Improved survival with enasidenib versus standard of care in relapsed/refractory acute myeloid leukemia associated with IDH2 mutations using historical data and propensity score matching analysis.
Improved survival with enasidenib versus standard of care in relapsed/refractory acute myeloid leukemia associated with IDH2 mutations using historical data and propensity score matching analysis.
复制标题
DOI：
10.1002/cam4.4182
复制
发表时间：
2021-09
期刊：
影响因子：
4
通讯作者：
Stein EM
中科院分区：
文献类型：
--
作者：
de Botton S;Brandwein JM;Wei AH;Pigneux A;Quesnel B;Thomas X;Legrand O;Recher C;Chantepie S;Hunault-Berger M;Boissel N;Nehme SA;Frattini MG;Tosolini A;Marion-Gallois R;Wang JJ;Cameron C;Siddiqui M;Hutton B;Milkovich G;Stein EM
本研究评估了依那西尼和现行护理标准(SOC)疗法对不符合造血干细胞移植(HSCT)条件的复发性/难治性(R/R)急性髓系白血病(AML)和异柠檬酸脱氢酶2(IDH2)突变患者的相对生存益处。倾向评分匹配(PSM)分析比较了在I/II期AG221-C-001试验中每天服用100 mg依那西尼观察到的生存结果与从法国患者的真实图表回顾中获得的SOC结果。在匹配之前，与SoC相比，enasidenib(n=9195)与数字上改善的总存活率(OS)相关(n=1080；风险比[HR]，0.82；95%可信区间[CI]，0.61-1.11)。在匹配和调整协变量后(每组378人)，依那西尼组的死亡风险显著低于SOC组(HR，0.67；95%CI，0.47-0.97)。依那西尼的中位OS为9.26个月(95%CI，7.72-13.24)，SoC的中位OS为4.76个月(95%CI，3.81-8.21)。在进行的所有敏感性分析中，结果仍然强劲。PSM分析表明，与SOC相比，依那西尼显著延长了不符合HSCT条件的R/R AML患者和IDH2突变患者的生存时间。未来的前瞻性研究需要使用其他数据来源来验证这些发现，并评估依那西尼对其他治疗结果的比较疗效。与SOC相比，依那西尼可提高患者的总体存活率。Enasidenib可能是治疗IDH2突变相关的R/R急性髓系白血病患者的重要进展，这些患者不符合造血干细胞移植的条件。
The present study evaluated the relative survival benefits associated with enasidenib and current standard of care (SoC) therapies for patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) and an isocitrate dehydrogenase 2 (IDH2) mutation who are ineligible for hematopoietic stem cell transplantation (HSCT). Propensity score matching (PSM) analysis compared survival outcomes observed with enasidenib 100 mg daily in the phase I/II AG221‐C‐001 trial and SoC outcomes obtained from a real‐world chart review of patients in France. Before matching, enasidenib (n = 195) was associated with numerically improved overall survival (OS) relative to SoC (n = 80; hazard ratio [HR], 0.82; 95% confidence interval [CI], 0.61–1.11). After matching and adjusting for covariates (n = 78 per group), mortality risk was significantly lower with enasidenib than with SoC (HR, 0.67; 95% CI, 0.47–0.97). The median OS was 9.26 months for enasidenib (95% CI, 7.72–13.24) and 4.76 months for SoC (95% CI, 3.81–8.21). Results remained robust across all sensitivity analyses conducted. PSM analyses indicate that enasidenib significantly prolongs survival relative to SoC among patients with R/R AML and an IDH2 mutation who are ineligible for HSCT. Future prospective studies are needed to validate these findings using other data sources and to assess the comparative efficacy of enasidenib for other treatment outcomes. Overall survival was improved with enasidenib compared with SoC. Enasidenib may be an important advance in treatment for patients with R/R acute myeloid leukemia associated with IDH2 mutations who are ineligible for hematopoietic stem cell transplantation.
登录
查看更多内容
影响因子：
50.3
作者：
Figueroa ME;Abdel-Wahab O;Lu C;Ward PS;Patel J;Shih A;Li Y;Bhagwat N;Vasanthakumar A;Fernandez HF;Tallman MS;Sun Z;Wolniak K;Peeters JK;Liu W;Choe SE;Fantin VR;Paietta E;Löwenberg B;Licht JD;Godley LA;Delwel R;Valk PJ;Thompson CB;Levine RL;Melnick A
通讯作者：
Melnick A
影响因子：
12.8
作者：
De Kouchkovsky I;Abdul-Hay M
通讯作者：
Abdul-Hay M
影响因子：
5.8
作者：
Dunkler, Daniela;Ploner, Meinhard;Heinze, Georg
通讯作者：
Heinze, Georg
影响因子：
1.7
作者：
Austin, Peter C.
通讯作者：
Austin, Peter C.
影响因子：
3.8
作者：
Austin PC
通讯作者：
Austin PC