ArticleThe European journal of health economics : HEPAC : health economics in prevention and care2020
Health state utilities associated with treatment for transfusion-dependent β-thalassemia.
Article in The European journal of health economics : HEPAC : health economics in prevention and care, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
15 citing papers in PubMed.
- Article
- Health state utilities associated with X-linked retinitis pigmentosa (XLRP).The European journal of health economics : HEPAC : health economics in prevention and care · 2025Article
- The Impact of Activated Phosphoinositide 3-Kinase δ Syndrome (APDS) on Health-Related Quality of Life (HRQoL): Elicitation of Health State Utility Values Through Time Trade-Off (TTO) and EQ-5D.Advances in therapy · 2025Article
- Health state utilities associated with invasive pneumococcal disease, pneumonia, and recurrent acute otitis media in young children.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2025Article
- A Vignette Study to Derive Health-Related Quality-of-Life Weights for Individuals with Steroid Refractory Chronic Graft-versus-Host Disease Receiving Third-Line Therapy in the United Kingdom.Journal of health economics and outcomes research · 2025Article
- Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD).The European journal of health economics : HEPAC : health economics in prevention and care · 2024Article
- Gene and cell therapy of human genetic diseases: Recent advances and future directions.Journal of cellular and molecular medicine · 2024Review
- Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States.PharmacoEconomics · 2024Article
- Health State Utilities Associated with False-Positive Cancer Screening Results.PharmacoEconomics - open · 2024Article
- Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States : A Cost-Effectiveness Analysis.Annals of internal medicine · 2024Article
- Health state utilities for beta-thalassemia: a time trade-off study.The European journal of health economics : HEPAC : health economics in prevention and care · 2023Article
- Disutilities Associated with Intravenous Iron Infusions: Results from a Time Trade-off Survey and Diminishing Marginal Utility Model for Treatment Attributes in China.Patient related outcome measures · 2023Article
- Health State Utilities for Adverse Events Associated with Chimeric Antigen Receptor T-Cell Therapy in Large B-Cell Lymphoma.PharmacoEconomics - open · 2022Article
- Cost Utility of Lifelong Immunoglobulin Replacement Therapy vs Hematopoietic Stem Cell Transplant to Treat Agammaglobulinemia.JAMA pediatrics · 2022Article
- Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia.Journal of market access & health policy · 2021Article
Corrections and comments
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Authors and funding
6 authors.
Funding
Abstract
objectivesTransfusion-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.
methodsGeneral population respondents in England valued eight health state vignettes (developed with clinician, patient, and parent input) in time trade-off interviews.
resultsA 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).
conclusionsUtilities 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.
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