Department of Brain Sciences, Faculty of Medicine, Imperial College London, Burlington Danes Building, Du Cane Road, Hammersmith Hospital Campus, London, W12 0NN, UK.
Department of Neurosciences, Drug and Child Health, University of Florence, Florence, Italy.
BioDrugs. 2020 Jun;34(3):307-325. doi: 10.1007/s40259-020-00414-1.
Autologous haematopoietic stem cell transplantation (AHSCT) is a treatment option for aggressive forms of multiple sclerosis (MS) that has been derived from haematological indications and repurposed for treatment of refractory autoimmune diseases. In the present review, a search for clinical studies on AHSCT was performed on the PubMed website and ClinicalTrials.gov databases. Papers were selected according to the following criteria: text written in English language, publication date between 2014 and August 2019, and reports including more than five patients. Prospective randomised and uncontrolled trials and retrospective case series were reviewed to examine the safety and efficacy of the procedure. Treatment protocols, pathological data and economic aspects of AHSCT were also succinctly covered. Growing evidence suggests that long-term suppression of inflammatory activity with stabilization or improvement of disability can be achieved in a high proportion of properly selected patients. More sophisticated outcome measures recently adopted, including effect on brain atrophy and disease biomarkers, are giving further insight into the effectiveness of transplant. The risks of the procedure have decreased to levels that can be considered acceptable for treatment of individuals with aggressive forms of MS. Careful selection of patients with an expected good benefit/risk profile, which is maximal when AHSCT is performed in early phases of the disease, and the expertise of transplant centres are critical to the success of treatment. Higher efficacy of AHSCT than with conventional treatments has recently been demonstrated by one randomised trial and further evidence is awaited from ongoing and planned trials comparing AHSCT with the most effective disease-modifying therapeutic agents.
自体造血干细胞移植（AHSCT）是一种治疗侵袭性多发性硬化症（MS）的方法，最初源自血液学适应证，并被重新用于治疗难治性自身免疫性疾病。在本综述中，在 PubMed 网站和 ClinicalTrials.gov 数据库中对 AHSCT 的临床研究进行了搜索。根据以下标准选择论文：英文书写的文本、发表日期在 2014 年至 2019 年 8 月之间、报告中包含超过 5 名患者。对前瞻性随机对照试验和回顾性病例系列进行了审查，以检查该程序的安全性和疗效。还简要介绍了 AHSCT 的治疗方案、病理数据和经济方面。越来越多的证据表明，在适当选择的患者中，可实现长期抑制炎症活动，稳定或改善残疾。最近采用的更复杂的结局指标，包括对脑萎缩和疾病生物标志物的影响，进一步深入了解移植的有效性。该程序的风险已降低到可以被认为是治疗侵袭性多发性硬化症个体可接受的水平。仔细选择具有预期良好获益/风险比的患者，当 AHSCT 在疾病早期阶段进行时，获益/风险比最大，并且移植中心的专业知识对于治疗的成功至关重要。最近，一项随机试验证明了 AHSCT 的疗效优于常规治疗，正在进行和计划中的试验正在比较 AHSCT 与最有效的疾病修正治疗药物，以提供更多的证据。
