Galanello R, Origa R. Beta-thalassemia. Orphanet J Rare Dis. 2010;5:11.

Article  PubMed  PubMed Central  Google Scholar 

Yardumian A. Standards for the clinical care of children and adults with thalassaemia in the UK. 3rd ed. UK Thalassaemia Society; 2016.

Taher AT, Weatherall DJ, Cappellini MD. Thalassaemia. Lancet. 2018;391:155–67.

Article  PubMed  Google Scholar 

Drahos J, Boateng-Kuffour A, Calvert M, Levine L, Dongha N, Li N, Pakbaz Z, Shah F, Ainsworth N, Martin A. Sustained humanistic burden and work impact in adults with transfusion-dependent beta-thalassemia (TDT): results from a global longitudinal survey. In Value Health, vol. 26. pp. S317. ISPOR 2023, May 7–10; 2023:S317.

Knoth RL, Gupta S, Perkowski K, Costantino H, Inyart B, Ashka L, Clapp K. Understanding the Association between Red Blood Cell Transfusion Utilization and Humanistic and Economic Burden in Patients with beta-Thalassemia from the Patients’ Perspective. J Clin Med 2023, 12.

Shah F, Telfer P, Velangi M, Pancham S, Wynn R, Pollard S, Chalmers E, Kell J, Carter AM, Hickey J, et al. Routine management, healthcare resource use and patient and carer-reported outcomes of patients with transfusion-dependent beta-thalassaemia in the United Kingdom: a mixed methods observational study. EJHaem. 2021;2:738–49.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Drahos J, Boateng-Kuffour A, Calvert M, Levine L, Dongha N, Li N, Pakbaz Z, Shah F, Martin AP. Health-Related Quality-of-Life Impacts Associated with Transfusion-Dependent beta-Thalassemia in the USA and UK: A Qualitative Assessment. Patient 2024.

Herdman M, Gudex C, Lloyd A, Janssen MF, Kind P, Parkin D, Bonsel G, Badia X. Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). Qual Life Res. 2011;20:1727–36.

Article  CAS  PubMed  PubMed Central  Google Scholar 

The EuroQol Group. EuroQol - a new facility for the measurement of health-related quality of life. Health Policy. 1990;16:199–208.

Article  Google Scholar 

National Institute for Health and Care Excellence: Position statement on use of the EQ-5D-5L value set for England (updated. October 2019). 2019.

Zhou T, Chen Z, Li H, Xie F. Using published health utilities in cost-utility analyses: discrepancies and issues in cardiovascular disease. Med Decis Mak. 2021;41:685–92.

Article  Google Scholar 

Thompson AJ, Turner AJ. A comparison of the EQ-5D-3L and EQ-5D-5L. PharmacoEconomics. 2020;38:575–91.

Article  PubMed  Google Scholar 

Buchholz I, Janssen MF, Kohlmann T, Feng YS. A systematic review of studies comparing the Measurement Properties of the three-level and five-level versions of the EQ-5D. PharmacoEconomics. 2018;36:645–61.

Article  PubMed  PubMed Central  Google Scholar 

Kangwanrattanakul K, Krageloh CU. EQ-5D-3L and EQ-5D-5L population norms for Thailand. BMC Public Health. 2024;24:1108.

Article  PubMed  PubMed Central  Google Scholar 

Seyedifar M, Dorkoosh FA, Hamidieh AA, Naderi M, Karami H, Karimi M, Fadaiyrayeny M, Musavi M, Safaei S, Ahmadian-Attari MM, et al. Health-related quality of life and health utility values in beta thalassemia major patients receiving different types of iron chelators in Iran. Int J Hematol Oncol Stem Cell Res. 2016;10:224–31.

PubMed  PubMed Central  Google Scholar 

Javanbakht M, Keshtkaran A, Shabaninejad H, Karami H, Zakerinia M, Delavari S. Comparison of blood transfusion plus chelation therapy and bone marrow transplantation in patients with β-thalassemia: application of SF-36, EQ-5D, and visual analogue scale measures. Int J Health Policy Manag. 2015;4:733–40.

