Skip to main content
Top
Published in: Health and Quality of Life Outcomes 1/2021

Open Access 01-12-2021 | Thalassemia | Research

EQ-5D-3L health state utility values in transfusion-dependent thalassemia patients in Malaysia: a cross-sectional assessment

Authors: Asrul Akmal Shafie, Irwinder Kaur Chhabra, Jacqueline Hui Yi Wong, Noor Syahireen Mohammed

Published in: Health and Quality of Life Outcomes | Issue 1/2021

Login to get access

Abstract

Purpose

There is a gap of information describing the health state utility values (HSUVs) of transfusion-dependent thalassemia (TDT) patients in Malaysia. These values are useful in the assessment of health-related quality of life (HRQoL), economic evaluations and provide guidance to disease management decisions. The objective of this study was to estimate and derive HSUVs associated with the treatment and complications of TDT patients in Malaysia using the EQ-5D-3L instrument.

Methods

A cross-sectional survey using the EQ-5D-3L instrument was conducted between May to September 2018 across various public hospitals in Malaysia. Using a multi-stage sampling, patients diagnosed with TDT and receiving iron chelating therapy were sampled. The findings on the EQ-5D-3L survey were converted into utility values using local tariff values. A two-part model was used to examine and derive the HSUVs associated with the treatment and complications of iron overload in TDT.

Results

A total of 585 patients were surveyed. The unadjusted mean (SD) EQ-5D-3L utility value for TDT patients were 0.893 (0.167) while mean (SD) EQ VAS score was 81.22 (16.92). Patients who had more than two iron overload complications had a significant decline in HRQoL. Patients who were on oral monotherapy had a higher utility value of 0.9180 compared to other regimen combinations.

