Autologous Stem Cell Transplantation for Multiple Sclerosis

Résumé fourni par la source

• Autologous stem cell transplantation has demonstrated efficacy in controlling symptoms and reducing relapses in patients with multiple sclerosis, particularly in relapsing–remitting multiple sclerosis. • Optimal outcomes of autologous stem cell transplantation are achieved when it is performed early in the disease course, following failure of standard disease-modifying therapies in relapsing–remitting multiple sclerosis. • Current evidence supports the use of intermediate- to low-intensity conditioning regimens, including BEAM/ATG or Cy/ATG. • Investigational approaches involving CAR T-cell therapy and bispecific antibodies are currently being explored as novel treatments for patients with multiple sclerosis. Multiple sclerosis (MS) is an autoimmune, inflammatory, and neurodegenerative disorder of the central nervous system, likely generated by an EBV infection with auto-antibodies targeting myelin antigens primarily through molecular mimicry. MS may have a relapsing-remitting (RRMS) course or can be primary progressive (PPMS). A proportion of patients may evolve to secondary progressive (SPMS) disease, with progressive debilitation. While disease-modifying therapies (DMTs) have remained the mainstay for treatment of MS, many patients continue to experience disease activity and increased debilitation on these treatments. Autologous hematopoietic stem cell transplantation (AHSCT), although much less utilized, has emerged as an effective therapeutic option for DMT-resistant RRMS, offering sustained remission in most patients with RRMS, and preventing further debilitation with improvement in neurological outcomes. AHSCT has had mixed results in patients with progressive disease. Overall, studies indicate that AHSCT can achieve higher rates of no evidence of disease activity (NEDA) compared with DMTs, a lower annualized relapse rate, and potential slowing of progressive disease with very low treatment-related mortality. This review summarizes the current evidence for the role of AHSCT in the treatment of this disease, with a focus on timing for transplantation, transplant outcomes and conditioning regimens used prior to transplantation. Further research is needed to define optimal patient selection, optimal conditioning regimens used prior to transplantation and eventually compare outcomes with chimeric antigen receptor T-cell therapy —a new form of targeted B-cell therapy —if it proves to be similarly effective in treating this disease.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.