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

Author

Listed:
  • 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)

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

Suggested Citation

  • Louis S. Matza & L. Clark Paramore & Katie D. Stewart & Hayley Karn & Minesh Jobanputra & Andrew C. Dietz, 2020. "Health state utilities associated with treatment for transfusion-dependent β-thalassemia," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 21(3), pages 397-407, April.
  • Handle: RePEc:spr:eujhec:v:21:y:2020:i:3:d:10.1007_s10198-019-01136-0
    DOI: 10.1007/s10198-019-01136-0
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    References listed on IDEAS

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    More about this item

    Keywords

    Utility; Transfusion-dependent β-thalassemia; Stem cell transplant; Time trade-off;
    All these keywords.

    JEL classification:

    • I10 - Health, Education, and Welfare - - Health - - - General
    • I19 - Health, Education, and Welfare - - Health - - - Other

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