Clinical Effectiveness, Quality of Life and Costs of Treatment in Transfusion-Dependent Thalassemia Patients Undergoing Iron-Chelating Therapy; Deferiprone

Abstract

A major complication of long-term blood transfusion in thalassemia patients is a serious iron overload. Excessive iron deposits in major organs and eventually results in organ damage. Deferiprone (DFP) is an effective oral iron chelator that promotes iron excretion and prevents the progression of iron accumulation assessed by serum ferritin levels. To evaluate the overall impact of DFP in transfusion-dependent thalassemia patients, this study aimed to determine the clinical effectiveness, health-related quality of life (HRQOL) and treatment costs of DFP. A cross-sectional study was conducted among 50 transfusion-dependent thalassemia patients who received DFP at Maharaj Nakorn Chiang Mai Hospital. The data of clinical effectiveness and costs of treatment were collected from the questionnaire and retrospectively reviewed over a 12-month period from medical records and hospital database. HRQOL was assessed by using the questionnaire, EQ-5D-3L (Thai version). The result showed the mean serum ferritin level (µg/L) at baseline and final level of 1367±935 and 1386±541, respectively. The mean EQ-5D-3L and VAS scores were 0.80±0.16 and 81.9±14.0, respectively. The adherence to treatment was found to be poor and the common adverse events reported were headache, back pain, arthralgia, fever, elevated liver enzyme, nausea/vomiting, abdominal pain and diarrhea. The total annual direct costs of treatment were estimated to be 54,000 Thai Baht (THB)/patient. In conclusion, DFP was effective in maintaining serum ferritin level in transfusion-dependent thalassemia patients with reasonable costs of treatment. Although patients’ HRQOL assessments were favorable, adherence to treatment was poor with more adverse events reported.