Conclusion

Lower EQ-5D-3L utility values were associated with patients who developed iron overload complications and were on multiple iron chelating agents. Emphasizing compliance to iron chelating therapy to prevent the development of complications is crucial in the effort to preserve the HRQoL of TDT patients.
Appendix
Available only for authorised users
Literature
2.
go back to reference Weatherall D, Clegg J. Historical perspectives: in The Thalassemia syndromes. 4th ed. Oxford UK: Blackwell Scientific; 2001.CrossRef Weatherall D, Clegg J. Historical perspectives: in The Thalassemia syndromes. 4th ed. Oxford UK: Blackwell Scientific; 2001.CrossRef
3.
go back to reference De Sanctis V, Kattamis C, Canatan D, et al. β-thalassemia distribution in the old world: an ancient disease seen from a historical standpoint. Mediterr J Hematol Infect Dis. 2017;9:e2017018.PubMedPubMedCentralCrossRef De Sanctis V, Kattamis C, Canatan D, et al. β-thalassemia distribution in the old world: an ancient disease seen from a historical standpoint. Mediterr J Hematol Infect Dis. 2017;9:e2017018.PubMedPubMedCentralCrossRef
4.
go back to reference Elizabeth G, Ann TM. Genotype-phenotype diversity of beta-thalassemia in Malaysia: treatment options and emerging therapies. Med J Malaysia. 2010;65:256–60. Elizabeth G, Ann TM. Genotype-phenotype diversity of beta-thalassemia in Malaysia: treatment options and emerging therapies. Med J Malaysia. 2010;65:256–60.
5.
go back to reference George E. Beta-thalassemia major in Malaysia, an ongoing public health problem. Med J Malaysia. 2001;56(4):397–400.PubMed George E. Beta-thalassemia major in Malaysia, an ongoing public health problem. Med J Malaysia. 2001;56(4):397–400.PubMed
6.
go back to reference Ibrahim MH. Malaysian Thalassaemia Registry Report. 1st ed. Medical Development Division, Ministry of Health Malaysia. 2019 Ibrahim MH. Malaysian Thalassaemia Registry Report. 1st ed. Medical Development Division, Ministry of Health Malaysia. 2019
7.
go back to reference Ibrahim HM, Hassan A, George E, Goh AI, et al. Management of Transfusion Dependent Thalassaemia. In: Health Technology Assessment Section MDD. Putrajaya: CPG Secretariat; 2009, p. 1–21. Ibrahim HM, Hassan A, George E, Goh AI, et al. Management of Transfusion Dependent Thalassaemia. In: Health Technology Assessment Section MDD. Putrajaya: CPG Secretariat; 2009, p. 1–21.
8.
go back to reference Cappellini M-D, Cohen A, Porter J, Taher A, Viprakasit V. Guidelines for the management of transfusion dependent thalassaemia (TDT). Cyprus: Thalassaemia International Federation Nicosia; 2014. Cappellini M-D, Cohen A, Porter J, Taher A, Viprakasit V. Guidelines for the management of transfusion dependent thalassaemia (TDT). Cyprus: Thalassaemia International Federation Nicosia; 2014.
9.
go back to reference Weatherall DJ. The evolving spectrum of the epidemiology of thalassemia. Hematol Oncol Clin. 2018;32:165–75.CrossRef Weatherall DJ. The evolving spectrum of the epidemiology of thalassemia. Hematol Oncol Clin. 2018;32:165–75.CrossRef
10.
go back to reference Telfer P, Constantinidou G, Andreou P, Christou S, Modell B, Angastiniotis M. Quality of life in thalassemia. Ann N Y Acad Sci. 2005;1054:273–82.PubMedCrossRef Telfer P, Constantinidou G, Andreou P, Christou S, Modell B, Angastiniotis M. Quality of life in thalassemia. Ann N Y Acad Sci. 2005;1054:273–82.PubMedCrossRef
11.
go back to reference Guyatt GH, Feeny DH, Patrick DL. Measuring health-related quality of life. Ann Intern Med. 1993;118:622–9.CrossRefPubMed Guyatt GH, Feeny DH, Patrick DL. Measuring health-related quality of life. Ann Intern Med. 1993;118:622–9.CrossRefPubMed
12.
go back to reference Ismail A, Campbell MJ, Ibrahim HM, Jones GL. Health related quality of life in Malaysian children with thalassaemia. Health Qual Life Outcomes. 2006;4:39.PubMedPubMedCentralCrossRef Ismail A, Campbell MJ, Ibrahim HM, Jones GL. Health related quality of life in Malaysian children with thalassaemia. Health Qual Life Outcomes. 2006;4:39.PubMedPubMedCentralCrossRef
13.
