Bertoli Sarah, Dumas Pierre-Yves, Bérard Emilie, Largeaud Laetitia, Bidet Audrey, Delabesse Eric, Tavitian Suzanne, Gadaud Noémie, Leguay Thibaut, Leroy Harmony, Rieu Jean-Baptiste, Vial Jean-Philippe, Vergez François, Lechevalier Nicolas, Luquet Isabelle, Klein Emilie, Sarry Audrey, Grande Anne-Charlotte De, Récher Christian, Pigneux Arnaud
Service d'Hématologie, Centre Hospitalier Universitaire de Toulouse, Institut Universitaire du Cancer de Toulouse-Oncopole, 31059 Toulouse, France.
University Toulouse III Paul Sabatier, 31000 Toulouse, France.
Cancers (Basel). 2020 Mar 25;12(4):773. doi: 10.3390/cancers12040773.
A recent phase 3 trial showed that the outcome of patients with relapsed/refractory (R/R) -mutated acute myeloid leukemia (AML) improved with gilteritinib, a single-agent second-generation FLT3 tyrosine kinase inhibitor (TKI), compared with standard of care. In this trial, the response rate with standard therapy was particularly low. We retrospectively assessed the characteristics and outcome of patients with R/R -mutated AML included in the Toulouse-Bordeaux DATAML registry. Among 347 patients who received FLT3 TKI-free intensive chemotherapy as first-line treatment, 174 patients were refractory ( = 48, 27.6%) or relapsed ( = 126, 72.4%). Salvage treatments consisted of intensive chemotherapy ( = 99, 56.9%), azacitidine or low-dose cytarabine ( = 9, 5.1%), other low-intensity treatments ( = 17, 9.8%), immediate allogeneic stem cell transplantation ( = 4, 2.3%) or best supportive care only ( = 45, 25.9%). Among the 114 patients who previously received FLT3 TKI-free intensive chemotherapy as first-line treatment (refractory, = 32, 28.1%; relapsed, = 82, 71.9%), the rate of CR (complete remission) or CRi (complete remission with incomplete hematologic recovery) after high- or low-intensity salvage treatment was 50.0%, with a bridge to transplant in 34.2% ( = 39) of cases. The median overall survival (OS) was 8.2 months (interquartile range, 3.0-32); 1-, 3- and 5-year OS rates were 36.0% (95%CI: 27-45), 24.7% (95%CI: 1-33) and 19.7% (95%CI: 1-28), respectively. In this real-word study, although response rate appeared higher than the controlled arm of the ADMIRAL trial, the outcome of patients with R/R -mutated AML remains very poor with standard salvage therapy.
一项近期的3期试验表明，与标准治疗相比，单药第二代FLT3酪氨酸激酶抑制剂（TKI）吉瑞替尼可改善复发/难治性（R/R）-突变急性髓系白血病（AML）患者的预后。在该试验中，标准治疗的缓解率特别低。我们回顾性评估了图卢兹-波尔多DATAML登记处纳入的R/R-突变AML患者的特征和预后。在347例接受不含FLT3 TKI的强化化疗作为一线治疗的患者中，174例患者难治（n = 48，27.6%）或复发（n = 126，72.4%）。挽救治疗包括强化化疗（n = 99，56.9%）、阿扎胞苷或小剂量阿糖胞苷（n = 9，5.1%）、其他低强度治疗（n = 17，9.8%）、立即进行异基因干细胞移植（n = 4，2.3%）或仅给予最佳支持治疗（n = 45，25.9%）。在114例先前接受不含FLT3 TKI的强化化疗作为一线治疗的患者中（难治，n = 32，28.1%；复发，n = 82，71.9%），高强度或低强度挽救治疗后的完全缓解（CR）或伴有血液学不完全恢复的完全缓解（CRi）率为50.0%，34.2%（n = 39）的病例有移植桥接。中位总生存期（OS）为8.2个月（四分位间距，3.0 - 32）；1年、3年和5年OS率分别为36.0%（95%CI：27 - 45）、24.7%（95%CI：1 - 33）和19.7%（95%CI：1 - 28）。在这项真实世界研究中，尽管缓解率似乎高于ADMIRAL试验的对照组，但R/R-突变AML患者采用标准挽救治疗的预后仍然很差。
