Long-term results from the AGILE study of azacitidine plus ivosidenib vs placebo in newly diagnosed IDH1-mutated AML.

This post hoc analysis reports long-term follow-up results from AGILE after a median follow-up of 28.6 months.

这项事后分析报告了AGILE研究经过28.6个月中位随访期后的长期随访结果。

Overall, 148 patients were randomized to receive ivosidenib-azacitidine (n = 73) or placebo-azacitidine (n = 75).

总体而言，148名患者被随机分配接受伊沃替尼-阿扎胞苷（n = 73）或安慰剂-阿扎胞苷（n = 75）。

Median OS was significantly longer with ivosidenib (29.3 months; 95% confidence interval[CI], 13.2 to not reached) than with placebo (7.9 months; 95% CI, 4.1-11.3; hazard ratio, 0.42 [95% CI, 0.27-0.65]; P < .0001).

伊沃替尼组的中位总生存期显著长于安慰剂组（伊沃替尼组为29.3个月；95%置信区间[CI]，13.2至未达到；安慰剂组为7.9个月；95% CI, 4.1-11.3；风险比，0.42 [95% CI, 0.27-0.65]；P < .0001）。

Hematologic recovery was faster, more durable, and conversion to transfusion independence (53.8% vs 17.1%; P = .0004) was more common with ivosidenib than with placebo.

与安慰剂相比，接受艾伏尼布治疗的患者血液恢复更快、更持久，且达到输血独立（53.8%对比17.1%；P = .0004）的情况更为常见。

Of 33 ivosidenib-treated patients evaluable for molecular measurable residual disease (MRD), 10 converted to MRD negativity.

在33名接受艾伏尼布治疗并可用于分子可测量残余病灶（MRD）评估的患者中，有10名患者转为MRD阴性。

Although OS did not differ significantly between MRD-negative and MRD-positive responders at the 0.1% variant allele frequency (VAF) threshold, MRD-negative patients had numerically longer survival. MRD status appeared more predictive of long-term OS when an exploratory 1% VAF threshold was applied.

尽管在0.1%的变异等位基因频率（VAF）阈值下，MRD阴性和MRD阳性反应者的总生存（OS）没有显著差异，但MRD阴性患者的生存期在数值上更长。当应用探索性的1% VAF阈值时，MRD状态似乎更能预测长期总生存。

MRD response was not associated with IDH1 variant, VAF, inferred clonality, or number of baseline comutations.

MRD反应与IDH1变异、VAF、推断的克隆性或基线共突变的数量无关。

The previously reported safety profile was maintained.

先前报告的安全性特征得到了保持。

These long-term efficacy and safety results confirm the benefit of ivosidenib-azacitidine in this challenging-to-treat population and support its use as a standard of care with the longest reported survival benefit for intensive chemotherapy-ineligible patients with IDH1-mutated AML.

这些长期疗效和安全性结果证实了伊沃替尼-阿糖胞苷在这一难以治疗的人群中的益处，并支持将其作为标准治疗，为无法接受强化疗的IDH1突变型急性髓细胞性白血病患者提供了迄今为止报告的最长生存益处。

This trial was registered at www.ClinicalTrials.gov as #NCT03173248.

该试验已在www.ClinicalTrials.gov注册，注册号为#NCT03173248。