Health state utilities associated with treatment for transfusion-dependent β-thalassemia
| العنوان: | Health state utilities associated with treatment for transfusion-dependent β-thalassemia |
|---|---|
| المؤلفون: | Katie D. Stewart, Hayley Karn, L Clark Paramore, Louis S. Matza, Minesh Jobanputra, Andrew C Dietz |
| المصدر: | The European Journal of Health Economics |
| بيانات النشر: | Springer Berlin Heidelberg, 2019. |
| سنة النشر: | 2019 |
| مصطلحات موضوعية: | Adult, Male, medicine.medical_specialty, medicine.medical_treatment, Thalassemia, Economics, Econometrics and Finance (miscellaneous), Population, Pilot Projects, Hematopoietic stem cell transplantation, Disease, Time-trade-off, Interviews as Topic, 03 medical and health sciences, 0302 clinical medicine, Quality of life, Utility, Internal medicine, medicine, Autologous transplantation, Humans, Blood Transfusion, I10, education, Aged, education.field_of_study, Original Paper, business.industry, 030503 health policy & services, Health Policy, Public health, beta-Thalassemia, Transfusion-dependent β-thalassemia, Patient Preference, Genetic Therapy, Middle Aged, medicine.disease, Time trade-off, I19, Chelation Therapy, England, 030220 oncology & carcinogenesis, Quality of Life, Stem cell transplant, Female, 0305 other medical science, business |
| الوصف: | 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 p 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. |
| اللغة: | English |
| تدمد: | 1618-7601 1618-7598 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f64beac1a0b40a3de34bc1728e8d15b8 http://europepmc.org/articles/PMC7188724 |
| حقوق: | OPEN |
| رقم الأكسشن: | edsair.doi.dedup.....f64beac1a0b40a3de34bc1728e8d15b8 |
| قاعدة البيانات: | OpenAIRE |
Find this article in full text from Springer Verlag. | تدمد: | 16187601 16187598 |
|---|