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自体造血干细胞移植与阿仑单抗治疗多发性硬化症患者的五年临床疗效比较分析

Comparative analysis of five-year clinical outcomes of autologous hematopoietic stem cell transplantation and alemtuzumab in multiple sclerosis patients.

作者信息

Manazoğlu Hazal Ceren, İşkan Gülce, Gündüz Tuncay, Emekli Ahmed Serkan, Akman Gülşen, Altunrende Burcu, Sucak Gülsan, Arat Mutlu, Özçelik Tülay, Beşışık Sevgi Kalayoğlu, Erden Sevda Öztürk, Kürtüncü Murat

机构信息

Department of Neurology, Istanbul Faculty of Medicine, Istanbul University, Istanbul, Türkiye; Department of Neurology, Umraniye Training and Research Hospital, Health Sciences University, Istanbul, Türkiye.

Department of Neurology, Istanbul Faculty of Medicine, Istanbul University, Istanbul, Türkiye.

出版信息

Mult Scler Relat Disord. 2025 Aug;100:106542. doi: 10.1016/j.msard.2025.106542. Epub 2025 May 24.

Abstract

INTRODUCTION

Alemtuzumab (ALZ) and autologous hematopoietic stem cell transplantation (aHSCT) are potent immune reconstitution therapies for patients with highly active multiple sclerosis (MS). This study aims to compare the five-year efficacy of aHSCT and ALZ in MS patients with high disease activity.

METHODS

This multicenter observational study evaluated MS patients treated with aHSCT and ALZ. Annual relapse rates (ARR), Expanded Disability Status Scale (EDSS) scores, and annual cumulative disease activity (NEDA-3) were compared between pre-treatment levels and outcomes at the five-year follow-up.

RESULTS

The study included 39 patients treated with ALZ and 18 who underwent aHSCT. The median ages at treatment initiation were 25 years (IQR: 15) for the ALZ group and 25 years (IQR: 14) for the aHSCT group, with median baseline EDSS scores of 6.5 (IQR: 1.5) and 6.8 (IQR: 1.0), respectively. The median follow-up durations were 1 year (IQR: 3) for ALZ-treated patients and 5 years (IQR: 6) for aHSCT-treated patients. Comparative analysis showed no significant differences between the groups in time to first relapse, EDSS worsening, or time to NEDA-3 loss over the five-year follow-up period.

CONCLUSION

This study is the first to compare the effectiveness of aHSCT and ALZ treatments in MS patients in Türkiye. The findings reveal that both treatments demonstrate comparable efficacy, particularly in the short-term follow-up period.

摘要

引言

阿仑单抗(ALZ)和自体造血干细胞移植(aHSCT)是用于高度活动性多发性硬化症(MS)患者的强效免疫重建疗法。本研究旨在比较aHSCT和ALZ对高疾病活动性MS患者的五年疗效。

方法

这项多中心观察性研究评估了接受aHSCT和ALZ治疗的MS患者。比较了治疗前水平与五年随访结果之间的年复发率(ARR)、扩展残疾状态量表(EDSS)评分和年累积疾病活动度(NEDA-3)。

结果

该研究纳入了39例接受ALZ治疗的患者和18例接受aHSCT治疗的患者。ALZ组治疗开始时的中位年龄为25岁(四分位间距:15),aHSCT组为25岁(四分位间距:14),基线EDSS评分中位数分别为6.5(四分位间距:1.5)和6.8(四分位间距:1.0)。接受ALZ治疗患者的中位随访时间为1年(四分位间距:3),接受aHSCT治疗患者的中位随访时间为5年(四分位间距:6)。比较分析显示,在五年随访期内,两组在首次复发时间、EDSS恶化或NEDA-3丧失时间方面无显著差异。

结论

本研究首次比较了aHSCT和ALZ治疗在土耳其MS患者中的有效性。研究结果表明,两种治疗方法疗效相当,尤其是在短期随访期。

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