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autologous stem cell transplants for SPMS

A board to discuss future MS therapies in early stage (Phase I or II) trials.

autologous stem cell transplants for SPMS

Postby dignan » Sat Oct 31, 2009 9:53 am

Not exactly a ground-breaking study, but at least it's an option for SPMS.


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

Bone Marrow Transplant. 2009 Oct 26. [Epub ahead of print]
Tappenden P, Saccardi R, Confavreux C, Sharrack B, Muraro PA, Mancardi GL, Kozak T, Farge-Bancel D, Madan J, Rafia R, Akehurst R, Snowden J.
Health Economics and Decision Science (HEDS), School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK.

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 pound3000 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.

http://www.ncbi.nlm.nih.gov/pubmed/19855441
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