Abstract

To estimate the disease burden of Chronic Myeloid Leukemia (CML) under current treatment pattern in China. A decision-analytic model was constructed to simulate the Chinese patients with CML and the age and gender-matched Chinese general population over lifetime time horizon for the differences in overall survival, quality-adjusted life years (QALY), and lifetime direct medical costs. Literature search was conducted to identify appropriate evidence to estimate the model variables. Base case analysis and sensitivity analysis were conducted for the point estimations and uncertainty of the disease burden of CML. The Chinese CML patients were associated with reduced average overall survival by 6.878 years (26.555 years vs. 33.433 years), reduced average QALY by 7.390 QALY (23.281 QALY vs. 30.671 QALY), and increased average life-time direct medical costs by ¥837,811 (¥1,014,394 vs. ¥176,583) under current treatment pattern. The reduced QALY associated with CML was highly sensitive to first-line treatment effect of imatinib for the chronic phase of CML (change: 1.818 QALY), utility of disease remission (change:1.809 QALY), the rate of allogeneic stem cell transplantation (difference: 1.624 QALY), and best supportive care rate (difference: -1.082). The lifetime direct medical costs associated with CML was highly sensitive to the rate of first-line imatinib treatment (change: ¥-141,906), mortality of blast phase of CML (change: ¥-130,926), and effects of first-line imatinib treatment (change: ¥-91,115). The probabilistic sensitivity analysis with 5,000 Monte Carlo simulations estimated the median and 95% credible interval of reduced QALY ( -7.399 QALY, -8.555 to -6.338 QALY) and increased lifetime direct medical costs (¥844,685, ¥748,593 to ¥964,283). The disease burden of CML in Chinese patients was mainly characterized with reduced overall survival, reduced QALY, and increased medical costs that were highly sensitive to first-line treatment with imatinib, stem cell transplantation rate, and mortality of blast phase of CML.

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