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Autologous haematopoietic stem cell transplantation for secondary progressive multiple sclerosis : an exploratory cost-effectiveness analysis
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Tappenden, P., Saccardi, R., Confavreux, C., Sharrack, B., Muraro, P. A., Mancardi, G. L., Kozak, T., Farge-Bancel, D., Madan, Jason, Rafia, R., Akehurst, R. and Snowden, J. (2010) Autologous haematopoietic stem cell transplantation for secondary progressive multiple sclerosis : an exploratory cost-effectiveness analysis. Bone Marrow Transplantation, 45 (6). 1014- 1021. doi:10.1038/bmt.2009.305 ISSN 0268-3369 .
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Official URL: https://doi.org/10.1038/bmt.2009.305
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.
| Item Type: | Journal Article | ||||||||
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| Divisions: | Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Clinical Trials Unit Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Health Sciences Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School |
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| Journal or Publication Title: | Bone Marrow Transplantation | ||||||||
| Publisher: | Nature Publishing Group | ||||||||
| ISSN: | 0268-3369 | ||||||||
| Official Date: | 26 October 2010 | ||||||||
| Dates: |
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| Volume: | 45 | ||||||||
| Number: | 6 | ||||||||
| Page Range: | 1014- 1021 | ||||||||
| DOI: | 10.1038/bmt.2009.305 | ||||||||
| Status: | Peer Reviewed | ||||||||
| Publication Status: | Published | ||||||||
| Access rights to Published version: | Restricted or Subscription Access | ||||||||
| Date of first compliant deposit: | 18 July 2018 |
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