Mariottini Alice, Bulgarini Giovanni, Cornacchini Sara, Damato Valentina, Saccardi Riccardo, Massacesi Luca
Department of Neurosciences, Drug and Child Health, University of Florence, 50139 Florence, Italy.
Department of Neurology 2, Careggi University Hospital, 50134 Florence, Italy.
Bioengineering (Basel). 2023 Jan 29;10(2):176. doi: 10.3390/bioengineering10020176.
Over the last two decades, haematopoietic stem cell transplantation (HSCT) has been explored as a potential therapeutic strategy for autoimmune diseases refractory to conventional treatments, including neurological disorders. Although both autologous (AHSCT) and allogeneic HSCT (allo-HSCT) were investigated, AHSCT was preferentially developed due to a more favourable safety profile compared to allo-HSCT. Multiple sclerosis (MS) represents the most frequent neurological indication for AHSCT, but increasing evidence on the potential effectiveness of transplant in other autoimmune neurological diseases is emerging, although with a risk-benefit ratio overall more uncertain than in MS. In the present work, the rationale for the use of HSCT in neurological diseases and the experimental models that prompted its clinical application will be briefly covered. Case series and prospective studies exploring the use of HSCT in autoimmune diseases other than MS will be discussed, covering both frequent and rare neurological disorders such as myasthenia gravis, myopathies, and stiff-person syndrome. Finally, an updated summary of ongoing and future studies focusing on this issue will be provided.
在过去二十年中，造血干细胞移植（HSCT）已被探索作为对包括神经系统疾病在内的传统治疗难治的自身免疫性疾病的一种潜在治疗策略。尽管对自体造血干细胞移植（AHSCT）和异基因造血干细胞移植（allo-HSCT）都进行了研究，但由于与allo-HSCT相比安全性更高，AHSCT得到了优先发展。多发性硬化症（MS）是AHSCT最常见的神经学适应症，但越来越多的证据表明移植在其他自身免疫性神经疾病中的潜在有效性正在显现，尽管总体风险效益比比MS更不确定。在本工作中，将简要介绍在神经疾病中使用HSCT的基本原理以及促使其临床应用的实验模型。将讨论探索HSCT在MS以外的自身免疫性疾病中的应用的病例系列和前瞻性研究，涵盖重症肌无力、肌病和僵人综合征等常见和罕见的神经疾病。最后，将提供专注于该问题的正在进行和未来研究的最新总结。
