Abstract

Thalassaemia is a common genetic disorder in Malaysia where the mainstay of supportive treatment is regular blood transfusion accompanied with iron-chelating therapy (ICT). Transfusion dependent thalassaemia (TDT) is defines as patient who requires blood transfusion >100mL/Kg annually. There are about 4500 patients in Malaysia who are suffering from TDT, however limited data is available on economic burden of this disease. Therefore, this study is designed to determine the economic burden of disease from a societal perspective. Lifetime healthcare cost (TC2) was simulated using Markov model. TC2 consists of cost of ICT, blood transfusion, routine blood and instrumental monitoring and management of iron overload (IOL) complications. Lifetime patient and family healthcare expenditure (TC3) was estimated through cross-sectional health survey approach. Survey was conducted using two-stage sampling method in 13 thalassaemia centers covering all regions in Malaysia. TDT patients who were aged 2 and prescribed with ICT were interviewed using TDT health utilization survey form (TDTHUS). TDTHUS collect information on out-of-pocket (OOP) expenditure, transportation cost and productivity losses. Total lifetime TDT cost (TC1) = TC2 + TC3. A TDT patient expected to incur TC2 of MYR 2,284,116 (US$ 561,208). ICT is the main cost driver accounting for 56.9 % of the total cost followed by blood transfusion cost, 13.1%. While, estimated TC3 was MYR185,021 (USD 45,458). Therefore, estimated TC1 of a TDT patient was RM 2,469,128(USD 606,665). Sensitivity analyses showed that if all patients were prescribed Deferasirox (DFX) for lifetime, total healthcare cost would increase by approximately 65%. The cost of TDT would vary by less than 5 % from the base-case value with changes of volume and frequency of blood transfusion. The lifetime cost per TDT patient was estimated and this would be useful for national planning and applied for prevention intervention and economic evaluation of treatment.

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