OBJECTIVES: Multiple myeloma (MM) is a hematologic malignancy mainly affecting the elderly population. It is incurable and patients experience a considerable reduction of health-related-quality-of-life (HRQoL). Some newer therapies have shown better clinical effects but are more costly. Pharmacoeconomic studies on MM have been widely conducted overseas but local data was lacking. This study aimed to examine the cost-effectiveness of the treatments for MM in Macao, China. METHODS: A retrospective cost-effectiveness study with HRQoL assessment was conducted. Forty patients from the largest public hospital in Macao from 1997-2007 with confirmed MM were studied. Data for costs and treatment effects were extracted from patients’ medical records including disease characteristics, treatment regimens, medications used and treatment outcomes. The study was conducted from the perspective of a Macao public hospital. The European Organization for Research and Treatment of Cancer quality-of-life questionnaire (EORTC QLQ-C30) (version 3.0, Chinese-Hong Kong) was used for the HRQoL assessment. RESULTS: The standard-dose melphalan-based and vincristine-doxorubicin-dexamethasone-based regimens (VAD) were the most common treatment regimens. There were 24 and 10 patients in the melphalan-based and VAD-based group respectively. Six patients were not studied due to incomplete data. Patients in the melphalan-based group were at a more advanced age (70.4 ± 7.4 years; p<0.001). The melphalan-based group showed a lower overall treatment cost (MOP 29,231 versus MOP 44,831; p=0.521, 1 USD = 8 MOP), especially on inpatient medication cost (MOP 5,809 versus MOP 13,908; p=0.096). The VAD-based group showed better clinical outcomes than the melphalan-based group in terms of overall survival, progression-free survival and survival probability with time. The incremental cost-effectiveness ratio of VAD-based regimens compared with melphalan-based regimens was MOP 6,695 per life-year-gained. CONCLUSIONS: The results of this study suggest that the VAD-based regimens are very cost-effective according to the WHO recommended thresholds for cost-effectiveness in patients with MM in Macao.