Health state utilities for beta-thalassemia: a time trade-off study
Background Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron che...
Ausführliche Beschreibung
Gespeichert in:
| Autor*in: |
Martin, Antony P. [verfasserIn] |
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| Format: |
Artikel |
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| Sprache: |
Englisch |
| Erschienen: |
2022 |
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| Schlagwörter: |
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| Anmerkung: |
© The Author(s) 2022 |
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| Übergeordnetes Werk: |
Enthalten in: The European journal of health economics - Springer Berlin Heidelberg, 2001, 24(2022), 1 vom: 26. März, Seite 27-38 |
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| Übergeordnetes Werk: |
volume:24 ; year:2022 ; number:1 ; day:26 ; month:03 ; pages:27-38 |
| Links: |
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| DOI / URN: |
10.1007/s10198-022-01449-7 |
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OLC2133551875 |
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| 520 | |a Background Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. Methods Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. Results The mean age of participants was 41.50 years (SD 16.01, range 18–81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies. | ||
| 650 | 4 | |a Beta-thalassemia | |
| 650 | 4 | |a Hemoglobin | |
| 650 | 4 | |a Anemia | |
| 650 | 4 | |a Time trade-off | |
| 650 | 4 | |a Transfusion burden | |
| 700 | 1 | |a Ferri Grazzi, Enrico |0 (orcid)0000-0002-0567-0260 |4 aut | |
| 700 | 1 | |a Mighiu, Claudia |4 aut | |
| 700 | 1 | |a Chevli, Manoj |4 aut | |
| 700 | 1 | |a Shah, Farrukh |4 aut | |
| 700 | 1 | |a Maher, Louise |4 aut | |
| 700 | 1 | |a Shaikh, Anum |4 aut | |
| 700 | 1 | |a Sagar, Aliah |4 aut | |
| 700 | 1 | |a Hubberstey, Hayley |4 aut | |
| 700 | 1 | |a Franks, Bethany |4 aut | |
| 700 | 1 | |a Ramos-Goñi, Juan M. |4 aut | |
| 700 | 1 | |a Oppe, Mark |0 (orcid)0000-0003-4286-8855 |4 aut | |
| 700 | 1 | |a Tang, Derek |4 aut | |
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10.1007/s10198-022-01449-7 doi (DE-627)OLC2133551875 (DE-He213)s10198-022-01449-7-p DE-627 ger DE-627 rakwb eng 330 610 VZ 610 VZ 44.05$jGesundheitsökonomie bkl 44.10$jGesundheitswesen: Allgemeines bkl Martin, Antony P. verfasserin (orcid)0000-0003-4383-6038 aut Health state utilities for beta-thalassemia: a time trade-off study 2022 Text txt rdacontent ohne Hilfsmittel zu benutzen n rdamedia Band nc rdacarrier © The Author(s) 2022 Background Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. Methods Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. Results The mean age of participants was 41.50 years (SD 16.01, range 18–81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies. Beta-thalassemia Hemoglobin Anemia Time trade-off Transfusion burden Ferri Grazzi, Enrico (orcid)0000-0002-0567-0260 aut Mighiu, Claudia aut Chevli, Manoj aut Shah, Farrukh aut Maher, Louise aut Shaikh, Anum aut Sagar, Aliah aut Hubberstey, Hayley aut Franks, Bethany aut Ramos-Goñi, Juan M. aut Oppe, Mark (orcid)0000-0003-4286-8855 aut Tang, Derek aut Enthalten in The European journal of health economics Springer Berlin Heidelberg, 2001 24(2022), 1 vom: 26. März, Seite 27-38 (DE-627)328188557 (DE-600)2045253-6 (DE-576)09442246X 1618-7598 nnns volume:24 year:2022 number:1 day:26 month:03 pages:27-38 https://doi.org/10.1007/s10198-022-01449-7 lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_OLC SSG-OLC-WIW GBV_ILN_267 GBV_ILN_2018 GBV_ILN_2026 GBV_ILN_4277 GBV_ILN_4310 44.05$jGesundheitsökonomie VZ 106409611 (DE-625)106409611 44.10$jGesundheitswesen: Allgemeines VZ 106409530 (DE-625)106409530 AR 24 2022 1 26 03 27-38 |