Article  PubMed  PubMed Central  Google Scholar 

Shafie AA, Chhabra IK, Wong JHY, Mohammed NS. EQ–5D–3L health state utility values in transfusion–dependent thalassemia patients in Malaysia: a cross–sectional assessment. Health Qual Life Outcomes. 2021;19:10.

Article  PubMed  PubMed Central  Google Scholar 

Emrani Z, Akbari Sari A, Zeraati H, Olyaeemanesh A, Daroudi R. Health-related quality of life measured using the EQ-5D-5L: population norms for the capital of Iran. Health Qual Life Outcomes. 2020;18:108.

Article  PubMed  PubMed Central  Google Scholar 

Purba FD, Hunfeld JAM, Iskandarsyah A, Fitriana TS, Sadarjoen SS, Passchier J, Busschbach JJV. Quality of life of the Indonesian general population: test-retest reliability and population norms of the EQ-5D-5L and WHOQOL-BREF. PLoS ONE. 2018;13:e0197098.

Article  PubMed  PubMed Central  Google Scholar 

Jiang R, Janssen MFB, Pickard AS. US population norms for the EQ-5D-5L and comparison of norms from face-to-face and online samples. Qual Life Res. 2021;30:803–16.

Article  PubMed  Google Scholar 

Kangwanrattanakul K. Normative profile of the EQ-5D-5L dimensions, EQ-5D-5L index and EQ-VAS scores for the general Thai population. Qual Life Res. 2023;32:2489–502.

Article  PubMed  Google Scholar 

Payakachat N, Ali MM, Tilford JM. Can the EQ-5D detect meaningful change? A systematic review. PharmacoEconomics. 2015;33:1137–54.

Article  PubMed  PubMed Central  Google Scholar 

Lin F-J, Longworth L, Pickard AS. Evaluation of content on EQ-5D as compared to disease-specific utility measures. Qual Life Res. 2013;22:853–74.

Article  PubMed  Google Scholar 

Whalley D, Globe G, Crawford R, Doward L, Tafesse E, Brazier J, Price D. Is the EQ-5D fit for purpose in asthma? Acceptability and content validity from the patient perspective. Health Qual Life Outcomes. 2018;16:160.

Article  PubMed  PubMed Central  Google Scholar 

Efthymiadou O, Mossman J, Kanavos P. Health related quality of life aspects not captured by EQ-5D-5L: results from an international survey of patients. Health Policy. 2019;123:159–65.

Article  PubMed  Google Scholar 

Lim-Watson MZ, Hays RD, Kingsberg S, Kallich JD, Murimi-Worstell IB. A systematic literature review of health-related quality of life measures for women with hypoactive sexual desire disorder and female sexual interest/arousal disorder. Sex Med Rev. 2022;10:23–41.

Article  PubMed  Google Scholar 

Tosh J, Brazier J, Evans P, Longworth L. A review of generic preference-based measures of health-related quality of life in visual disorders. Value Health. 2012;15:118–27.

Article  PubMed  PubMed Central  Google Scholar 

Solem CT, Vera-Llonch M, Liu S, Botteman M, Castiglione B. Impact of pulmonary exacerbations and lung function on generic health-related quality of life in patients with cystic fibrosis. Health Qual Life Outcomes. 2016;14:63.

Article  PubMed  PubMed Central  Google Scholar 

Janssen B, Szende A. Population norms for the EQ-5D. In Self-reported population health: an international perspective based on EQ-5D Edited by Szende A, Janssen B, Cabases J. Dordrecht (NL): Springer; 2014: 19–30.

National Institute for Health and Care Excellence. NICE health technology evaluations: the manual. 2022.

Weidlich D, Kefalas P, Guest JF. Healthcare costs and outcomes of managing beta-thalassemia major over 50 years in the United Kingdom. Transfusion. 2016;56:1038–45.

Article  PubMed  Google Scholar 

Etemad K, Mohseni P, Aghighi M, Bahadorimonfared A, Hantooshzadeh R, Taherpour N, Piri N, Sotoodeh Ghorbani S, Malek F, Kheiry F, et al. Quality of life and related factors in β-thalassemia patients. Hemoglobin. 2021;45:245–9.