go back to reference Sazlina S, Asauji Y, Juni MH. Predictors of health related quality of life among children and adolescents with beta thalassemia in three hospitals in Malaysia: a cross sectional study. Int J Public Health Clin Sci. 2015;2:1–12. Sazlina S, Asauji Y, Juni MH. Predictors of health related quality of life among children and adolescents with beta thalassemia in three hospitals in Malaysia: a cross sectional study. Int J Public Health Clin Sci. 2015;2:1–12.
14.
go back to reference Ismail M, Chun CY, Shahar S, et al. Quality of life among thalassaemia children, adolescent and their caregivers. Sains Malaysiana. 2013;42:373–80. Ismail M, Chun CY, Shahar S, et al. Quality of life among thalassaemia children, adolescent and their caregivers. Sains Malaysiana. 2013;42:373–80.
15.
go back to reference Dahlui M, Hishamshah MI, Rahman A, Aljunid SM. Quality of life in transfusion-dependent thalassaemia patients on desferrioxamine treatment. Singapore Med J. 2009;50:794.PubMed Dahlui M, Hishamshah MI, Rahman A, Aljunid SM. Quality of life in transfusion-dependent thalassaemia patients on desferrioxamine treatment. Singapore Med J. 2009;50:794.PubMed
16.
go back to reference Suzanah A, Zulaiha M, Faszrul AA, Kamaruzaman W. Thalassaemia: a study on the perception of patients and family members. Med J Malaysia. 2011;66:327. Suzanah A, Zulaiha M, Faszrul AA, Kamaruzaman W. Thalassaemia: a study on the perception of patients and family members. Med J Malaysia. 2011;66:327.
17.
go back to reference Karimi M, Brazier J. Health, health-related quality of life, and quality of life: what is the difference? Pharmacoeconomics. 2016;34:645–9.CrossRefPubMed Karimi M, Brazier J. Health, health-related quality of life, and quality of life: what is the difference? Pharmacoeconomics. 2016;34:645–9.CrossRefPubMed
18.
19.
go back to reference Shafie AA, Thakumar AV. Multiplicative modelling of EQ-5D-3L TTO and VAS values. Eur J Health Econ. 2020;21:1411–20.PubMedCrossRef Shafie AA, Thakumar AV. Multiplicative modelling of EQ-5D-3L TTO and VAS values. Eur J Health Econ. 2020;21:1411–20.PubMedCrossRef
20.
go back to reference Shafie AA, Vasan Thakumar A, Lim CJ, Luo N, Rand-Hendriksen K, Md Yusof FA. EQ-5D-5L valuation for the malaysian population. Pharmacoeconomics. 2019;37:715–25.PubMedCrossRef Shafie AA, Vasan Thakumar A, Lim CJ, Luo N, Rand-Hendriksen K, Md Yusof FA. EQ-5D-5L valuation for the malaysian population. Pharmacoeconomics. 2019;37:715–25.PubMedCrossRef
21.
go back to reference Naing L, Winn T, Rusli B. Practical issues in calculating the sample size for prevalence studies. Arch Orofac Sci. 2006;1:9–14. Naing L, Winn T, Rusli B. Practical issues in calculating the sample size for prevalence studies. Arch Orofac Sci. 2006;1:9–14.
22.
go back to reference Shafie AA, Hassali MA, Liau SY. A cross-sectional validation study of EQ-5D among the Malaysian adult population. Qual Life Res. 2011;20:593–600.PubMedCrossRef Shafie AA, Hassali MA, Liau SY. A cross-sectional validation study of EQ-5D among the Malaysian adult population. Qual Life Res. 2011;20:593–600.PubMedCrossRef
23.
go back to reference StataCorp. Stata statistical software: release 14. College Station, TX: StataCorp LP.2015. StataCorp. Stata statistical software: release 14. College Station, TX: StataCorp LP.2015.
24.
go back to reference Shafie AA, Thakumar AV. Multiplicative modelling of EQ-5D-3L TTO and VAS values. Eur J Health Econ. 2020;21(9):1411–20.PubMedCrossRef Shafie AA, Thakumar AV. Multiplicative modelling of EQ-5D-3L TTO and VAS values. Eur J Health Econ. 2020;21(9):1411–20.PubMedCrossRef
26.
go back to reference Huang IC, Frangakis C, Atkinson MJ, et al. Addressing ceiling effects in health status measures: a comparison of techniques applied to measures for people with HIV disease. Health Serv Res. 2008;43:327–39.PubMedPubMedCentralCrossRef Huang IC, Frangakis C, Atkinson MJ, et al. Addressing ceiling effects in health status measures: a comparison of techniques applied to measures for people with HIV disease. Health Serv Res. 2008;43:327–39.PubMedPubMedCentralCrossRef
27.
go back to reference Li L, Fu AZ. Some methodological issues with the analysis of preference-based EQ-5D index score. Health Serv Outcomes Res Method. 2009;9:162–76.CrossRef Li L, Fu AZ. Some methodological issues with the analysis of preference-based EQ-5D index score. Health Serv Outcomes Res Method. 2009;9:162–76.CrossRef
