The management of beta thalassemia in the United Kingdom: Healthcare costs and outcomes over a 50 year period

This study aimed to calculate the costs of treating β-thalassemia major (BTM) to the United Kingdom's National Health Service (NHS) over a patient's first 50 years of life, considering healthcare resource utilization, related expenses, and the resulting health outcomes. The focus was on incidence-based costs.

Information obtained from a systematic review of published literature, and clinicians involved in managing BTM in the United Kingdom was used as the basis for this modeling study.To evaluate the healthcare management of BTM over five decades, a state transition model was built. Its purpose was to determine the incidence-based economic burden that BTM places on the NHS, as well as the impact on the health status of patients, measured in quality-adjusted life-years (QALYs) over a 50-year period.

The expected probability of survival at 50 years is 0.63. Of patients who survive, 33% are expected to be without any complication and the other 67% are expected to experience at least one complication. Patients' health status over this period was estimated to be a mean of 11.5 discounted QALYs per patient. Total healthcare expenditure attributable to managing BTM was estimated to be £483,454 ($720,201) at 2013/14 prices over 50 years. The cost of managing BTM could be potentially reduced by up to 37% if one in two patients had a bone marrow transplant, with an ensuing improvement in health-related quality of life.

This analysis offers the most accurate estimation currently available of NHS resource consumption and costs. It provides essential information for policymakers and budgetary decision-makers regarding this rare disease.

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