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      Healthcare resource utilization and direct costs of transfusion-dependent thalassemia patients in Dubai, United Arab Emirates: a retrospective cost-of-illness study

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          Abstract

          Background

          Patients with transfusion-dependent thalassemia (TDT) require lifelong blood transfusions and iron chelation therapy. Thus, patients afflicted with TDT often have to undergo blood transfusion and iron chelation therapy, which causes a major economic burden on them. However, this topic has not been reported in Dubai, United Arab Emirates (UAE). Hence, this study aimed to evaluate healthcare resource utilization and associated direct costs related to patients with TDT in Dubai, UAE.

          Methods

          For this study, a retrospective prevalence-based cost-of-illness analysis based on the UAE healthcare system and patient perspectives was conducted among patients with TDT treated at the Dubai Thalassemia Center in 2019. Information regarding healthcare resource utilization and direct medical costs was collected from the billing system connected to the electronic medical record system. Patients and their families were interviewed for direct non-medical cost estimations.

          Results

          A total of 255 patients with TDT were included in the study. The mean annual direct medical cost was estimated at AED 131,156 (USD 35,713) (95% CI: 124,735 – 137,578). The main driver of the medical cost for the participants as iron chelation therapy AED 78,372 (95% CI: 72,671 – 84,074) (59.8%), followed by blood transfusions, which accounted for AED 34,223 (95% CI: 32,854 – 35,593) 26.1% of the total direct medical costs. The mean annual direct non-medical costs was AED 2,223 (USD 605) (95% CI: 1,946 – 2,500). Age ( p < 0.001), severe serum ferritin levels ( p = 0.016), the presence of complications ( p < 0.001), and the type of iron chelation therapy ( p < 0.001) were significant predictors of higher direct medical costs incurred by the participants.

          Conclusion

          Transfusion-dependent thalassemia poses a substantial economic burden on the healthcare system, patients, and their families. Our results show that the highest medical cost proportion was due to iron chelation therapy. In this regard, efforts must be made to improve the patients’ acceptance and satisfaction with their iron chelation therapy to increase their compliance and improve the effectiveness of treatment, which could play an essential role in controlling the economic burden of this disease. Moreover, greater support is essential for families that suffer catastrophic out-of-pocket expenses.

          Supplementary Information

          The online version contains supplementary material available at 10.1186/s12913-022-07663-6.

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          Most cited references39

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          The Hemoglobin E Thalassemias

          Hemoglobin E (HbE) is an extremely common structural hemoglobin variant that occurs at high frequencies throughout many Asian countries. It is a β-hemoglobin variant, which is produced at a slightly reduced rate and hence has the phenotype of a mild form of β thalassemia. Its interactions with different forms of α thalassemia result in a wide variety of clinical disorders, whereas its coinheritance with β thalassemia, a condition called hemoglobin E β thalassemia, is by far the most common severe form of β thalassemia in Asia and, globally, comprises approximately 50% of the clinically severe β-thalassemia disorders.
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            Effect of transfusional iron intake on response to chelation therapy in beta-thalassemia major.

            The success of chelation therapy in controlling iron overload in patients with thalassemia major is highly variable and may partly depend on the rate of transfusional iron loading. Using data from the 1-year phase III study of deferasirox, including volumes of transfused red blood cells and changes in liver iron concentration (LIC) in 541 patients, the effect of iron loading on achieving neutral or negative iron balance was assessed in patients receiving different doses of deferasirox and the comparator deferoxamine. After dose adjustment, reductions in LIC after 1 year of deferasirox or deferoxamine therapy correlated with transfusional iron intake. At a deferasirox dose of 20 mg/kg per day, neutral or negative iron balance was achieved in 46% and 75% of patients with the highest and lowest transfusional iron intake, respectively; 30 mg/kg per day produced successful control of iron stores in 96% of patients with a low rate of transfusional iron intake. Splenectomized patients had lower transfusional iron intake and greater reductions in iron stores than patients with intact spleens. Transfusional iron intake should be monitored on an ongoing basis in thalassemia major patients, and the rate of transfusional iron loading should be considered when choosing the appropriate dose of an iron-chelating agent. This study is registered at http://clinicaltrials.gov as NCT00061750.
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              A scoring system for the classification of beta-thalassemia/Hb E disease severity.

              Beta-thalassemia intermediate patients show a remarkable clinical heterogeneity. We examined the phenotypic diversity of 950 beta-thalassemia/Hb E patients in an attempt to construct a system for classifying disease severity. A novel scoring system based on six independent parameters, hemoglobin level, age at disease presentation, age at receiving first blood transfusion, requirement for transfusion, spleen size, and growth and development, was able to separate patients into three distinctive severity categories: mild, moderate, and severe courses. This system, therefore, can increase the accuracy of studies of genotype-phenotype interactions and facilitate decisions for appropriate patient management. Copyright 2008 Wiley-Liss, Inc.
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                Author and article information

                Contributors
                Shaikha34@gmail.com
                Journal
                BMC Health Serv Res
                BMC Health Serv Res
                BMC Health Services Research
                BioMed Central (London )
                1472-6963
                5 March 2022
                5 March 2022
                2022
                : 22
                : 304
                Affiliations
                [1 ]GRID grid.444522.1, ISNI 0000 0004 1808 226X, School of Health and Environment Studies, , Hamdan Bin Mohammed Smart University, ; Dubai, UAE
                [2 ]GRID grid.508740.e, ISNI 0000 0004 5936 1556, Department of Health Management, , Istinye University, ; Istanbul, Turkey
                Author information
                http://orcid.org/0000-0002-1981-4544
                Article
                7663
                10.1186/s12913-022-07663-6
                8897869
                35248046
                697738c0-5be3-44f1-a058-42db44a15df3
                © The Author(s) 2022

                Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver ( http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data.

                History
                : 25 July 2021
                : 22 February 2022
                Categories
                Research Article
                Custom metadata
                © The Author(s) 2022

                Health & Social care
                healthcare resource utilization,direct costs,transfusion-dependent thalassemia,health policy,dubai,united arab emirates

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