Department of Neuroscience, Uppsala University, Uppsala, Sweden Department of Neurology, Uppsala University Hospital, Uppsala, Sweden.
Neuroimmunology Unit, Department of Clinical Neuroscience, Karolinska Institute Solna, Center for Molecular Medicine, Stockholm, Sweden.
J Neurol Neurosurg Psychiatry. 2014 Oct;85(10):1116-21. doi: 10.1136/jnnp-2013-307207. Epub 2014 Feb 19.
Autologous haematopoietic stem cell transplantation (HSCT) is a viable option for treatment of aggressive multiple sclerosis (MS). No randomised controlled trial has been performed, and thus, experiences from systematic and sustained follow-up of treated patients constitute important information about safety and efficacy. In this observational study, we describe the characteristics and outcome of the Swedish patients treated with HSCT for MS.
Neurologists from the major hospitals in Sweden filled out a follow-up form with prospectively collected data. Fifty-two patients were identified in total; 48 were included in the study and evaluated for safety and side effects; 41 patients had at least 1 year of follow-up and were further analysed for clinical and radiological outcome. In this cohort, 34 patients (83%) had relapsing-remitting MS, and mean follow-up time was 47 months.
At 5 years, relapse-free survival was 87%; MRI event-free survival 85%; expanded disability status scale (EDSS) score progression-free survival 77%; and disease-free survival (no relapses, no new MRI lesions and no EDSS progression) 68%. Presence of gadolinium-enhancing lesions prior to HSCT was associated with a favourable outcome (disease-free survival 79% vs 46%, p=0.028). There was no mortality. The most common long-term side effects were herpes zoster reactivation (15%) and thyroid disease (8.4%).
HSCT is a very effective treatment of inflammatory active MS and can be performed with a high degree of safety at experienced centres.
自体造血干细胞移植（HSCT）是治疗侵袭性多发性硬化症（MS）的可行选择。尚未进行随机对照试验，因此，对接受治疗的患者进行系统和持续随访所获得的经验构成了关于安全性和疗效的重要信息。在这项观察性研究中，我们描述了瑞典接受 HSCT 治疗 MS 的患者的特征和结局。
瑞典主要医院的神经科医生填写了一份随访表格，其中包含前瞻性收集的数据。总共确定了 52 名患者；其中 48 名患者被纳入研究并评估安全性和副作用；41 名患者的随访时间至少为 1 年，进一步分析其临床和影像学结局。在这一组中，34 名患者（83%）患有复发缓解型 MS，平均随访时间为 47 个月。
在 5 年内，无复发存活率为 87%；无 MRI 事件存活率为 85%；扩展残疾状态量表（EDSS）评分无进展存活率为 77%；无疾病存活率（无复发、无新的 MRI 病变和 EDSS 无进展）为 68%。HSCT 前存在钆增强病变与良好结局相关（无疾病存活率为 79%比 46%，p=0.028）。无死亡病例。最常见的长期副作用是带状疱疹再激活（15%）和甲状腺疾病（8.4%）。
HSCT 是治疗活动性 MS 的一种非常有效的方法，在有经验的中心进行时具有高度的安全性。
