国产阿扎胞苷治疗较高危骨髓增生异常综合征患者的疗效和安全性分析：多中心、前瞻性、单臂研究

OBJECTIVE: To evaluate the efficacy, safety, and pharmacokinetics of the generic azacitidine in Chinese patients with higher-risk myelodysplastic syndromes（MDS）. METHODS: Between October 2013 and 2016, 72 patients were eligible for enrollment at 9 sites from China received generic subcutaneous azacitidine 75 mg·m(−2)·d(−1) for 7 days per 28-day cycle, for ≥6 cycles. Pharmacokinetic blood samples were collected on day 1 of a single-dose. RESULTS: For each patient at cycle 6 or at the time of study discontinuation, whichever came first, the overall response rate, which included complete remission（CR）and partial remission（PR）, was 6.9％（5/72）, the rate of patients who had the best effect with CR or PR during the treatment was 12.5％（9/72）. Patients who were dependent on red-blood-cell transfusions and platelet transfusions at baseline became transfusion independent were 46.3％ （19/41） and 41.2％（7/17）, respectively. The median time of treatment was 6 cycles, and the median OS was 16.1 months（95％ CI 10.9–20.6 months）. For 36 patients（50％）received treatment at ≥6 cycles, and the median OS was 22.3 months（95％ CI 16.1–not evaluative）. Most common grade Ⅲ–Ⅳ hematologic treatment-emergent adverse events were neutropenia（55％）, leukopenia（47％）, and thrombocytopenia（61％）. Pharmacokinetic profiles were similar for generic and original azacitidine in Chinese patients. CONCLUSION: Generic azacitidine treatment was favorable and safe and can be used as a standard treatment for patients with higher-risk MDS.