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10.1007/s10198-022-01449-7 doi (DE-627)OLC2133551875 (DE-He213)s10198-022-01449-7-p DE-627 ger DE-627 rakwb eng 330 610 VZ 610 VZ 44.05$jGesundheitsökonomie bkl 44.10$jGesundheitswesen: Allgemeines bkl Martin, Antony P. verfasserin (orcid)0000-0003-4383-6038 aut Health state utilities for beta-thalassemia: a time trade-off study 2022 Text txt rdacontent ohne Hilfsmittel zu benutzen n rdamedia Band nc rdacarrier © The Author(s) 2022 Background Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. Methods Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. Results The mean age of participants was 41.50 years (SD 16.01, range 18–81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies. Beta-thalassemia Hemoglobin Anemia Time trade-off Transfusion burden Ferri Grazzi, Enrico (orcid)0000-0002-0567-0260 aut Mighiu, Claudia aut Chevli, Manoj aut Shah, Farrukh aut Maher, Louise aut Shaikh, Anum aut Sagar, Aliah aut Hubberstey, Hayley aut Franks, Bethany aut Ramos-Goñi, Juan M. aut Oppe, Mark (orcid)0000-0003-4286-8855 aut Tang, Derek aut Enthalten in The European journal of health economics Springer Berlin Heidelberg, 2001 24(2022), 1 vom: 26. März, Seite 27-38 (DE-627)328188557 (DE-600)2045253-6 (DE-576)09442246X 1618-7598 nnns volume:24 year:2022 number:1 day:26 month:03 pages:27-38 https://doi.org/10.1007/s10198-022-01449-7 lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_OLC SSG-OLC-WIW GBV_ILN_267 GBV_ILN_2018 GBV_ILN_2026 GBV_ILN_4277 GBV_ILN_4310 44.05$jGesundheitsökonomie VZ 106409611 (DE-625)106409611 44.10$jGesundheitswesen: Allgemeines VZ 106409530 (DE-625)106409530 AR 24 2022 1 26 03 27-38 |
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10.1007/s10198-022-01449-7 doi (DE-627)OLC2133551875 (DE-He213)s10198-022-01449-7-p DE-627 ger DE-627 rakwb eng 330 610 VZ 610 VZ 44.05$jGesundheitsökonomie bkl 44.10$jGesundheitswesen: Allgemeines bkl Martin, Antony P. verfasserin (orcid)0000-0003-4383-6038 aut Health state utilities for beta-thalassemia: a time trade-off study 2022 Text txt rdacontent ohne Hilfsmittel zu benutzen n rdamedia Band nc rdacarrier © The Author(s) 2022 Background Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. Methods Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. Results The mean age of participants was 41.50 years (SD 16.01, range 18–81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies. Beta-thalassemia Hemoglobin Anemia Time trade-off Transfusion burden Ferri Grazzi, Enrico (orcid)0000-0002-0567-0260 aut Mighiu, Claudia aut Chevli, Manoj aut Shah, Farrukh aut Maher, Louise aut Shaikh, Anum aut Sagar, Aliah aut Hubberstey, Hayley aut Franks, Bethany aut Ramos-Goñi, Juan M. aut Oppe, Mark (orcid)0000-0003-4286-8855 aut Tang, Derek aut Enthalten in The European journal of health economics Springer Berlin Heidelberg, 2001 24(2022), 1 vom: 26. März, Seite 27-38 (DE-627)328188557 (DE-600)2045253-6 (DE-576)09442246X 1618-7598 nnns volume:24 year:2022 number:1 day:26 month:03 pages:27-38 https://doi.org/10.1007/s10198-022-01449-7 lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_OLC SSG-OLC-WIW GBV_ILN_267 GBV_ILN_2018 GBV_ILN_2026 GBV_ILN_4277 GBV_ILN_4310 44.05$jGesundheitsökonomie VZ 106409611 (DE-625)106409611 44.10$jGesundheitswesen: Allgemeines VZ 106409530 (DE-625)106409530 AR 24 2022 1 26 03 27-38 |
| allfieldsGer |
10.1007/s10198-022-01449-7 doi (DE-627)OLC2133551875 (DE-He213)s10198-022-01449-7-p DE-627 ger DE-627 rakwb eng 330 610 VZ 610 VZ 44.05$jGesundheitsökonomie bkl 44.10$jGesundheitswesen: Allgemeines bkl Martin, Antony P. verfasserin (orcid)0000-0003-4383-6038 aut Health state utilities for beta-thalassemia: a time trade-off study 2022 Text txt rdacontent ohne Hilfsmittel zu benutzen n rdamedia Band nc rdacarrier © The Author(s) 2022 Background Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. Methods Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. Results The mean age of participants was 41.50 years (SD 16.01, range 18–81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies. Beta-thalassemia Hemoglobin Anemia Time trade-off Transfusion burden Ferri Grazzi, Enrico (orcid)0000-0002-0567-0260 aut Mighiu, Claudia aut Chevli, Manoj aut Shah, Farrukh aut Maher, Louise aut Shaikh, Anum aut Sagar, Aliah aut Hubberstey, Hayley aut Franks, Bethany aut Ramos-Goñi, Juan M. aut