We sought to appraise the value of overall response and salvage chemotherapy, inclusive of allogeneic hematopoietic stem cell transplant (AHSCT), in primary refractory acute myeloid leukemia (prAML). For establishing consistency in clinical practice, the 2017 European LeukemiaNet (ELN) defines prAML as failure to attain CR after at least 2 courses of intensive induction chemotherapy. Among 60 consecutive patients (median age 63 years) correspondent with ELN-criteria for prAML, salvage was documented in 48 cases, 30/48 (63%) being administered intensive chemotherapy regimens and 2/48 consolidated with AHSCT as first line salvage. 13/48 (27%) attained response: CR, 7/13 (54%), CRi, 2/13 (15%), MLFS, 4/13 (31%). The CR/CRi rate was 9/48 (19%), with CR rate of 7/48 (15%). On univariate analysis, intermediate-risk karyotype was the only predictor of response (44% vs 17% in unfavorable karyotype; P = 0.04). Administration of any higher-dose (>1 g/m²) cytarabine intensive induction (P = 0.50), intensive salvage chemotherapy (P = 0.72), targeted salvage (FLT3 or IDH inhibitors) (P = 0.42), greater than 1 salvage regimen (P = 0.89), age < 60 years (P = 0.30), and de novo AML (P = 0.10) did not enhance response achievement, nor a survival advantage. AHSCT was performed in 12 patients with (n = 8) or without (n = 4) CR/CRi/MLFS. 1/2/5-year overall survival (OS) rates were 63%/38%/33% in patients who received AHSCT (n = 12) vs 27%/0%/0% in those who achieved CR/CRi/MLFS but were not transplanted (n = 5), vs 14%/0%/0% who were neither transplanted nor achieved CR/CRi/MLFS (n = 43; P < 0.001); the median OS was 18.6, 12.6 and 5.6 months, respectively. Although CR/CRi/MLFS bridged to AHSCT (n = 8), appeared to manifest a longer median OS (20 months), vs (13.4 months) for those with no response consolidated with AHSCT (n = 4), the difference was not significant P = 0.47. We conclude AHSCT as indispensable for securing long-term survival in prAML (p = 0.03 on multivariate analysis), irrespective of response achievement.

我们试图评估在原发性难治性急性髓细胞白血病 (prAML) 中的总体反应和挽救化疗的价值，包括同种异体造血干细胞移植 (AHSCT)。为了在临床实践中建立一致性，2017 年欧洲白血病网 (ELN) 将 prAML 定义为在至少 2 个强化诱导化疗疗程后未能达到 CR。在符合 prAML 的 ELN 标准的 60 例连续患者（中位年龄 63 岁）中，48 例记录到挽救，30/48（63%）接受强化化疗方案，2/48 合并 AHSCT 作为一线挽救。13/48 (27%) 获得响应：CR, 7/13 (54%), CRi, 2/13 (15%), MLFS, 4/13 (31%)。CR/CRi率为9/48（19%），CR率为7/48（15%）。在单变量分析中，P  = 0.04）。给予任何更高剂量 (>1 g/m ² ) 的阿糖胞苷强化诱导 ( P  = 0.50)、强化抢救化疗 ( P  = 0.72)、靶向抢救 (FLT3 或 IDH 抑制剂) ( P  = 0.42)、大于 1 次抢救方案 ( P  = 0.89)、年龄 < 60 岁 ( P  = 0.30) 和新发 AML ( P  = 0.10) 并没有提高反应成就，也没有生存优势。AHSCT 对 12 名患有 ( n  = 8) 或不患有 ( n  = 4) CR/CRi/MLFS 的患者进行。接受 AHSCT ( n = 12)的患者的 1/2/5 年总生存 (OS) 率为 63%/38%/33%，而接受 AHSCT 的患者为 27%/0%/0%达到 CR/CRi/MLFS 但未移植的患者（n  = 5），与既未移植也未达到 CR/CRi/MLFS 的 14%/0%/0%（n  = 43；P  < 0.001）；中位 OS 分别为 18.6、12.6 和 5.6 个月。尽管 CR/CRi/MLFS 桥接到 AHSCT（n  = 8），似乎表现出更长的中位 OS（20 个月），而与 AHSCT 合并无反应的患者（13.4 个月）相比（n  = 4），差异不显着P  = 0.47。我们得出结论，AHSCT 对于确保 prAML 的长期生存是必不可少的（ 多变量分析p = 0.03），无论反应成就如何。