OBJECTIVES: To estimate the disease burden of Waldenstrom’s macroglobulinemia (WM) under the current treatment pattern in China.

METHODS: A decision-analytic model was constructed to simulate the Chinese patients with WM 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. A 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 WM.

RESULTS: The Chinese WM patients were associated with reduced average overall survival by 5.715 years (9.768 years vs. 15.483 years), reduced average QALY by 4.788 QALY (7.894 QALY vs. 12.682 QALY), and increased average life-time direct medical costs by ¥564,159 (¥663,760 vs. ¥99,601). The reduced QALY associated with WM was sensitive to patient age (change: 2.012 QALY), best supportive care rate (change: 1.044 QALY), and treatment effect of fludarabine (change: -0.963 QALY). The increased lifetime direct medical costs associated with WM was sensitive to best supportive care rate (change: ¥-137,001), patient age (change: ¥-99,528), mortality of progressive disease (change: ¥-93,018), and drug acquisition costs of bortezomib (change: ¥78,496). The probabilistic sensitivity analysis with 10,000 Monte Carlo simulations estimated the median and 95% credible interval of reduced QALY ( -4.067 QALY, -5.538 QALY to -2.819 QALY) and increased lifetime direct medical costs (¥566,592, ¥466,262 to ¥713,860).

CONCLUSIONS: The disease burden of WM in Chinese patients was characterized by reduced overall survival, reduced QALY, and increased medical costs that were sensitive to patient age, best supportive care rate, treatment effects of fludarabine, mortality of progressive disease, and drug acquisition costs of bortezomib.