Outcomes and Cost-Effectiveness of Autologous Hematopoietic Cell Transplant for Multiple Sclerosis
Purpose of review
This review presents a critical appraisal of the use of autologous hematopoietic cell transplant (AHCT) for the treatment of multiple sclerosis. We present the reader with a brief review on the AHCT procedure, its immunomodulatory mechanism of action in MS, the most recent evidence in support of its use in patients with relapsing-remitting multiple sclerosis (RRMS), as well as its cost considerations.
The first meta-analysis of clinical trials of AHCT for patients with MS demonstrated durable 5-year progression-free survival rates and low treatment-related mortality. Recently, the first randomized controlled phase III clinical trial demonstrated AHCT to be superior to best available therapy for a subset of patients with RRMS. This led to the American society for transplant and cellular therapies (ASTCT) to recommend AHCT “for patients with relapsing forms of MS who have prognostic factors that indicate a high risk of future disability.”
AHCT should be considered for patients with RRMS with evidence of clinical activity who have failed 2 lines of therapy or at least one highly active disease-modifying therapy.
KeywordsMultiple sclerosis Autologous stem cell transplant Outcomes Cost-effectiveness
Drs. Dunn-Pirio and Heyman co-first authored the manuscript, both conceiving the idea for work, and equally contributed to writing the manuscript. Drs. Kaufman and Kinkel conceived the idea for work, helped write the manuscript, provided critical feedback, edited, and contributed to the final manuscript.
Compliance with Ethical Standards
Conflict of Interest
Dan S. Kaufman reports grants and personal fees from Fate Therapeutics, for work unrelated to the topic of this manuscript. Revere P. Kinkel reports honararia for scientific consultation and/or educational programs sponsored by the company from Biogen, Genzyme/Sanofi and Genetech/Roche outside the submitted work.
Anastasie M. Dunn-Pirio and Benjamin M. Heyman each declare no potential conflicts of interest.
Human and Animal Rights and Informed Consent
This article does not contain any studies with human or animal subjects performed by any of the authors.
References and Recommended Reading
Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance
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