Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE)
Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE)
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DOI：
10.1038/s41409-019-0684-0
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发表时间：
2020-02-01
影响因子：
4.8
通讯作者：
Zaccara, Eleanora
中科院分区：
文献类型：
--
作者：
Sharrack, Basil;Saccardi, Riccardo;Zaccara, Eleanora
这些更新的EBMT指南回顾了造血干细胞移植（HSCT）治疗多发性硬化症（MS）和其他免疫介导的神经系统疾病的临床证据、登记活动和作用机制，并为患者选择、移植技术、随访和未来发展提供了建议。自体造血干细胞移植（aHSCT）已用于多发性硬化症治疗超过20年，目前是欧洲该治疗增长最快的适应症，越来越多的证据支持其用于对疾病修饰治疗无效的高活性复发缓解型多发性硬化症。aHSCT可能在治疗具有显著炎症成分的进行性多发性硬化症和其他免疫介导的神经系统疾病中具有潜在作用，包括慢性炎症性脱髓鞘性多发性神经病、视神经脊髓炎、重症肌无力和僵硬者综合征。只有在潜在风险合理的情况下才应考虑同种异体造血干细胞移植。与其他免疫调节治疗相比，造血干细胞移植具有更大的短期风险，需要移植医生和在经认证的造血干细胞移植中心治疗之前、期间和之后对这些神经系统疾病有特殊兴趣的神经科医生之间的密切专业合作。其他针对这些疾病的实验性细胞疗法尚处于发展阶段，患者只能在临床试验中接受治疗。
These updated EBMT guidelines review the clinical evidence, registry activity and mechanisms of action of haematopoietic stem cell transplantation (HSCT) in multiple sclerosis (MS) and other immune-mediated neurological diseases and provide recommendations for patient selection, transplant technique, follow-up and future development. The major focus is on autologous HSCT (aHSCT), used in MS for over two decades and currently the fastest growing indication for this treatment in Europe, with increasing evidence to support its use in highly active relapsing remitting MS failing to respond to disease modifying therapies. aHSCT may have a potential role in the treatment of the progressive forms of MS with a significant inflammatory component and other immune-mediated neurological diseases, including chronic inflammatory demyelinating polyneuropathy, neuromyelitis optica, myasthenia gravis and stiff person syndrome. Allogeneic HSCT should only be considered where potential risks are justified. Compared with other immunomodulatory treatments, HSCT is associated with greater short-term risks and requires close interspeciality collaboration between transplant physicians and neurologists with a special interest in these neurological conditions before, during and after treatment in accredited HSCT centres. Other experimental cell therapies are developmental for these diseases and patients should only be treated on clinical trials.