Abstract

Background: β-thalassemia is a common disorder in Turkey relative to many other countries. To our knowledge, there are no recent data in the literature on the direct medical care costs associated with β-thalassemia. We conducted a β-thalassemia treatment center survey and chart review to estimate the numbers of patients with transfusion-dependent (TD) or non-transfusion-dependent (NTD) β-thalassemia currently receiving care and the associated direct medical care costs in Turkey.

Methods: A preliminary list of β-thalassemia-treating centers was generated from the IMS Health audit of hospitals and treatment centers across Turkey based on the use of iron chelation therapy (ICT) agents. One or more hematologists or pediatricians in each potential hospital ward were contacted to determine the number of patients managed in their ward with β-thalassemia major, TD β-thalassemia intermedia, and NTD β-thalassemia intermedia. TD status was determined using local definitions by each physician. A geographically stratified sample of 60 physicians was then asked to complete a web-based questionnaire on healthcare resource utilization for the four most recent patients under their care: one patient with β-thalassemia major, one with TD β-thalassemia intermedia, one with NTD β-thalassemia intermedia, and one patient at their discretion. Healthcare resource utilization focused on transfusions, ICT, laboratory work, β-thalassemia-associated complications, and inpatient hospital stay. The unit cost of each healthcare utilization was extracted from relevant literature and published government fee schedules. For prescription drug prices, IMS Health sales data in USD were referenced.

Results: We identified 107 treatment centers, 91 (85%) of which were successfully surveyed. A total of 2,637 TD β-thalassemia patients (2,059 with β-thalassemia major plus 578 with TD β-thalassemia intermedia) and 2,040 NTD β-thalassemia intermedia patients were reported. Taking into consideration the sampling frame, missing data, infrequent care for NTD β-thalassemia patients, and errors (including the fact that ICT can be used to manage conditions other than β-thalassemia), we estimated there are approximately 4,000 TD and 4,000 NTD β-thalassemia patients in Turkey. A total of 212 β-thalassemia patient records were reviewed (72 β-thalassemia major, 72 TD β-thalassemia intermedia, and 68 NTD β-thalassemia intermedia). Both the mean and median ages were about 20 years old, and the ages were similar between patients with TD and NTD β-thalassemia. For patients with β-thalassemia major, the total annual direct costs per patient were USD 14,360, of which ICT was the most expensive cost component at USD 9,919, followed by in-hospital stay (USD 1,786), transfusions (USD 1,718), complications (USD 800), and laboratory tests (USD 193). Patients with TD β-thalassemia intermedia incurred nearly identical medical care costs of USD 14,940, of which USD 10,301 was attributable to ICT. Not all TD β-thalassemia patients received transfusions at β-thalassemia treatment centers. For patients who did receive transfusions at β-thalassemia treatment centers (n = 85), patients with β-thalassemia major (n = 45) and TD β-thalassemia intermedia (n = 40) received on average 1.6 and 1.5 red blood cell units per month, respectively. Every patient with TD β-thalassemia received ICT, and deferasirox was the most commonly administered chelation treatment, prescribed to 91 of 144 patients. The annual direct cost per patient with NTD β-thalassemia intermedia was lower at USD 9,286, primarily owing to lower healthcare utilization of ICT and transfusions. Combining the costs of TD and NTD β-thalassemia treatment, we estimated that the total annual direct medical care cost was approximately USD 95,491,900 in Turkey, with TD β-thalassemia patients accounting for USD 58,347,900 (61.1%).

Conclusions: β-thalassemia remains a prevalent disease in Turkey where there are an estimated 4,000 TD and 4,000 NTD patients. The disease is associated with significant consumption of healthcare resources, with the total annual direct medical care cost estimated to be over USD 95 million. The costs reported in this study were likely underestimated, as many patients who received transfusions outside of β-thalassemia treatment centers were not captured in the chart review.

Disclosures

Antmen: Celgene: Honoraria, Other: Advisory for the project Burden of illness in thalassemia; Novartis: Other: Advisory board; Jazz: Other: Advisory board . Angelucci: Roche: Other: Advisory board: biosimilars; Celgene: Other: protocoll ACE 536 B-Thal 001: DMC Chair; Bluebird Bio: Other: Advisory board: Gene therapy in Thalassemia; Novartis Oncology: Other: Protocol Telesto: sterring committee Chair; Jazz: Other: Advisory board: AML; Novartis Oncology: Other: Advisory board: iron toxicity; Celgene: Honoraria, Other: Advisory: research project ; Novartis Oncology Swiss: Other: Invited speakers sponsored satellite meeting during . Losi: QuintilesIMS: Consultancy; Celgene: Other: Commissioned to carry out the data collection survey as specified in the abstract as an employee of QuintilesIMS. Burrows: QuintilesIMS: Consultancy; Celgene: Other: Commissioned to carry out the data collection survey as specified in the abstract as an employee of QuintilesIMS. Bartiromo: Celgene: Employment, Equity Ownership. Hu: Celgene: Employment, Equity Ownership.

Author notes

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Asterisk with author names denotes non-ASH members.