28.
go back to reference Jalkanen K, Aarnio E, Lavikainen P, Jauhonen H-M, Enlund H, Martikainen J. Impact of type 2 diabetes treated with non-insulin medication and number of diabetes-coexisting diseases on EQ-5D-5 L index scores in the Finnish population. Health Qual Life Outcomes. 2019;17:117.PubMedPubMedCentralCrossRef Jalkanen K, Aarnio E, Lavikainen P, Jauhonen H-M, Enlund H, Martikainen J. Impact of type 2 diabetes treated with non-insulin medication and number of diabetes-coexisting diseases on EQ-5D-5 L index scores in the Finnish population. Health Qual Life Outcomes. 2019;17:117.PubMedPubMedCentralCrossRef
29.
go back to reference Belotti F, Deb P, Manning WG, Norton EC. twopm: Two-part models. Stata J. 2015;15:3–20.CrossRef Belotti F, Deb P, Manning WG, Norton EC. twopm: Two-part models. Stata J. 2015;15:3–20.CrossRef
30.
go back to reference Janssen MF, Bonsel GJ, Luo N. Is EQ-5D-5L better than EQ-5D-3L? A head-to-head comparison of descriptive systems and value sets from seven countries. Pharmacoeconomics. 2018;36:675–97.PubMedPubMedCentralCrossRef Janssen MF, Bonsel GJ, Luo N. Is EQ-5D-5L better than EQ-5D-3L? A head-to-head comparison of descriptive systems and value sets from seven countries. Pharmacoeconomics. 2018;36:675–97.PubMedPubMedCentralCrossRef
31.
go back to reference Vitale MG, Levy DE, Johnson MG, et al. Assessment of quality of life in adolescent patients with orthopaedic problems: are adult measures appropriate? J Pediatr Orthop. 2001;21:622–8.PubMedCrossRef Vitale MG, Levy DE, Johnson MG, et al. Assessment of quality of life in adolescent patients with orthopaedic problems: are adult measures appropriate? J Pediatr Orthop. 2001;21:622–8.PubMedCrossRef
32.
go back to reference Noyes J, Edwards R. EQ-5D for the assessment of health-related quality of life and resource allocation in children: a systematic methodological review. Value Health. 2011;14:1117–29.CrossRef Noyes J, Edwards R. EQ-5D for the assessment of health-related quality of life and resource allocation in children: a systematic methodological review. Value Health. 2011;14:1117–29.CrossRef
33.
go back to reference Brussoni M, Kruse S, Walker K. Validity and reliability of the EQ-5D-3L™ among a paediatric injury population. Health Qual of Life Outcomes. 2013;11:157.CrossRef Brussoni M, Kruse S, Walker K. Validity and reliability of the EQ-5D-3L™ among a paediatric injury population. Health Qual of Life Outcomes. 2013;11:157.CrossRef
34.
go back to reference Thavorncharoensap M, Torcharus K, Nuchprayoon I, Riewpaiboon A, Indaratna K, Ubol B-O. Factors affecting health-related quality of life in Thai children with thalassemia. BMC Hematol. 2010;10:1.CrossRef Thavorncharoensap M, Torcharus K, Nuchprayoon I, Riewpaiboon A, Indaratna K, Ubol B-O. Factors affecting health-related quality of life in Thai children with thalassemia. BMC Hematol. 2010;10:1.CrossRef
35.
go back to reference Seyedifar M, Dorkoosh FA, Hamidieh AA, 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.PubMedPubMedCentral Seyedifar M, Dorkoosh FA, Hamidieh AA, 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.PubMedPubMedCentral
36.
go back to reference Deb P, Norton EC, Manning WG. Health econometrics using Stata. College Station: Stata Press; 2017. Deb P, Norton EC, Manning WG. Health econometrics using Stata. College Station: Stata Press; 2017.
37.
go back to reference Osborne RH, Lourenço RDA, Dalton A, et al. Quality of life related to oral versus subcutaneous iron chelation: a time trade-off study. Value Health. 2007;10:451–6.PubMedCrossRef Osborne RH, Lourenço RDA, Dalton A, et al. Quality of life related to oral versus subcutaneous iron chelation: a time trade-off study. Value Health. 2007;10:451–6.PubMedCrossRef
38.
go back to reference Karnon J, Tolley K, Oyee J, Jewitt K, Ossa D, Akehurst R. Cost-utility analysis of deferasirox compared to standard therapy with desferrioxamine for patients requiring iron chelation therapy in the United Kingdom. Curr Med Res Opin. 2008;24:1609–21.PubMedCrossRef Karnon J, Tolley K, Oyee J, Jewitt K, Ossa D, Akehurst R. Cost-utility analysis of deferasirox compared to standard therapy with desferrioxamine for patients requiring iron chelation therapy in the United Kingdom. Curr Med Res Opin. 2008;24:1609–21.PubMedCrossRef
