Abstract
Objective
To estimate differences in treatment costs and health outcomes between non-myeloablative
hematopoietic stem cell transplantation (HSCT) and disease-modifying therapies (DMTs)
for the treatment of relapsing-remitting multiple sclerosis (RRMS).
Methods
We collected data on costs and reimbursements for patients who underwent HSCT for
RRMS at Northwestern Memorial Hospital in Chicago (USA) between January 2017 and January
2019. The costs of HSCT were compared against those for DMTs in the United States,
obtained from the literature. We also conducted a literature review to interpret the
cost comparisons in terms of disease control and patients’ wellbeing defined as no
evidence of disease activity (NEDA), neurologic disability by the Expanded Disability
Status Scale (EDSS), and quality of life by the short form SF-36, respectively.
Results
Outside of the data, herein, no other studies on cost of HSCT for RRMS were found
in the literature. HSCT mean total costs, based on our own hospital, were $85,184
(range $70,635 to $120,260). Mean revenue collected was $95,268 (range $16,544 to
$173,204). In comparison, according to the literature, 2019 DMT costs in the USA ranged
from $80,000 to $100,000 per year per patient. Compared to DMTs, studies of HSCT reported
greater improvement in no evidence of disease activity, disability, and quality of
life.
Limitations
Costs of HSCT would be expected to vary by conditioning regimen utilized, patient
selection, center experience, and regional variation. No cost data on other HSCT regimens
or on the three most recently licensed DMTs, alemtuzumab, ocrelizumab, and cladribine,
are available. Randomized trials for cost comparisons are missing and variations in
HSCT designs, populations, and methodology preclude more precise cost estimates.
Conclusion
Costs of non-myeloablative HSCT after which DMTs are indefinitely discontinued, are
approximately the same cost as those for one year of prescription DMTs. Since DMTs
assessed in this analysis are given on an ongoing basis, whilst HSCT is not, HSCT
is expected to produce long-term cost-savings. When considered alongside the available
clinical evidence, which suggests that HSCT may generate more health gains than DMTs,
HSCT is likely to represent a cost-effective use of resources. Model-based health
economic analyses are required to substantiate this conclusion.
Keywords
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Article info
Publication history
Published online: July 16, 2020
Accepted:
July 15,
2020
Received in revised form:
June 23,
2020
Received:
May 5,
2020
Identification
Copyright
© 2020 Elsevier B.V. All rights reserved.