Article  CAS  PubMed  Google Scholar 

Greco F, Marino F. Social impact and quality of life of patients with β-thalassaemia: a systematic review. EMJ Hematol 2022.

Cappellini MD, Kattamis A, Viprakasit V, Sutcharitchan P, Pariseau J, Laadem A, Jessent-Ciaravino V, Taher A. Quality of life in patients with β-thalassemia: a prospective study of transfusion-dependent and non-transfusion-dependent patients in Greece, Italy, Lebanon, and Thailand. Am J Hematol. 2019;94:E261–4.

Article  PubMed  Google Scholar 

Li N, Drahos J, Boateng-Kuffour A, Calvert M, Levine L, Dongha N, Pakbaz Z, Oran FK, Iqbal K, Shah F, Martin AP. Health-related quality of life and disease impacts in adults with transfusion-dependent β-thalassemia: preliminary results from the global longitudinal survey. In Blood, vol. 140. pp. 10869–10870. 64th ASH Annual Meeting, December 10–13; 2022:10869–10870.

Boateng-Kuffour A, Drahos J, Kohli P, Skrobanski H, Forster K, Acaster S, Pakbaz Z, Li N, Williams K. Evaluating the appropriateness of the EQ-5D-5L descriptive system and the derived health utility index scores in adults with transfusion-dependent beta-thalassemia: a mixed-methods study. In Value Health, vol. 26. pp. S7. ISPOR 2023, May 7–10; 2023:S7.

Boateng-Kuffour A, Drahos J, Kohli P, Skrobanski H, Forster K, Acaster S, Pakbaz Z, Li N, Williams K. Symptoms and impacts of transfusion-dependent beta-thalassemia: a qualitative interview study and the development of a conceptual model. In Value Health, vol. 26. pp. S336. ISPOR 2023, May 7–10; 2023:S336.

Udeze C, Kunzweiler C, Li N, Baldwin J, Tuzin P, Dietmar Barth S, Vetter C, Dombrowski S, Georgiadou-Schmidt E, Meisel R. Clinical complications among patients with transfusion-dependent beta-thalassemia in Germany. In Hemasphere, vol. 7. pp. e62709d62705. EHA2023, Frankfurt and virtual, June 8–15; 2023:e62709d62705.

Udeze C, Dovizio M, Veronesi C, Esposti LD, Li N, Mai Patricia Dang TX, Forni GL. Mortality and clinical complications among patients with transfusion-dependent beta-thalassemia in Italy. In Hemasphere, vol. 7. pp. e91927ad. EHA2023, Frankfurt and virtual, June 8–15; 2023:e91927ad.

Udeze C, Evans K, Yang Y, Lillehaugen T, Manjelievskaia J, Mujumdar U, Li N, Andemariam B. Clinical and economic outcomes in patients with transfusion-dependent β-thalassemia and patients with sickle cell disease with recurrent vaso-occlusive crises receiving hematopoietic stem cell transplants in the United States. In Blood, vol. 140. pp. 10741–10742. 64th ASH Annual Meeting, December 10–13,; 2022:10741–10742.

Udeze C, Evans KA, Yang Y, Lillehaugen T, Manjelievskaia J, Mujumdar U, Li N, Andemariam B. Economic and clinical burden of managing transfusion-dependent β-thalassemia in the United States. J Med Econ. 2023;26:924–32.

Article  PubMed  Google Scholar 

Tedone F, Lamendola P, Lopatriello S, Cafiero D, Piovani D, Forni GL. Quality of life and burden of disease in Italian patients with transfusion-dependent beta-thalassemia. J Clin Med. 2022;11:15.

Article  Google Scholar 

Shafie AA, Chhabra IK, Wong JHY, Mohammed NS, Ibrahim HM, Alias H. Health-related quality of life among children with transfusion-dependent thalassemia: a cross-sectional study in Malaysia. Health Qual Life Outcomes. 2020;18:141.

Article  PubMed  PubMed Central