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Multiple Sclerosis

Autologous haematopoietic stem cell transplantation for secondary progressive multiple sclerosis: an exploratory cost-effectiveness analysis

Bone Marrow Transplantation volume 45, pages 1014–1021 (2010)Cite this article

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.

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Acknowledgements

This study was undertaken without commercial or research funding.

Author information

Authors and Affiliations

  1. Health Economics and Decision Science (HEDS), School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK

    P Tappenden, J Madan, R Rafia & R Akehurst

  2. Bone Marrow Transplant Unit, UO Ematologia, Policlinico Careggi, Florence, Italy

    R Saccardi

  3. H,

    C Confavreux

  4. ô,

    C Confavreux

  5. pital Neurologique Pierre Wertheimer, Hospices Civils de Lyon, Service de Neurologie A and EDMUS Coordinating Center, Lyon, France

    C Confavreux

  6. Department of Neurology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK

    B Sharrack

  7. Department of Cellular and Molecular Neuroscience, Division of Neuroscience and Mental Health, Faculty of Medicine, Imperial College, London, UK

    P A Muraro

  8. Department of Neurosciences, Ophtalmology and Genetics, University of Genova, Genova, Italy

    G L Mancardi

  9. Charles University Prague, Clinical Haematology, Prague, Czech Republic

    T Kozak

  10. H,

    D Farge-Bancel

  11. ô,

    D Farge-Bancel

  12. pital Saint-Louis, Service de M,

    D Farge-Bancel

  13. é,

    D Farge-Bancel

  14. decine Interne et Pathologie Vasculaire, Paris, France

    D Farge-Bancel

  15. Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK

    J Snowden

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  1. P Tappenden
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Correspondence to P Tappenden.

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Tappenden, P., Saccardi, R., Confavreux, C. et al. Autologous haematopoietic stem cell transplantation for secondary progressive multiple sclerosis: an exploratory cost-effectiveness analysis. Bone Marrow Transplant 45, 1014–1021 (2010). https://doi.org/10.1038/bmt.2009.305

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