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- Title
Autologous haematopoietic stem cell transplantation for secondary progressive multiple sclerosis: an exploratory cost-effectiveness analysis.
- Authors
Tappenden, P.; Saccardi, R.; Confavreux, C.; Sharrack, B.; Muraro, P. A.; Mancardi, G. L.; Kozak, T.; Farge-Bancel, D.; Madan, J.; Rafia, R.; Akehurst, R.; Snowden, J.
- Abstract
Treatment options for secondary progressive multiple sclerosis (SPMS) are limited. Mitoxantrone is routinely used to stabilize disease progression; however, evolving evidence suggests clinical benefit from intensive treatment with autologous haematopoietic stem cell transplantation (HSCT). Given differences in cost and outcomes, preliminary cost-effectiveness studies are warranted if this approach is to be developed for more widespread application in SPMS. We developed a decision-analytic Markov model to explore the potential cost-effectiveness of autologous HSCT versus mitoxantrone in SPMS, using patient-level data from registry sources. The model evaluates the lifetime costs and health outcomes associated with disability progression and relapse. Sensitivity analyses were undertaken to examine the uncertainty surrounding cost-effectiveness outcomes. In the absence of randomised controlled trial (RCT) evidence, conditions for comparative analysis were not ideal. Under optimistic assumptions, HSCT is estimated to cost below £3000 per quality adjusted life year gained. However, when a strict 6-month sustained progression rule is adopted, HSCT may be less effective and more expensive than mitoxantrone. The model results were sensitive to reducing procedural costs and HSCT-related mortality. We conclude that HSCT could potentially achieve an acceptable level of cost-effectiveness. However, caution should be exercised as large, high-quality RCTs comparing HSCT versus mitoxantrone are necessary to validate these findings.
- Subjects
MULTIPLE sclerosis treatment; MULTIPLE sclerosis prevention; MITOXANTRONE hydrochloride; ANTINEOPLASTIC agents; HEMATOPOIETIC stem cells; BONE marrow cells
- Publication
Bone Marrow Transplantation, 2010, Vol 45, Issue 6, p1014
- ISSN
0268-3369
- Publication type
Article
- DOI
10.1038/bmt.2009.305