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

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

doi:10.1186/s12955-020-01645-0

Abstract

PurposeThere 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.MethodsA 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.ResultsA 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.ConclusionLower 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.

Highlights

Thalassemia is the most prevalent hereditary hematologic disorder worldwide [1, 2]
This study aims to derive health state utility values (HSUVs) based on the iron chelating agent regimen used and the various iron overload complications associated with the condition
13.0% of the sampled population had any history of a serious adverse event with iron chelating therapy

Summary

Introduction

Thalassemia is the most prevalent hereditary hematologic disorder worldwide [1, 2]. Approximately 1.5% of the global population are estimated to be carriers of the β-thalassemia, with higher incidence in populations from the Mediterranean basin, Middle East, IndianShafie et al Health Qual Life Outcomes (2021) 19:10 followed by iron chelating therapy to remove the excess iron resulting from the transfusion [8]. Several studies have been conducted in Malaysia to assess the HRQoL of TDT patients between 2006 to 2013 Most of these studies utilized instruments such as the PedsQL 4.0 Generic Core Scales (GCS) [12,13,14] and Medical Outcomes Study Short Form 36-item (MOS SF-36) [15]. They showed that the diagnosis of TDT negatively impacts the HRQoL of patients. The long-term use of medications, frequent hospital visits and fatigue brought about by the chronic anaemia disrupts their ability to function and can cause emotional distress [16] These studies have been limited to the paediatric and adolescent TDT population. These instruments quantify a health state in terms of functioning and symptoms, its scores are not weighted neither is there ‘value’ attached to it, making it difficult to use the outcomes in economic evaluations

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