Authors' objectives: The aim of this report is to review the clinical effectiveness and cost-effectiveness of AHSCT for the treatment of MS. The report also reviews the evidence-based guidelines regarding the use of AHSCT for MS. This report supports a CADTH Early Assessment Bulletin on MS.

Authors' results and conclusions: Evidence from 2 randomized controlled trials and 4 retrospective studies with limited methodological quality suggests that treatment with autologous hematopoietic stem cell transplantation was associated with significant improvement in clinical outcomes (e.g., disease progression, clinical relapse), MRI outcomes, the composite outcome “No Evidence of Disease Activity,” and quality of life compared to disease-modifying therapies. Treatment with autologous hematopoietic stem cell transplantation was associated with no treatment-related mortality or life-threatening complications including progressive multifocal leukoencephalopathy. However, autologous hematopoietic stem cell transplantation was associated with expected short-term adverse events including febrile neutropenia, organ infections, sepsis, and viral reactivations; and long-term adverse events including the development of new autoimmune diseases, mainly thyroid disease. Both identified guidelines recommend the use of autologous hematopoietic stem cell transplantation as standard of care for the treatment of highly active relapsing-remitting multiple sclerosis patients refractory to disease-modifying therapies and suggest that the treatment may be appropriate for progressive forms of multiple sclerosis with an active inflammatory component. No cost-effectiveness studies were identified.

Project Status: Completed

URL for project: https://www.cadth.ca/autologous-hematopoietic-cell-transplant-patients-multiple-sclerosis

Year Published: 2021

URL for published report: https://www.cadth.ca/sites/default/files/pdf/htis/2021/RC1356%20Cell%20Transplant%20for%20MS%20Final.pdf

English language abstract: An English language summary is available

Publication Type: Rapid Review

Country: Canada

Organisation Name: Canadian Agency for Drugs and Technologies in Health

Contact Address: 600-865 Carling Avenue, Ottawa, ON K1S 5S8 Canada. Tel: +1 613 226 2553; Fax: +1 613 226 5392;

Contact Name: requests@cadth.ca

Contact Email: requests@cadth.ca

Copyright: <p>Canadian Agency for Drugs and Technologies in Health (CADTH)</p>