Abstract
To evaluate demographic and clinical characteristics of transfusion-dependent beta-thalassemia (TDT) patients, the pattern of blood transfusion and iron-chelation therapy (ICT), healthcare-resource consumptions and healthcare direct costs in TDT patients. A retrospective study was carried out by integrating administrative databases of a pool of Italian Healthcare-Entities, with data reproportioned to the Italian population. Patients with ≥6 episodes of blood transfusion over a 12-month period together with ≥1 ICT prescription between 01/01/2010-31/12/2016, were included. Index-date was that of first blood transfusion within the inclusion period. Patients were characterized 12-month before index-date and followed-up from index-date to death or study-ending. Annual healthcare-resources/patient and direct healthcare-cost/patient [covered by NHS, for drugs, outpatients specialistic services (OSS), and hospitalizations] were evaluated during the follow-up. Overall, 2,137 TDT patients were estimated, with a mean age of 41.3 and 45% male. Previous hospitalizations were observed in 34% of patients. Anti-infectives drugs were the most prescribed (74%). During follow-up, hepatic diseases represented a complication in 38% of patients. Mean number of blood transfusions administrated over 12-month period was 11.9. Median time from first transfusion to first ICT prescription was 216 days. Concerning ICT treatments, 69% of patients were with the same ICT, while 26% and 5% presented 2-3 different types of ICT prescriptions, respectively. Mean annual number/patient of drug prescriptions was 23.4, of hospitalizations was 2.2 (median length of stay 82 days), and of OSS was 103.7. The overall annual direct healthcare-cost/patient averaged 20,343€ (9,832€ for drugs, 9,449€ for hospitalizations, and 1,062€ for OSS). This real-world study provided an in-depth characterization of the clinical features, pharmaco-utilization and economic burden of TDT patients, showing the high resource consumption and direct health-care costs for these patients. The mean age observed reflected the life-expectancy increment of TDT population reported in literature, suggesting that aging should be considered in the management of beta-thalassemia.
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