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
Division of Infectious Diseases, Department of Medicine, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
Open Forum Infect Dis. 2020 Jan 31;7(2):ofaa035. doi: 10.1093/ofid/ofaa035. eCollection 2020 Feb.
Adult-onset immunodeficiency associated with interferon-γ autoantibody (IGA) is an emerging disease. The majority of patients require both antimicrobial and immunosuppressive treatments. However, anti-CD20 therapy is not fully accessible in a resource-limited setting to date.
The objectives of this work were to study the efficacy of cyclophosphamide treatment and the role of laboratory biomarkers for disease progression monitoring.
A prospective pilot cohort study was conducted among patients with anti-interferon-γ autoantibodies (IGA) who had recurrent infections and required long-term antimicrobial therapy between 2015 and 2018. The patients were categorized into 2 groups: receipt of intravenous cyclophosphamide (IVCY) and receipt of anti-CD20 therapy (RTX). Clinical and laboratory data were determined.
A total of 17 IGA patients were enrolled. Prolonged fever was the most common manifestation, and the most common infection identified was nontuberculous mycobacterial infections. Both were found in 88.24% of all patients.After completion of IVCY, 9/11 patients achieved complete remission and tended to reach remission faster compared with individuals in the RTX group. The median duration from treatment initiation to remission (interquartile range) was 84 (42-154) days in the IVCY group and 99 (51-202) days in the RTX group. In remission patients, the biomarkers of interest had normalized after treatment, except interferon γ autoantibody titers. There were no differences in adverse events among the 2 groups.
IVCY may be considered as alternative therapy in this population, especially in resource-limited countries. A comparable clinical outcome to RTX may support its use on a larger scale. However, further study is encouraged.
与干扰素-γ自身抗体（IGA）相关的成人发病免疫缺陷是一种新出现的疾病。大多数患者需要抗菌和免疫抑制治疗。然而，迄今为止，在资源有限的环境中，抗CD20疗法尚无法完全普及。
本研究的目的是探讨环磷酰胺治疗的疗效以及实验室生物标志物在疾病进展监测中的作用。
对2015年至2018年间患有抗干扰素-γ自身抗体（IGA）、反复感染且需要长期抗菌治疗的患者进行了一项前瞻性试点队列研究。患者分为两组：接受静脉注射环磷酰胺（IVCY）组和接受抗CD20治疗（RTX）组。测定了临床和实验室数据。
共纳入17例IGA患者。长期发热是最常见的表现，最常见的感染是非结核分枝杆菌感染。在所有患者中，88.24%的患者同时出现这两种情况。完成IVCY治疗后，11例患者中有9例实现完全缓解，与RTX组相比，缓解速度更快。IVCY组从治疗开始到缓解的中位持续时间（四分位间距）为84（42-154）天，RTX组为99（51-202）天。在缓解的患者中，除干扰素γ自身抗体滴度外，相关生物标志物在治疗后已恢复正常。两组之间不良事件无差异。
在这一人群中，尤其是在资源有限的国家，IVCY可被视为替代疗法。与RTX相当的临床结果可能支持其更广泛的应用。然而，鼓励进一步研究。
