Nine-year follow up after hematopoietic stem cell transplantation in five multiple sclerosis patients

ABSTRACT Multiple sclerosis (MS) is an immune-mediated disease of the central nervous system. Its treatment has focused on inflammation control as early as possible to avoid disability. Autologous hematopoietic stem cell transplantation (AHSCT) has been used for treating MS since 1996, with recent decisive results regarding benefits in long-term efficacy. Five patients followed up at an MS center in Belo Horizonte, Brazil, who had relapsing-remitting MS with high disease activity, underwent AHSCT between 2009 and 2011. They were evaluated clinically, with magnetic resonance imaging, and by the EDSS every six months after transplantation, up to July 2018. The patients were four women and one man, with ages ranging from 25-50 years, and time since disease onset ranging from 4-17 years at the time of the procedure. Four patients improved, one patient was stabilized, and all patients were free of disease activity after 5-9 years. Through improving patient selection and decreasing the time from disease onset, AHSCT could stop epitope spreading and disease progression. Despite multiple other therapeutic choices being approved for relapsing-remitting MS, AHSCT continues to be a treatment to consider for aggressive MS disease.

摘要：多发性硬化症（multiple sclerosis, MS）是一类中枢神经系统免疫介导性疾病，其临床治疗的核心目标为尽早控制炎症，以避免残疾发生。自体造血干细胞移植（autologous hematopoietic stem cell transplantation, AHSCT）自1996年起便应用于MS的治疗，近年相关研究已证实其在长期疗效获益方面的关键性结论。
本研究纳入巴西贝洛奥里藏特某MS中心随访的5例复发缓解型MS且疾病活动度较高的患者，上述受试者于2009年至2011年间接受了AHSCT治疗。在移植后至2018年7月的随访周期内，研究人员每6个月对患者开展临床评估、磁共振成像（magnetic resonance imaging）检查，并采用扩展残疾状况评分量表（Expanded Disability Status Scale, EDSS）进行评分。
本次研究的5例患者中，4例为女性、1例为男性，年龄介于25至50岁之间，手术实施时距疾病首发的时长为4至17年。随访至术后5~9年时，4例患者病情获得改善，1例患者病情保持稳定，所有患者均未出现疾病活动迹象。
通过优化患者筛选标准并缩短疾病发病至接受治疗的间隔时间，AHSCT可阻断表位扩散并延缓疾病进展。尽管目前已有多种其他治疗方案获批用于复发缓解型MS，但AHSCT仍是侵袭性MS患者的重要可选治疗策略。