系统性硬化症患者自体造血干细胞移植的长期疗效:与利妥昔单抗和传统免疫抑制剂治疗患者的比较

IF 4.9 2区 医学 Q1 Medicine Arthritis Research & Therapy Pub Date : 2024-10-23 DOI:10.1186/s13075-024-03408-4
Nicoletta Del Papa, Silvia Cavalli, Andrea Rindone, Francesco Onida, Giorgia Saporiti, Antonina Minniti, Maria Rosa Pellico, Claudia Iannone, Giorgia Trignani, Nicoletta D’Angelo, Manuel Sette, Raffaella Greco, Claudio Vitali, Roberto Caporali
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引用次数: 0

摘要

自体造血干细胞移植(AHSCT)比传统的免疫抑制疗法(CIT)更能有效改善快速进展性弥漫性皮肤系统性硬化症(dcSSc)患者的预后。迄今为止,将 AHSCT 与利妥昔单抗(RTX)进行比较的数据仍然很少。本研究旨在回顾性比较 AHSCT 与 RTX 和 CIT 对 dcSSc 患者的疗效。研究将 35 例接受 AHSCT 治疗的 dcSSc 患者与分别接受 RTX 和 CIT 治疗的 29 例和 36 例匹配病例进行了比较。对患者进行了为期5年的随访,每年对选定的结果指标进行评估。总生存期、改良罗德南皮肤评分(mRSS)、肺功能测试(FVC和DLCO)以及修订后的EUSTAR活动指数(REAI)是评估疗效的结果指标。在延长生存期方面,AHSCT明显比RTX和CIT更有效,它能迅速降低mRSS和REAI,并在更长时间内维持肺功能测试的基线水平。在降低REAI、mRSS和挽救肺功能方面,RTX疗法也优于CIT疗法。与 RTX 和 CIT 相比,AHSCT 在延长快速进展的 dcSSc 患者的生存期和诱导长期缓解方面更为有效。
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Long-term outcome of autologous haematopoietic stem cell transplantation in patients with systemic sclerosis: a comparison with patients treated with rituximab and with traditional immunosuppressive agents
Autologous haematopoietic stem cell transplantation (AHSCT) is more effective than conventional immunosuppressive therapies (CIT) in improving the outcome of patients with rapidly progressive diffuse cutaneous systemic sclerosis (dcSSc). So far, there is still a paucity of data comparing AHSCT with rituximab (RTX). Aim of the study is to retrospectively compare, in patients with dcSSc, the effectiveness of AHSCT with that of RTX and CIT. Thirty-five dcSSc AHSCT-treated patients were compared with 29 and 36 matched cases treated with RTX and CIT, respectively. The patients were followed up for 5 years by assessing selected outcome measures every year. Overall survival, modified Rodnan skin score (mRSS), lung function tests (FVC and DLCO), and the revised EUSTAR Activity Index (REAI) were the outcome measures chosen to evaluate the therapy efficacy. AHSCT was significantly more effective than RTX and CIT in prolonging survival, inducing a rapid reduction of the mRSS and REAI and maintaining the baseline level of lung function tests for a longer time. RTX therapy was also superior to CIT in reducing REAI, mRSS and in saving lung function. AHSCT is more effective than both RTX and CIT in prolonging survival and inducing prolonged remission in patients with rapidly progressive dcSSc.
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来源期刊
CiteScore
8.60
自引率
2.00%
发文量
261
审稿时长
14 weeks
期刊介绍: Established in 1999, Arthritis Research and Therapy is an international, open access, peer-reviewed journal, publishing original articles in the area of musculoskeletal research and therapy as well as, reviews, commentaries and reports. A major focus of the journal is on the immunologic processes leading to inflammation, damage and repair as they relate to autoimmune rheumatic and musculoskeletal conditions, and which inform the translation of this knowledge into advances in clinical care. Original basic, translational and clinical research is considered for publication along with results of early and late phase therapeutic trials, especially as they pertain to the underpinning science that informs clinical observations in interventional studies.
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