Background:TP53mutations (TP53m) define the most treatment-refractory acute myeloid leukemia (AML) subtype. Optimal treatment approaches have not been established in this setting. We reviewed our institutional experience to identify therapy sequencing, treatment response, and survival patterns in these patients. Methods: This study was a single-center, retrospective cohort analysis. Results: Our cohort includes 86TP53m and 337TP53wild-type (TP53wt) adult AML patients.TP53m AML patients presented with lower bone marrow and peripheral blasts; none presented with hyperleukocytosis. Patients who received intensive treatment up front demonstrated superior overall survival (OS) over those receiving first-line non-intensive therapy (2-year OS 22% versus 7%;p= 0.02). However, the complete remission (CR) rates among the first-line intensive and non-intensive therapy groups were comparable (21.9% and 29.4%, respectively,p= 0.49). The improved OS is therefore attributed to superior cumulative CR in the intensive group. First-line intensively treated patients were more likely to receive and respond to salvage, leading to a cumulative CR rate of 65.7% (versus 29.4%,p= 0.003). Achieving CR at any point is strongly associated with superior survival outcomes with 2-year OS of 31% versus 0% for those not achieving CR ever (p< 0.01). Conclusions: We find thatTP53m AML rarely presents with oncological emergencies, suggesting that clinical trial enrollment is feasible in this group. Additionally, in our cohort, intensive induction therapies lead to superior survival outcomes attributed to successful salvage therapy. These data suggest that strategic therapy sequencing and salvage therapy may be important in optimizing outcomes forTP53m AML patients.
背景：TP53突变（TP53m）是急性髓系白血病（AML）中治疗最为棘手的亚型。目前针对该亚型尚未确立最佳治疗方案。本研究回顾了本机构的临床经验，旨在分析此类患者的治疗顺序、治疗反应及生存模式。方法：本研究为单中心回顾性队列分析。结果：队列共纳入86例TP53m及337例TP53野生型（TP53wt）成人AML患者。TP53m AML患者初诊时骨髓及外周血原始细胞比例较低，且均未出现高白细胞血症。接受强化诱导治疗的患者总生存期（OS）显著优于一线接受非强化治疗者（2年OS：22%对7%；p=0.02）。然而，一线强化治疗组与非强化治疗组的完全缓解（CR）率相当（分别为21.9%和29.4%，p=0.49）。因此OS的改善归因于强化治疗组更高的累积CR率。一线接受强化治疗的患者更可能接受挽救治疗并获得应答，累积CR率达65.7%（非强化组为29.4%，p=0.003）。在任何治疗阶段获得CR均与显著改善的生存结局相关：获得CR者2年OS为31%，而未获CR者则为0%（p<0.01）。结论：本研究发现TP53m AML极少出现肿瘤急症，提示该群体具备临床试验入组可行性。此外，在本队列中，强化诱导治疗通过成功的挽救治疗实现了更优的生存结局。这些数据表明，策略性治疗排序及挽救治疗对优化TP53m AML患者的临床预后具有重要意义。
肿瘤(癌症)患者之家