Song Yu-Hua, Peng Peng, Qiao Chun, Li Jian-Yong, Long Qi-Qiang, Lu Hua
Department of Haematology, The Second Hospital of Nanjing, Nanjing University of Chinese Medicine, Nanjing, Jiangsu, People's Republic of China.
Department of Haematology, First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Nanjing, Jiangsu, People's Republic of China.
Cancer Manag Res. 2021 Mar 12;13:2371-2378. doi: 10.2147/CMAR.S297421. eCollection 2021.
To evaluate the influence of mutations on the treatment response and long-term survival of newly-diagnosed patients with acute promyelocytic leukemia (APL) treated with all-trans retinoic acid and arsenic trioxide.
The long-term survival of 90 newly-diagnosed APL patients (age range 12-75 years) was retrospectively analyzed.The mutation rate was assayed by polymerase chain reaction (PCR) amplification and sequencing analysis. Its impact on the treatment response, event-free survival(EFS), or overall survival(OS) was investigated in patients with and without the mutations.
The mutation rate in newly-diagnosed APL patients was 20% (18/90). The white blood cell (WBC) count at diagnosis in patients with mutations was significantly higher than that in patients without mutations while the mutation rate was higher in the high-risk group than in the low/intermediate-risk group. Patients with mutations had a significantly higher early death (ED) rate (16.67% vs 1.39%) for those lacking the mutation ( =0.024). However, the complete remission (CR) and differentiation syndrome (DS) rates in the two groups were similar. Kaplan Meier analysis for EFS and OS at five years showed a significant difference between the patients stratified by mutation status (log-rank =0.010 and =0.009, respectively).
mutations can be related to high peripheral WBC counts in APL patients. APL patients with mutations displayed a higher ED rate compared to those without mutations. Patients carrying mutations had reduced five-year EFS and OS rates. Thus, reducing the overall death rate during induction treatment might be an effective way to improve the prognosis of patients with mutations.
评估突变对接受全反式维甲酸和三氧化二砷治疗的新诊断急性早幼粒细胞白血病（APL）患者治疗反应和长期生存的影响。
回顾性分析90例新诊断的APL患者（年龄范围12 - 75岁）的长期生存情况。通过聚合酶链反应（PCR）扩增和测序分析检测突变率。在有和无突变的患者中研究其对治疗反应、无事件生存期（EFS）或总生存期（OS）的影响。
新诊断的APL患者中突变率为20%（18/90）。有突变患者诊断时的白细胞（WBC）计数显著高于无突变患者，且高危组的突变率高于低/中危组。有突变患者的早期死亡率（ED）显著高于无突变患者（16.67%对1.39%，P = 0.024）。然而，两组的完全缓解（CR）率和分化综合征（DS）率相似。对EFS和OS的五年Kaplan Meier分析显示，按突变状态分层的患者之间存在显著差异（对数秩检验P分别为0.010和0.009）。
突变可能与APL患者外周血白细胞计数高有关。有突变的APL患者与无突变患者相比显示出更高的ED率。携带突变的患者五年EFS和OS率降低。因此，降低诱导治疗期间的总体死亡率可能是改善有突变患者预后的有效方法。
