Hematopoietic stem cell transplantation for multiple sclerosis.

Over the past decades, several effective disease-modifying therapies have been approved for the treatment of multiple sclerosis (MS); however, achieving long-term disease remission remains challenging, particularly for patients with aggressive forms of MS. Intense immunosuppression followed by hematopoietic stem cell transplantation (HSCT) has been increasingly explored as a treatment strategy for aggressive MS. To date, more than 1800 MS patients have undergone HSCT worldwide. In this chapter, we provide a brief overview of the HSCT procedure, with a special focus on the unique considerations for transplanting MS patients (such as fertility preservation, prior therapy washout, and posttransplant monitoring). We also discuss the main evidence of efficacy and safety of the procedure in this context, as well as present preliminary data on the impact of HSCT on cerebrospinal fluid findings, magnetic resonance imaging metrics, and novel serum biomarkers of neurodegeneration and demyelination. In addition, we provide recommendations for patient selection from international guidelines.

Introduction to HSCT for neurologic diseases.

The use of hematopoietic stem cell transplantation (HSCT) as a treatment option for severe autoimmune diseases originated from animal studies, with promising results in human patients reported through clinical responses seen in individuals who underwent HSCT for other reasons. Currently, over 3800 HSCT procedures have been performed specifically for autoimmune disorders and the procedure has become a standard of care for conditions such as multiple sclerosis and systemic sclerosis. Despite the growing interest among the neurology community in using HSCT to treat refractory autoimmune disorders, several obstacles must still be overcome for the procedure to become widely accepted. These include increasing the safety of the procedure, determining the most effective conditioning regimen, fostering collaboration between neurology and hematology teams, and providing robust phase III study data to demonstrate the superiority of HSCT over standard immunotherapy. This Handbook aims to provide a valuable resource for all those involved in the care of patients undergoing HSCT, and we hope it will contribute to the efforts to find the correct placement of HSCT in the therapeutic strategy of the treatment of autoimmune disorders of the nervous system.