Objective: To investigate the clinical features and outcomes of high-risk acute promyelocytic leukemia (APL) patients.
Methods: A retrospective analysis was conducted to compare the clinical characteristics and prognosis of 118 high-risk APL patients (WBC≥10 × 10(9)/L) and 234 low and intermedia-risk patients (WBC <10×10(9)/L) from January 2003 to April 2015, who were treated in the First Affiliated Hospital of Zhejiang University and Yinzhou People's Hospital affiliated to Medical College of Ningbo University.
Results: The initial platelet counts of high-risk APL were significantly lower than that of low and intermediate-risk groups (P=0.003); the major type of PML-RARα isoforms in high-risk patients was short-form (51.8% vs 28.2%, P <0.001); the early death (ED) rate of high-risk patients was higher than low and intermedia-risk patients (20.3% vs 2.6%, P<0.001); in contrast, the complete remission (CR) rate and 5 years estimated overall survival (OS) rate of the former were lower than the latter (76.3% vs 94.9%, P <0.001; 74.2% vs 93.7%, P <0.001). However, the CR rate (P=0.682) and 5 years estimated OS rate (P=0.481) did not have difference when the ED patients were excluded. The 5 years estimated relapse-free survival (RFS) and central nervous system (CNS) relapse were 82.7%, 9.4%, respectively, which were lower than low and intermediate-risk groups (87.8%, 1.4% ) with statistic difference (P=0.048, 0.002). High-dose cytarabine and intrathecal chemotherapy may reduce the risk of CNS relapse.
Conclusion: The outcomes of high-risk APL patients were worse than low and intermediate-risk group owing to the high ED rate and CNS relapse, it was important to decrease the ED rate and emphasis the CNS prophylaxis for high-risk APL patients.
目的: 分析高危急性早幼粒细胞白血病（APL）的临床特征及预后。
方法: 回顾性分析2003年1月至2015年4月连续收治的APL患者352例，其中高危组（WBC≥10×109/L) 118例，中低危组（WBC<10×109/L）234例。比较两组患者的临床特征及预后差异。
结果: 高危组APL患者初诊PLT水平明显低于中低危组（P=0.003）；高危组患者PML-RARα融合基因异构体S型比例高于中低危组（51.8％对28.2%，P<0.001）；高危组患者早期死亡率为20.3%，显著高于中低危组患者的2.6%（P<0.001），其完全缓解（CR）率及预计5年总生存（OS）率均低于中低危组（76.3％对94.9%，P<0.001；74.2％对93.7%，P<0.001）；若除去早期死亡患者，则两组CR率与5年预计OS率差异均无统计学意义（P值分别为0.682、0.481）。高危组患者预计5年无复发生存率与中枢神经系统白血病（CNSL）发生率分别为82.7%、9.4%，与中低危组的87.8%、1.4％相比差异均有统计学意义（P值分别为0.048、0.002）；中剂量阿糖胞苷化疗及增加鞘内注射次数能降低高危组APL的CNSL发生率。
结论: 高危组APL因有较高的早期死亡率和CNSL发生率，其预后明显较中低危组患者差；因此对于高危组APL患者的治疗更应重视降低早期死亡率及加强CNSL的预防性化疗。