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. 2024 Jul 11;22(1):54.
doi: 10.1186/s12955-024-02265-8.

Appropriateness of the EQ-5D-5L in capturing health-related quality of life in individuals with transfusion-dependent β-thalassemia: a mixed methods study

Adriana Boateng-Kuffour  1   2 , Hanna Skrobanski  3 , Jennifer Drahos  4 , Puja Kohli  4 , Katie Forster  3 , Sarah Acaster  3 , Zahra Pakbaz  5 , Nanxin Li  4 , Kate Williams  3
Affiliations

Appropriateness of the EQ-5D-5L in capturing health-related quality of life in individuals with transfusion-dependent β-thalassemia: a mixed methods study

Adriana Boateng-Kuffour et al. Health Qual Life Outcomes. .

Abstract

Background: Individuals with transfusion-dependent β-thalassemia (TDT) experience symptoms and functional impacts that reduce their health-related quality of life. However, EQ-5D-derived health utility index scores in TDT often indicate good HRQoL, suggesting the EQ-5D may not adequately capture the impact of TDT. This study explored the disease and treatment burden of TDT and examined the appropriateness of the EQ-5D-5L descriptive system (DS) in measuring HRQoL in TDT.

Methods: Adults with TDT in the United Kingdom, United States, and France completed a background questionnaire and EQ-5D-5L DS, followed by 60-minute semi-structured interviews on symptoms and HRQoL impacts of TDT (concept elicitation) and appropriateness of EQ-5D-5L DS (cognitive debrief). Transcribed interviews were analyzed using thematic and content analyses. The relationship between TDT symptoms and impacts were summarized in a conceptual model. EQ-5D-5L DS was mapped to concepts identified in the qualitative data to assess its capture of HRQoL concepts. Participants' EQ-5D-5L DS scores were compared to their qualitative descriptions for each dimension to assess their concordance.

Results: Thirty participants in the United States (n = 14 [46.7%]), United Kingdom. (n = 12 [40.0%]), and France (n = 4 [13.3%]) completed the study (73.3% female; mean age = 28.4 years [standard deviation (SD) = 5.1]; mean annual red blood cell transfusion [RBCT] frequency = 18.4 [SD = 7.6]). Participants reported TDT symptoms and impacts on HRQoL, all fluctuating across the RBCT cycle. EQ-5D-5L DS did not fully capture 11 of 16 (68.8%) HRQoL concepts reported. Most participants (n = 20/27 [74.1%]) reported that EQ-5D-5L DS did not capture important aspects of living with TDT, and 42.9% (n = 12/28) reported negative/neutral overall impressions of EQ-5D-5L DS. The highest degree of discordance between participants' qualitative data and EQ-5D-5L DS dimension scores was observed with mobility (42.3%) and self-care (34.6%), where the qualitative descriptions relating to these dimensions were worse than their quantitative scores.

Conclusion: Current findings suggest that EQ-5D-5L DS lacks content validity and the derived health utility index score may not fully represent the burden of disease in TDT.

Keywords: Content validity; EQ-5D-5L DS; Health state utility; Health-related quality of life; Mixed methods analysis; Transfusion-dependent β-thalassemia.

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Conflict of interest statement

AB-K, JD, PK, and NL are employees of Vertex Pharmaceuticals Incorporated and may hold stock or stock options in the company. HS, KF, and KW are employees of Acaster Lloyd Consulting and may hold stock or stock options in the company. SA is a company director of Acaster Lloyd Consulting. ZP has received research grants from Amgen, Pfizer, Novo Nordisk, Global Blood Therapeutics, Farma, and Novartis; consulting fees from Amgen, Dova, Global Blood Therapeutics, Guidepoint, Novartis, Sanofi, Sobi, and Vertex Pharmaceuticals Incorporated; has been a speaker for Dova, Global Blood Therapeutics, and Terumo; and has acted as a continuing medical education course director for the Cayenne Wellness Center and Children’s Foundation and planning committee member for their annual education symposium.

Figures

Fig. 1
Fig. 1
Conceptual model on the patient experience of TDT.TDT transfusion-dependent β-thalassemia
Fig. 2
Fig. 2
Coverage of identified concepts across the five EQ-5D-5L dimensionsDS descriptive system
Fig. 3
Fig. 3
Degree of discordance between participants’ EQ-5D-5L DS quantitative scores and qualitative data. DS descriptive system
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