Hematopoietic Stem Cell Transplantation for the Treatment of Autoimmune Neurological Diseases: An Update

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.

Automated summary

Over the past two decades, haematopoietic stem cell transplantation (HSCT) has been investigated as a potential treatment for autoimmune diseases, including neurological disorders, that do not respond to conventional therapies. Autologous HSCT (AHSCT) has been favored over allogeneic HSCT (allo-HSCT) due to its better safety profile. While multiple sclerosis (MS) is the most common neurological indication for AHSCT, evidence on the effectiveness of HSCT in other autoimmune neurological diseases is emerging, though with a more uncertain risk-benefit ratio. This article provides a brief overview of the rationale for using HSCT in neurological diseases, the experimental models that led to its clinical application, and discusses the use of HSCT in autoimmune diseases other than MS, including myasthenia gravis, myopathies, and stiff-person syndrome. It also summarizes ongoing and future studies on this topic.