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.

中文翻译：

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系统性硬化症患者自体造血干细胞移植的长期结局：与利妥昔单抗治疗和传统免疫抑制剂治疗的比较


自体造血干细胞移植 （AHSCT） 在改善快速进展性弥漫性皮肤系统性硬化症 （dcSSc） 患者的预后方面比常规免疫抑制疗法 （CIT） 更有效。到目前为止，仍然缺乏比较 AHSCT 与利妥昔单抗 （RTX） 的数据。该研究的目的是回顾性比较 ahsct 与 RTX 和 CIT 患者的有效性。将 35 例 dcSSc AHSCT 治疗的患者分别与 29 例和 36 例接受 RTX 和 CIT 治疗的匹配病例进行了比较。通过每年评估选定的结局指标，对患者进行为期 5 年的随访。总生存期、改良 Rodnan 皮肤评分 （mRSS） 、肺功能测试 （FVC 和 DLCO） 和改良的 EUSTAR 活动指数 （REAI） 是评价治疗效果的结局指标。AHSCT 在延长生存期、诱导 mRSS 和 REAI 快速降低以及更长时间维持肺功能测试的基线水平方面明显优于 RTX 和 CIT。RTX 疗法在降低 REAI、mRSS 和保留肺功能方面也优于 CIT。AHSCT 在延长快速进展性 dcSSc 患者的生存期和诱导延长缓解方面比 RTX 和 CIT 更有效。