Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas.
Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas.
Cancer. 2021 Nov 15;127(22):4213-4220. doi: 10.1002/cncr.33814. Epub 2021 Aug 3.
Relapsed/refractory (R/R) acute myeloid leukemia (AML) has poor outcomes. Although lower-intensity venetoclax-containing regimens are standard for older/unfit patients with newly diagnosed AML, it is unknown how such regimens compare with intensive chemotherapy (IC) for R/R AML.
Outcomes of R/R AML treated with 10-day decitabine and venetoclax (DEC10-VEN) were compared with IC-based regimens including idarubicin with cytarabine, with or without cladribine, clofarabine, or fludarabine, with or without additional agents. Propensity scores derived from patient baseline characteristics were used to match DEC10-VEN and IC patients to minimize bias.
Sixty-five patients in the DEC10-VEN cohort were matched to 130 IC recipients. The median ages for the DEC10-VEN and IC groups were 64 and 58 years, respectively, and baseline characteristics were balanced between the 2 cohorts. DEC10-VEN conferred significantly higher responses compared with IC including higher overall response rate (60% vs 36%; odds ratio [OR], 3.28; P < .001), complete remission with incomplete hematologic recovery (CRi, 19% vs 6%; OR, 3.56; P = .012), minimal residual disease negativity by flow cytometry (28% vs 13%; OR, 2.48; P = .017), and lower rates of refractory disease. DEC10-VEN led to significantly longer median event-free survival compared with IC (5.7 vs 1.5 months; hazard ratio [HR], 0.46; 95% CI, 0.30-0.70; P < .001), as well as median overall survival (OS; 6.8 vs 4.7 months; HR, 0.56; 95% CI, 0.37-0.86; P = .008). DEC10-VEN was independently associated with improved OS compared with IC in multivariate analysis. Exploratory analysis for OS in 27 subgroups showed that DEC10-VEN was comparable with IC as salvage therapy for R/R AML.
DEC10-VEN represents an appropriate salvage therapy and may offer better responses and survival compared with IC in adults with R/R AML.
复发/难治性（R/R）急性髓系白血病（AML）预后较差。虽然对于新诊断的 AML 中年龄较大/不适合的患者，采用低强度维奈克拉联合治疗方案已成为标准治疗方法，但对于 R/R AML，此类方案与强化化疗（IC）相比的疗效如何尚不清楚。
比较了 10 天阿扎胞苷联合维奈克拉（DEC10-VEN）治疗 R/R AML 的结果与包括阿糖胞苷联合柔红霉素、阿糖胞苷联合 cladribine、clofarabine 或 fludarabine 以及联合或不联合其他药物的 IC 治疗方案。通过患者基线特征得出的倾向评分用于匹配 DEC10-VEN 和 IC 患者，以尽量减少偏倚。
DEC10-VEN 队列中的 65 例患者与 130 例 IC 接受者相匹配。DEC10-VEN 和 IC 组的中位年龄分别为 64 岁和 58 岁，两组的基线特征平衡。与 IC 相比，DEC10-VEN 诱导的反应更高，包括更高的总缓解率（60% vs 36%；优势比 [OR]，3.28；P<0.001）、不完全血液学恢复的完全缓解（CRi，19% vs 6%；OR，3.56；P=0.012）、流式细胞术检测微小残留病灶阴性（28% vs 13%；OR，2.48；P=0.017）和较低的难治性疾病发生率。与 IC 相比，DEC10-VEN 显著延长了中位无事件生存期（5.7 个月 vs 1.5 个月；风险比 [HR]，0.46；95%置信区间，0.30-0.70；P<0.001）和中位总生存期（OS；6.8 个月 vs 4.7 个月；HR，0.56；95%置信区间，0.37-0.86；P=0.008）。多变量分析表明，DEC10-VEN 与 OS 改善独立相关。在 27 个亚组的 OS 探索性分析中，与 IC 相比，DEC10-VEN 作为 R/R AML 的挽救性治疗是相当的。
与 IC 相比，DEC10-VEN 作为 R/R AML 的挽救性治疗方案具有更好的疗效和生存获益。
