接受强化化疗治疗的核心结合因子急性髓系白血病老年患者中有相当一部分可以实现长期生存。
Long-term survival can be achieved in a significant fraction of older patients with core binding factor acute myeloid leukemia treated with intensive chemotherapy.
发表日期：2024 Oct 10
作者：
Federico Mosna, Erika Borlenghi, Mark Litzow, John C Byrd, Cristina Papayannidis, Cristina Tecchio, Felicetto Ferrara, Guido Marcucci, Roberto Cairoli, Elizabeth A Morgan, Carmela Gurrieri, Cecilia C S Yeung, H Joachim Deeg, Debora Capelli, Anna Candoni, Jason R Gotlib, Monia Lunghi, Sheeja Pullarkat, Francesco Lanza, Sara Galimberti, Fabio Forghieri, Adriano Venditti, Moreno Festuccia, Ernesta Audisio, Denise Marvalle, Gian Matteo Rigolin, Giovanni Roti, Eros DiBona, Giuseppe Visani, Francesco Albano, Ann-Kathrin Eisfeld, Peter Valent, Gerwin Huls, Gautam Borthakur, Mauro Krampera, Giovanni Martinelli, Nicolaus Kröger, Alessandra Sperotto, Michele Gottardi
来源：
HAEMATOLOGICA
摘要：
急性髓系白血病主要是一种老年人疾病：然而，对老年人群核心结合因子 AML (CBFAML) 治疗结果的了解有限。我们回顾性收集了过去二十年长期随访的 229 名 CBF-AML 患者的数据。观察到 5 年总生存 (OS) 为 44.2% (95% CI, 39.9-47.5)，5 年无事件生存 (EFS) 为 32.9% (95% CI, 25.5-40.1)。在完成强化治疗的 70 岁以上患者亚组中（诱导 >3 个巩固疗程，包括自体干细胞移植：10 名患者），中位 EFS 为 11.8 个月（95% CI，9.4 - 15.2），OS 为 40.0%（ 5 年时为 95% CI，36.4 - 44.1）。在单变量分析中，年龄 >70（风险比 (HR) 1.78，[95%CI，1.15 - 2.54]，p=.008），诱导后未能达到缓解（HR，8.96 [95%CI，5.5 - 13.8] ，p=<.0001），无巩固治疗（HR，0.75 [95%CI，0.47 - 1.84]，p=.04）且巩固治疗周期少于 3 个周期（HR，1.48 [95%CI，0.75 - 3.2]） ，p=.0004)，预测 EFS 较差。我们的研究表明，对选定的老年 CBF-AML 患者进行强化治疗可以延长生存期。实现 CR 似乎是最重要的第一步，而至少 3 个巩固周期是重要的第二步。分析表明，不应将这些患者排除在强化治疗研究之外。
Acute Myeloid Leukemia is mainly a disease of the elderly: however, the knowledge on the outcomes of treatment in core binding factor AML (CBFAML) in older population, is limited. We retrospectively collected data on 229 patients with CBF- AML followed long-term in the last two decades. A 5-year overall survival (OS) of 44.2% (95%CI, 39.9-47.5) and a 5-year event - free survival (EFS) of 32.9% (95%CI, 25.5-40.1) was observed. In a subgroup of >70-year patients who completed intensive therapy (induction + >3 courses of consolidation including autologous stem cell transplant: 10 patients) the median EFS was 11.8 months (95%CI, 9.4 - 15.2) and OS was 40.0% (95%CI, 36.4 - 44.1) at 5yr. In univariate analysis, age >70 (hazard ratio (HR) 1.78, [95%CI, 1.15 - 2.54], p=.008), failure to achieve remission following induction (HR, 8.96 [95%CI, 5.5 - 13.8], p=<.0001), no consolidation therapy (HR, 0.75 [95%CI, 0.47 - 1.84], p=.04) and less than 3 cycles of consolidation (HR, 1.48 [95%CI, 0.75 - 3.2], p=.0004), predicted poorer EFS. Our study shows that intensive therapy, in selected older CBF-AML patients, leads to longer survival. Achieving a CR seems to be the most important first step and at least 3 cycles of consolidation, an important second one. The analysis suggests that these patients should not be excluded from studies with intensive therapies.