西罗莫司对儿童复发-难治性自身免疫性细胞减少症既有效又安全:一项多中心研究。

IF 4.1 4区 医学 Q2 IMMUNOLOGY
Scandinavian Journal of Immunology Pub Date : 2024-08-01 Epub Date: 2024-05-13 DOI:10.1111/sji.13376
Sultan Okur Acar, Neryal Tahta, Işık Odaman Al, Melek Erdem, Salih Gözmen, Tuba Hilkay Karapınar, Burcu Kılınç, Tiraje Celkan, Serap Kirkiz, Ülker Koçak, Hale Ören, Ayşen Türedi Yıldırım, Esra Arslantaş, Aylin Canbolat Ayhan, Yeşim Oymak
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引用次数: 0

摘要

自身免疫性细胞减少症是一类以免疫介导的造血细胞系破坏为特征的异质性疾病。对于复发难治性免疫性细胞减少症,有效且耐受性良好的治疗方案十分有限。本研究旨在评估西罗莫司在儿科这一疾病群体中的疗效和安全性。研究招募了 2010 年 12 月至 2020 年 12 月期间使用西罗莫司并确诊为免疫细胞减少症的儿科年龄组患者,并在土耳其的六个中心进行了随访。在17名患者中,5人(29.4%)因自身免疫性溶血性贫血(AIHA)接受治疗,6人(35.2%)因免疫性血小板减少性紫癜(ITP)接受治疗,6人(35.2%)因埃文斯综合征(ES)接受治疗。平均反应时间为 2.7 个月(0-9 个月)。17名患者中有13名(76.4%)获得了完全应答(CR)和部分应答(PR),4名患者(23.5%)获得了无应答(NR)。在获得 CR 的 13 名患者中,有 3 人在随访中出现 NR,其中 2 人在使用小剂量类固醇和西罗莫司后病情得到缓解。因此,17例患者中有12例(70.5%)达到了总体反应率(ORR)。总之,西罗莫司可能是复发-难治性免疫细胞减少症儿科患者的一种有效而安全的选择。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Sirolimus is effective and safe in childhood relapsed-refractory autoimmune cytopenias: A multicentre study.

Autoimmune cytopenias are a heterogeneous group of disorders characterized by immune-mediated destruction of haematopoietic cell lines. Effective and well-tolerated treatment options for relapsed-refractory immune cytopenias are limited. In this study, the aim was to evaluate the efficacy and safety of sirolimus in this disease group within the paediatric age group. The study enrolled patients in the paediatric age group who used sirolimus with a diagnosis of immune cytopenia between December 2010 and December 2020, followed at six centres in Turkey. Of the 17 patients, five (29.4%) were treated for autoimmune haemolytic anaemia (AIHA), six (35.2%) for immune thrombocytopenic purpura (ITP) and six (35.2%) for Evans syndrome (ES). The mean response time was 2.7 months (range, 0-9 months). Complete response (CR) and partial response (PR) were obtained in 13 of 17 patients (76.4%) and nonresponse (NR) in four patients (23.5%). Among the 13 patients who achieved CR, three of them were NR in the follow-up and two of them had remission with low-dose steroid and sirolimus. Thus, overall response rate (ORR) was achieved in 12 of 17 patients (70.5%). In conclusion, sirolimus may be an effective and safe option in paediatric patients with relapsed-refractory immune cytopenia.

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来源期刊
CiteScore
7.70
自引率
5.40%
发文量
109
审稿时长
1 months
期刊介绍: This peer-reviewed international journal publishes original articles and reviews on all aspects of basic, translational and clinical immunology. The journal aims to provide high quality service to authors, and high quality articles for readers. The journal accepts for publication material from investigators all over the world, which makes a significant contribution to basic, translational and clinical immunology.
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