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The European Journal of Health Economics
Published in: The European Journal of Health Economics 3/2020

Open Access 01-04-2020 | Stem Cell Transplantion | Original Paper

Health state utilities associated with treatment for transfusion-dependent β-thalassemia

Authors: Louis S. Matza, L. Clark Paramore, Katie D. Stewart, Hayley Karn, Minesh Jobanputra, Andrew C. Dietz

Published in: The European Journal of Health Economics | Issue 3/2020

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Abstract

Objectives

Transfusion-dependent β-thalassemia (TDT) is a genetic disease that affects production of red blood cells. Conventional treatment involves regular red blood cell transfusions and iron chelation, which has a substantial impact on quality of life. While potentially curative, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is associated with risk of complications, including graft-versus-host disease (GvHD). Gene addition therapy, a novel treatment approach, involves autologous transplantation of the patient’s own genetically modified hematopoietic stem cells. The purpose of this study was to estimate utilities associated with treatment approaches for TDT.

Methods

General population respondents in England valued eight health state vignettes (developed with clinician, patient, and parent input) in time trade-off interviews.

Results

A total of 207 participants completed interviews (49.8% female; mean age = 43.2 years). Mean (SD) utilities for the pre-transplant health states were 0.73 (0.25) with oral chelation and 0.63 (0.32) with subcutaneous chelation. Mean utilities for the transplant year were 0.62 (0.35) for gene addition therapy, 0.47 (0.39) for allo-HSCT, and 0.39 (0.39) for allo-HSCT with acute GvHD. Post-transplant utilities were 0.93 (0.15) for transfusion independent, 0.75 (0.25) for 60% transfusion reduction, and 0.51 (0.38) for chronic GvHD. Acute and chronic GvHD were associated with significant disutility (acute = − 0.09, p < 0.0001; chronic = − 0.42, p < 0.0001).

Conclusions

Utilities followed expected patterns, with logical differences between treatment options for TDT and substantially greater utility for transfusion independence than for ongoing treatment involving transfusion and chelation. These utilities may be useful in cost-utility models estimating the value of treatments for TDT.
Appendix
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Metadata
Title
Health state utilities associated with treatment for transfusion-dependent β-thalassemia
Authors
Louis S. Matza
L. Clark Paramore
Katie D. Stewart
Hayley Karn
Minesh Jobanputra
Andrew C. Dietz
Publication date
01-04-2020
Publisher
Springer Berlin Heidelberg
Published in
The European Journal of Health Economics / Issue 3/2020
Print ISSN: 1618-7598
Electronic ISSN: 1618-7601
DOI
https://doi.org/10.1007/s10198-019-01136-0

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