Venetoclax联合低剂量阿糖胞苷治疗不耐受强化化疗的初治急性髓系白血病患者：一项Ⅲ期随机、安慰剂对照试验中中国队列结果

OBJECTIVE: To investigate the safety and efficacy of venetoclax with low-dosecytarabine（LDAC）in Chinese patients with acute myeloid leukemia（AML）who are unable to tolerateintensive induction chemotherapy. METHODS: Adults ≥ 18 years with newly diagnosed AML who wereineligible for intensive chemotherapy were enrolled in this international, randomized, double-blind, placebocontrolledtrial. Globally, patients（n＝211）were randomized 2∶1 to either venetoclax with LDAC orplacebo with LDAC in 28-d cycles, with LDAC on days 1–10. The primary endpoint was OS; thesecondary endpoints included response rates, event-free survival, and adverse events. RESULTS: A total of15 Chinese patients were enrolled（venetoclax arm, n＝9; placebo arm, n＝6）. The median age was 72years（range, 61–86）. For the primary analysis, the venetoclax arm provided a 38％ reduction in deathrisk compared with the placebo［hazard ratio（HR）, 0.62（95％ CI 0.12–3.07）］. An unplanned analysiswith an additional 6 months of follow-up demonstrated a median OS of 9.0 months for venetoclaxcompared with 4.1 months for placebo. The complete remission（CR）rates with CR with incomplete bloodcount recovery（CRi）were 3/9（33％）and 0/6（0％）, respectively. The most common non-hematologicadverse effects（venetoclax vs placebo）were hypokalemia［5/9（56％）vs 4/6（67％）］, vomiting［4/9（44％）vs 3/6（50％）］, constipation［2/9（22％）vs 4/6（67％）］, and hypoalbuminemia［1/9（11％）vs 4/6（67％）］. CONCLUSION: Venetoclax with LDAC demonstrated meaningful efficacy and a manageable safetyprofile in Chinese patients consistent with the observations from the global VIALE-C population, making itan important treatment option for patients with newly diagnosed AML who are otherwise ineligible forintensive chemotherapy.