Sheffield Institute for Translational Neuroscience, University of Sheffield, UK/Academic Department of Neurology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.
Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.
Mult Scler. 2021 Jul;27(8):1198-1204. doi: 10.1177/1352458520985238. Epub 2021 Feb 10.
Autologous haematopoietic stem cell transplantation (AHSCT) is an effective treatment for patients with multiple sclerosis (MS) who have highly active disease, despite the use of standard disease-modifying therapies (DMTs). However, the optimal time for offering AHSCT to patients with 'aggressive' MS is yet to be established.
The objective was to explore the safety and efficacy of AHSCT as a first-line DMT in patients with 'aggressive' MS.
All patients with 'aggressive' MS who received AHSCT as a first-line DMT in five European and North American centres were retrospectively evaluated.
Twenty patients were identified. The median interval between diagnosis and AHSCT was 5 (1-20) months. All had multiple poor prognostic markers with a median pre-transplant Expanded Disability Status Scale (EDSS) score of 5.0 (1.5-9.5). After a median follow-up of 30 (12-118) months, the median EDSS score improved to 2.0 (0-6.5, < 0.0001). No patient had further relapses. Three had residual magnetic resonance imaging (MRI) disease activities in the first 6 months post-transplant, but no further new or enhancing lesions were observed in subsequent scans.
AHSCT is safe and effective as a first-line DMT in inducing rapid and sustained remission in patients with 'aggressive' MS.
自体造血干细胞移植（AHSCT）是一种有效的治疗方法，适用于患有多发性硬化症（MS）且疾病活动度高的患者，尽管已经使用了标准的疾病修正疗法（DMT）。然而，为“侵袭性”MS 患者提供 AHSCT 的最佳时机尚未确定。
本研究旨在探讨 AHSCT 作为“侵袭性”MS 一线 DMT 的安全性和疗效。
回顾性评估了在五个欧洲和北美中心接受 AHSCT 作为一线 DMT 的所有“侵袭性”MS 患者。
共确定了 20 名患者。诊断与 AHSCT 之间的中位间隔为 5（1-20）个月。所有患者均具有多个不良预后标志物，移植前扩展残疾状况量表（EDSS）评分的中位数为 5.0（1.5-9.5）。在中位随访 30（12-118）个月后，EDSS 评分中位数改善至 2.0（0-6.5，<0.0001）。无患者进一步复发。3 名患者在移植后 6 个月内仍存在残留的磁共振成像（MRI）疾病活动，但随后的扫描中未观察到新的或增强的病变。
AHSCT 作为一线 DMT，在诱导“侵袭性”MS 患者快速和持续缓解方面是安全有效的。
