Objective: To investigate the efficacy and safety of Venetoclax combined with CACAG regimen in treatment of patients with refractory/relapse acute myeloid leukemia(R/R AML).
Methods: The study was a singlecenter prospective clinical trial. The enrolled patients met the criteria for R/R AML. Treatment included Azacidine(75 mg/m2，d 1-7), Ara-C (75-100 mg/m2, q12h, d 1-5), Aclacinomycin(20 mg d1,d3,d5), Chidamide(30 mg d1,d4), Venetoclax(100 mg d1, 200 mg d2, 400 mg d3-d14, in combination with Triazole Drug, reduced to 100 mg/d), and granulocyte colony-stimulating factor (300 μg /d until neutrophil recovery). The primary endpoint of observation was overall response rate after 1 course of treatment.
Results: A total of 19 patients were enrolled from January 2022 to April 2023. After 1 course of treatmen, the overall response rate was 81.3%(13/16), the CR rate was 68.8%(11/16), and the PR was 12.5%(2/16). Among the 11 patients who got CR/CRi, 8 cases achieved CRm (minimal residual disease negative CR) and 3 cases did not. As of March 27, 2023, the median follow-up time was 111(19-406) days. The six-month overall survival and progression-free survival rates were both 55.7%, the 1-year overall survival and progression-free survival rates were 46.4% and 47.7%, respectively. In addition, compared with the non-CRm group, CRm patients had a better PFS (377 days vs 111 days, P =0.046). Treatment-related adverse events were mainly 3-4 degrees of bone marrow suppression, complicated by various degrees of infection(n=12), hypokalemia(n=12) and hypocalcemia (n=10) and elevated liver enzymes (n=8), of which 3/4 degrees accounted for 47.4%(9/19).
Conclusion: The Venetoclax combined with CACAG regimen is an effective salvage therapy for patients with R/R AML, with high remission rate and safety profile.
题目: 维奈克拉联合CACAG方案治疗难治/复发急性髓系白血病的前瞻性临床研究.
目的: 探讨维奈克拉联合CACAG方案治疗难治/复发急性髓系白血病（R/R AML）的有效性和安全性。.
方法: 本研究为前瞻性单臂临床研究，入组患者均符合R/R AML诊断标准。采用阿扎胞苷（75 mg/m2，d 1-7）、阿糖胞苷（75-100 mg/m2, q12h, d 1-5）、阿柔比星（20 mg, d 1、3和5）、西达本胺（30 mg, d 1和4）、维奈克拉（100 mg d 1，200 mg d 2，400 mg d 3-14，与唑类药物联用，减量为100 mg/d）、重组人粒细胞集落刺激因子（300 μg/d，直至中性粒细胞恢复）的治疗方案，主要研究终点为1疗程后总反应率。.
结果: 自2022年1月至2023年4月共入组19例患者，1个疗程后总反应率为81.3%（13/16），完全缓解率为 68.8%（11/16），部分缓解率为12.5%（2/16），在11例获得完全缓解（CR/CRi）的患者中，达 CRm 8例，未达 CRm 3 例。截至 2023年3月27日，中位随访时间为 111（19-406）d。6个月的总生存率和无进展生存率均为55.7%；1年的总生存率和无进展生存率分别为46.4%和47.7% 。此外，相比于未达CRm组，CRm组患者具有较高的 PFS（377 vs 111 d，P =0.046）。治疗相关不良反应主要是3-4级骨髓抑制，并发不同程度的感染（n=12）、低钾血症（n=12）、低钙血症（n=10）和肝酶升高（n=8）等，其中3-4级占比为47.4%(9/19)。.
结论: 维奈克拉联合CACAG方案是 R/R AML 患者有效的挽救性治疗方案，缓解率高且安全性良好。.
Keywords: prospective clinical study; refractory; relapse; acute myeloid leukemia; venetoclax combined with CACAG regimen.