Oppe, Mark (orcid)0000-0003-4286-8855 aut Tang, Derek aut Enthalten in The European journal of health economics Springer Berlin Heidelberg, 2001 24(2022), 1 vom: 26. März, Seite 27-38 (DE-627)328188557 (DE-600)2045253-6 (DE-576)09442246X 1618-7598 nnns volume:24 year:2022 number:1 day:26 month:03 pages:27-38 https://doi.org/10.1007/s10198-022-01449-7 lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_OLC SSG-OLC-WIW GBV_ILN_267 GBV_ILN_2018 GBV_ILN_2026 GBV_ILN_4277 GBV_ILN_4310 44.05$jGesundheitsökonomie VZ 106409611 (DE-625)106409611 44.10$jGesundheitswesen: Allgemeines VZ 106409530 (DE-625)106409530 AR 24 2022 1 26 03 27-38 |
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10.1007/s10198-022-01449-7 doi (DE-627)OLC2133551875 (DE-He213)s10198-022-01449-7-p DE-627 ger DE-627 rakwb eng 330 610 VZ 610 VZ 44.05$jGesundheitsökonomie bkl 44.10$jGesundheitswesen: Allgemeines bkl Martin, Antony P. verfasserin (orcid)0000-0003-4383-6038 aut Health state utilities for beta-thalassemia: a time trade-off study 2022 Text txt rdacontent ohne Hilfsmittel zu benutzen n rdamedia Band nc rdacarrier © The Author(s) 2022 Background Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. Methods Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. Results The mean age of participants was 41.50 years (SD 16.01, range 18–81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies. Beta-thalassemia Hemoglobin Anemia Time trade-off Transfusion burden Ferri Grazzi, Enrico (orcid)0000-0002-0567-0260 aut Mighiu, Claudia aut Chevli, Manoj aut Shah, Farrukh aut Maher, Louise aut Shaikh, Anum aut Sagar, Aliah aut Hubberstey, Hayley aut Franks, Bethany aut Ramos-Goñi, Juan M. aut Oppe, Mark (orcid)0000-0003-4286-8855 aut Tang, Derek aut Enthalten in The European journal of health economics Springer Berlin Heidelberg, 2001 24(2022), 1 vom: 26. März, Seite 27-38 (DE-627)328188557 (DE-600)2045253-6 (DE-576)09442246X 1618-7598 nnns volume:24 year:2022 number:1 day:26 month:03 pages:27-38 https://doi.org/10.1007/s10198-022-01449-7 lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_OLC SSG-OLC-WIW GBV_ILN_267 GBV_ILN_2018 GBV_ILN_2026 GBV_ILN_4277 GBV_ILN_4310 44.05$jGesundheitsökonomie VZ 106409611 (DE-625)106409611 44.10$jGesundheitswesen: Allgemeines VZ 106409530 (DE-625)106409530 AR 24 2022 1 26 03 27-38 |
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Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. 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Martin, Antony P. Ferri Grazzi, Enrico Mighiu, Claudia Chevli, Manoj Shah, Farrukh Maher, Louise Shaikh, Anum Sagar, Aliah Hubberstey, Hayley Franks, Bethany Ramos-Goñi, Juan M. Oppe, Mark Tang, Derek |
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health state utilities for beta-thalassemia: a time trade-off study |
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Health state utilities for beta-thalassemia: a time trade-off study |
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Background Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. Methods Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. Results The mean age of participants was 41.50 years (SD 16.01, range 18–81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies. © The Author(s) 2022 |
| abstractGer |
Background Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. Methods Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. Results The mean age of participants was 41.50 years (SD 16.01, range 18–81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies. © The Author(s) 2022 |
| abstract_unstemmed |
Background Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. Methods Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. Results The mean age of participants was 41.50 years (SD 16.01, range 18–81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies. © The Author(s) 2022 |
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Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. Conclusions This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. 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