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

Louis S. Matza (), L. Clark Paramore (), Katie D. Stewart (), Hayley Karn (), Minesh Jobanputra () and Andrew C. Dietz ()
Additional contact information
Louis S. Matza: Patient-Centered Research, Evidera
L. Clark Paramore: bluebird bio
Katie D. Stewart: Patient-Centered Research, Evidera
Hayley Karn: Patient-Centered Research, Evidera
Minesh Jobanputra: bluebird bio
Andrew C. Dietz: bluebird bio

The European Journal of Health Economics, 2020, vol. 21, issue 3, No 7, 397-407

Abstract: 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

Keywords: Utility; Transfusion-dependent β-thalassemia; Stem cell transplant; Time trade-off (search for similar items in EconPapers)
JEL-codes: I10 I19 (search for similar items in EconPapers)
Date: 2020
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Citations: View citations in EconPapers (1)

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DOI: 10.1007/s10198-019-01136-0

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