IDEAS home Printed from https://ideas.repec.org/a/spr/eujhec/v21y2020i3d10.1007_s10198-019-01136-0.html
   My bibliography  Save this article

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
    as

    Download full text from publisher

    File URL: http://link.springer.com/10.1007/s10198-019-01136-0
    File Function: Abstract
    Download Restriction: Access to the full text of the articles in this series is restricted.

    File URL: https://libkey.io/10.1007/s10198-019-01136-0?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    As the access to this document is restricted, you may want to search for a different version of it.

    References listed on IDEAS

    as
    1. Victoria Brennan & Simon Dixon, 2013. "Incorporating Process Utility into Quality Adjusted Life Years: A Systematic Review of Empirical Studies," PharmacoEconomics, Springer, vol. 31(8), pages 677-691, August.
    2. F. E. van Nooten & X. Koolman & W. B. F. Brouwer, 2009. "The influence of subjective life expectancy on health state valuations using a 10 year TTO," Health Economics, John Wiley & Sons, Ltd., vol. 18(5), pages 549-558, May.
    3. Han Bleichrodt & Magnus Johannesson, 1997. "The Validity of QALYs," Medical Decision Making, , vol. 17(1), pages 21-32, February.
    4. Louis Matza & Sandhya Sapra & John Dillon & Anupama Kalsekar & Evan Davies & Mary Devine & Jessica Jordan & Amanda Landrian & David Feeny, 2015. "Health state utilities associated with attributes of treatments for hepatitis C," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 16(9), pages 1005-1018, December.
    5. Paul Dolan & Claire Gudex & Paul Kind & Alan Williams, 1996. "The time trade‐off method: Results from a general population study," Health Economics, John Wiley & Sons, Ltd., vol. 5(2), pages 141-154, March.
    Full references (including those not matched with items on IDEAS)

    Citations

    Citations are extracted by the CitEc Project, subscribe to its RSS feed for this item.
    as


    Cited by:

    1. Louis S. Matza & Katie D. Stewart & Marie Fournier & Donna Rowen & Robin Lachmann & Maurizio Scarpa & Eugen Mengel & Travis Obermeyer & Evren Ayik & Fernando Laredo & Ruth Pulikottil-Jacob, 2024. "Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD)," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 25(8), pages 1437-1448, November.

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Arthur Attema & Werner Brouwer, 2012. "The way that you do it? An elaborate test of procedural invariance of TTO, using a choice-based design," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 13(4), pages 491-500, August.
    2. Louis Matza & Sandhya Sapra & John Dillon & Anupama Kalsekar & Evan Davies & Mary Devine & Jessica Jordan & Amanda Landrian & David Feeny, 2015. "Health state utilities associated with attributes of treatments for hepatitis C," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 16(9), pages 1005-1018, December.
    3. Anne-Gaëlle Corroller & Julia Bonastre, 2023. "Patient-reported measures: how useful in health economics?," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 24(1), pages 1-4, February.
    4. Ryan Edwards, 2013. "The cost of uncertain life span," Journal of Population Economics, Springer;European Society for Population Economics, vol. 26(4), pages 1485-1522, October.
    5. R. Hoorn & A. Donders & M. Oppe & P. Stalmeier, 2014. "The Better than Dead Method: Feasibility and Interpretation of a Valuation Study," PharmacoEconomics, Springer, vol. 32(8), pages 789-799, August.
    6. Bleichrodt, Han & Filko, Martin, 2008. "New tests of QALYs when health varies over time," Journal of Health Economics, Elsevier, vol. 27(5), pages 1237-1249, September.
    7. Attema, Arthur E. & Brouwer, Werner B.F., 2012. "A test of independence of discounting from quality of life," Journal of Health Economics, Elsevier, vol. 31(1), pages 22-34.
    8. Emelie Heintz & Marieke Krol & Lars-Åke Levin, 2013. "The Impact of Patients’ Subjective Life Expectancy on Time Tradeoff Valuations," Medical Decision Making, , vol. 33(2), pages 261-270, February.
    9. Patricia Cubi-Molla & Koonal Shah & Kristina Burström, 2018. "Experience-Based Values: A Framework for Classifying Different Types of Experience in Health Valuation Research," The Patient: Patient-Centered Outcomes Research, Springer;International Academy of Health Preference Research, vol. 11(3), pages 253-270, June.
    10. Lipman, Stefan A. & Brouwer, Werner B.F. & Attema, Arthur E., 2020. "Living up to expectations: Experimental tests of subjective life expectancy as reference point in time trade-off and standard gamble," Journal of Health Economics, Elsevier, vol. 71(C).
    11. David J. Mott & Iain Leslie & Koonal Shah & Jennifer Rowell & Nicolas Scheuer, 2021. "Impact of Including Carer Information in Time Trade-Off Tasks: Results from a Pilot Study," PharmacoEconomics - Open, Springer, vol. 5(4), pages 665-675, December.
    12. Trude Arnesen & Mari Trommald, 2005. "Are QALYs based on time trade‐off comparable? – A systematic review of TTO methodologies," Health Economics, John Wiley & Sons, Ltd., vol. 14(1), pages 39-53, January.
    13. Richard Norman & Jane Hall & Deborah Street & Rosalie Viney, 2013. "Efficiency And Equity: A Stated Preference Approach," Health Economics, John Wiley & Sons, Ltd., vol. 22(5), pages 568-581, May.
    14. Mônica Viegas Andrade & Kenya Noronha & Paul Kind & Carla de Barros Reis & Lucas Resende de Carvalho, 2016. "Logical Inconsistencies in 3 Preference Elicitation Methods for EQ-5D Health States," Medical Decision Making, , vol. 36(2), pages 242-252, February.
    15. Rachel Baker & Angela Robinson, 2004. "Responses to standard gambles: are preferences ‘well constructed’?," Health Economics, John Wiley & Sons, Ltd., vol. 13(1), pages 37-48, January.
    16. Louis S. Matza & Katherine J. Kim & Holly Yu & Katherine A. Belden & Antonia F. Chen & Mark Kurd & Bruce Y. Lee & Jason Webb, 2019. "Health state utilities associated with post-surgical Staphylococcus aureus infections," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 20(6), pages 819-827, August.
    17. James Hammitt & Nicolas Treich, 2007. "Statistical vs. identified lives in benefit-cost analysis," Journal of Risk and Uncertainty, Springer, vol. 35(1), pages 45-66, August.
    18. Richard Norman & Rebecca Mercieca‐Bebber & Donna Rowen & John E. Brazier & David Cella & A. Simon Pickard & Deborah J. Street & Rosalie Viney & Dennis Revicki & Madeleine T. King & On behalf of the Eu, 2019. "U.K. utility weights for the EORTC QLU‐C10D," Health Economics, John Wiley & Sons, Ltd., vol. 28(12), pages 1385-1401, December.
    19. John Mullahy, 2001. "Live long, live well: quantifying the health of heterogeneous populations," Health Economics, John Wiley & Sons, Ltd., vol. 10(5), pages 429-440, July.
    20. Bernt Kartman & Gudrun Gatz & Magnus Johannesson, 2004. "Health State Utilities in Gastroesophageal Reflux Disease Patients with Heartburn: A Study in Germany and Sweden," Medical Decision Making, , vol. 24(1), pages 40-52, January.

    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

    Statistics

    Access and download statistics

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:spr:eujhec:v:21:y:2020:i:3:d:10.1007_s10198-019-01136-0. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Sonal Shukla or Springer Nature Abstracting and Indexing (email available below). General contact details of provider: http://www.springer.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.