Muraro Paolo A, Mariottini Alice, Greco Raffaella, Burman Joachim, Iacobaeus Ellen, Inglese Matilde, Snowden John A, Alexander Tobias, Amato Maria Pia, Bø Lars, Boffa Giacomo, Ciccarelli Olga, Cohen Jeffrey A, Derfuss Tobias, Farge Dominique, Freedman Mark S, Gaughan Maria, Heesen Christoph, Kazmi Majid, Kirzigov Kirill, Ljungman Per, Mancardi Gianluigi, Martin Roland, Mehra Varun, Moiola Lucia, Saccardi Riccardo, Tintoré Mar, Stankoff Bruno, Sharrack Basil
Department of Brain Sciences, Faculty of Medicine, Imperial College, London, UK.
Department of Neurosciences, Psychology, Drug Research and Child Health (NEUROFARBA), University of Florence, Florence, Italy.
Nat Rev Neurol. 2025 Mar;21(3):140-158. doi: 10.1038/s41582-024-01050-x. Epub 2025 Jan 15.
Autologous haematopoietic stem cell transplantation (AHSCT) is a treatment option for relapsing forms of multiple sclerosis (MS) that are refractory to disease-modifying therapy (DMT). AHSCT after failure of high-efficacy DMT in aggressive forms of relapsing-remitting MS is a generally accepted indication, yet the optimal placement of this approach in the treatment sequence is not universally agreed upon. Uncertainties also remain with respect to other indications, such as in rapidly evolving, severe, treatment-naive MS, progressive MS, and neuromyelitis optica spectrum disorder (NMOSD). Furthermore, treatment and monitoring protocols, rehabilitation and other supportive care before and after AHSCT need to be optimized. To address these issues, we convened a European Committee for Treatment and Research in Multiple Sclerosis Focused Workshop in partnership with the European Society for Blood and Marrow Transplantation Autoimmune Diseases Working Party, in which evidence and key questions were presented and discussed by experts in these diseases and in AHSCT. Based on the workshop output and subsequent written interactions, this Consensus Statement provides practical guidance and recommendations on the use of AHSCT in MS and NMOSD. Recommendations are based on the available evidence, or on consensus when evidence was insufficient. We summarize the key evidence, report the final recommendations, and identify areas for further research.
自体造血干细胞移植（AHSCT）是复发型多发性硬化症（MS）的一种治疗选择，这些复发型MS对疾病修饰治疗（DMT）无效。对于侵袭性复发缓解型MS患者，在高效DMT治疗失败后进行AHSCT是普遍认可的适应症，但这种治疗方法在治疗序列中的最佳位置尚未得到普遍认同。对于其他适应症，如快速进展型、严重型、未经治疗的MS、进展型MS和视神经脊髓炎谱系障碍（NMOSD），仍存在不确定性。此外，AHSCT前后的治疗和监测方案、康复及其他支持性护理都需要优化。为了解决这些问题，我们与欧洲血液和骨髓移植学会自身免疫性疾病工作组合作，召开了一次欧洲多发性硬化症治疗和研究委员会重点研讨会，由这些疾病及AHSCT领域的专家介绍并讨论了相关证据和关键问题。基于研讨会成果及后续书面交流，本共识声明就AHSCT在MS和NMOSD中的应用提供了实用指南和建议。建议基于现有证据，证据不足时则基于共识得出。我们总结了关键证据，报告了最终建议，并确定了需要进一步研究的领域。