39.
go back to reference Delea TE, Sofrygin O, Thomas SK, Baladi J-F, Phatak PD, Coates TD. Cost effectiveness of once-daily oral chelation therapy with deferasirox versus infusional deferoxamine in transfusion-dependent thalassaemia patients. Pharmacoeconomics. 2007;25:329–42.PubMedCrossRef Delea TE, Sofrygin O, Thomas SK, Baladi J-F, Phatak PD, Coates TD. Cost effectiveness of once-daily oral chelation therapy with deferasirox versus infusional deferoxamine in transfusion-dependent thalassaemia patients. Pharmacoeconomics. 2007;25:329–42.PubMedCrossRef
40.
go back to reference Fryback DG, Dasbach EJ, Klein R, et al. The Beaver Dam Health Outcomes Study: initial catalog of health-state quality factors. Med Decis Making. 1993;13:89–102.PubMedCrossRef Fryback DG, Dasbach EJ, Klein R, et al. The Beaver Dam Health Outcomes Study: initial catalog of health-state quality factors. Med Decis Making. 1993;13:89–102.PubMedCrossRef
41.
go back to reference Lubetkin EI, Jia H, Franks P, Gold MR. Relationship among sociodemographic factors, clinical conditions, and health-related quality of life: examining the EQ-5D in the US general population. Qual Life Res. 2005;14:2187–96.PubMedCrossRef Lubetkin EI, Jia H, Franks P, Gold MR. Relationship among sociodemographic factors, clinical conditions, and health-related quality of life: examining the EQ-5D in the US general population. Qual Life Res. 2005;14:2187–96.PubMedCrossRef
42.
go back to reference Delea TE, Edelsberg J, Sofrygin O, et al. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion. 2007;47:1919–29.PubMedCrossRef Delea TE, Edelsberg J, Sofrygin O, et al. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion. 2007;47:1919–29.PubMedCrossRef
43.
go back to reference Abetz L, Baladi J-F, Jones P, Rofail D. The impact of iron overload and its treatment on quality of life: results from a literature review. Health Qual Life Outcomes. 2006;4:73.PubMedPubMedCentralCrossRef Abetz L, Baladi J-F, Jones P, Rofail D. The impact of iron overload and its treatment on quality of life: results from a literature review. Health Qual Life Outcomes. 2006;4:73.PubMedPubMedCentralCrossRef
44.
go back to reference Porter J, Bowden DK, Economou M, et al. Health-related quality of life, treatment satisfaction, adherence and persistence in β-thalassemia and myelodysplastic syndrome patients with iron overload receiving deferasirox: results from the EPIC clinical trial. Anemia. 2012;2012:297641.PubMedPubMedCentralCrossRef Porter J, Bowden DK, Economou M, et al. Health-related quality of life, treatment satisfaction, adherence and persistence in β-thalassemia and myelodysplastic syndrome patients with iron overload receiving deferasirox: results from the EPIC clinical trial. Anemia. 2012;2012:297641.PubMedPubMedCentralCrossRef
45.
go back to reference Scalone L, Mantovani LG, Krol M, et al. Costs, quality of life, treatment satisfaction and compliance in patients with β-thalassemia major undergoing iron chelation therapy: the ITHACA study. Curr Med Res Opin. 2008;24:1905–17.PubMedCrossRef Scalone L, Mantovani LG, Krol M, et al. Costs, quality of life, treatment satisfaction and compliance in patients with β-thalassemia major undergoing iron chelation therapy: the ITHACA study. Curr Med Res Opin. 2008;24:1905–17.PubMedCrossRef
46.
go back to reference Jaeschke R, Singer J, Guyatt GH. Measurement of health status: ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10:407–15.PubMedCrossRef Jaeschke R, Singer J, Guyatt GH. Measurement of health status: ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10:407–15.PubMedCrossRef
Metadata
Title
EQ-5D-3L health state utility values in transfusion-dependent thalassemia patients in Malaysia: a cross-sectional assessment
Authors
Asrul Akmal Shafie
Irwinder Kaur Chhabra
Jacqueline Hui Yi Wong
Noor Syahireen Mohammed
Publication date
01-12-2021
Publisher
BioMed Central
Keyword
Thalassemia
Published in
Health and Quality of Life Outcomes / Issue 1/2021
Electronic ISSN: 1477-7525
DOI
https://doi.org/10.1186/s12955-020-01645-0

Other articles of this Issue 1/2021

Health and Quality of Life Outcomes 1/2021 Go to the issue