儿童急性髓系白血病82例长期随访研究

A long-term follow-up study of 82 children with acute myeloid leukemia

目的分析中国医学科学院血液病医院儿童血液病诊疗中心2005方案（CAMS-2005）治疗儿童急性髓系白血病（AML）的疗效及预后相关因素。方法对2005年4月至2009年7月中国医学科学院血液病医院儿童血液病诊疗中心符合入组标准的初诊AML患儿的临床特征、远期预后和预后相关因素进行回顾性分析。采用Kaplan-Meier方法评估患儿生存率，组间生存率的比较采用Log-Rank检验。预后因素分析采用COX回归模型。结果共82例患儿符合入组标准纳入统计分析,其中核心结合因子（CBF）相关AML（CBF-AML）患儿34例，非CBF-AML患儿48例。82例患儿中男45例，女37例，中位年龄8.0（0.7-16.0）岁。治疗相关早期病死率为4%（3/82），1个疗程CR率为65%（53/82），总CR率为77%（63/82）,累积复发率为33%（21/63）。CBF-AML和非CBF-AML患儿的CR率分别为91%（31/34）和67%（32/48）（χ2=5.410，P=0.020），复发率分别为29%（9/31）和38%（12/32）（χ2=0.508，P=0.476）。82例AML患儿8年总生存率（OS）和无事件生存率（EFS）分别为59%（48/82）和51%（42/82）。CBF-AML患儿和非CBF-AML患儿的8年OS分别为74%（25/34）和48%（23/48） （χ2=5.812，P=0.016），8年EFS分别为71%（24/34）和38%（18/48）（χ2=8.682，P=0.003），组间比较差异均有统计学意义。1个疗程获得CR的患儿和未获得CR患儿的8年OS分别为68%（36/53）和46%（12/26）（χ2=9.606，P=0.002），8年EFS分别为66%（35/53）和27%（7/26）（χ2=19.471，P=0.000），差异均有统计学意义。COX回归分析显示是否为CBF-AML和1个疗程是否获得CR对OS（RR：2.538、2.561）和EFS（RR：3.050、3.686）的影响均有统计学意义（P均〈0.05）。结论CAMS-2005方案治疗儿童AML的疗效较好，是否为CBF-AML和1个疗程是否获得CR是影响儿童AML的独立预后因素。

ObjectiveTo investigate the efficacy and the prognostic factors of Chinese Academy of Medical Sciences 2005 （CAMS-2005） regimen in the treatment of pediatric acute myeloid leukemia （AML）.MethodsEighty-eight cases of newly-diagnosed AML patients, who were treated with the CAMS-2005 regimen from April 2005 to July 2009, were enrolled in this case observational study. Clinical characteristics, long-term prognosis and prognostic factors were analyzed retrospectively. Overall survival （OS） and event free survival （EFS） rates were estimated by the Kaplan-Meier method. Rates of survival between the groups were compared by the Log-rank test. Prognostic factors were evaluated by COX regression analysis. ResultsA total of 82 cases were enrolled in this study, including 34 core binding factor（CBF）-AML patients and 48 non-CBF-AML patients. There were 45 males and 37 females. The median age at diagnosis was 8.0 （0.7-16.0） years. During the induction therapy, 3 patients （4%） developed treatment-related early-death, while 63 patients （77%） achieved complete remission （CR） and 53 patients （65%） achieved CR after 1 course. Twenty-one patients （33%） had relapsed disease. The CR rates of CBF-AML patients and non-CBF-AML patients were 91% （31/34） and 67% （32/48） （χ2=5.410, P=0.020） , while the relapse rates were 29% （9/31） and 38% （12/32） （χ2=0.508, P=0.476） . The 8-year OS and EFS rates of all 82 patients were 59%（48/82） and 51%（42/82）. The 8-year OS rates of CBF-AML patients and non-CBF-AML patients were 74% （25/34） and 48%（23/48） （χ2=5.812, P=0.016）, while the 8-year EFS rates of CBF-AML patients and non-CBF-AML patients were 71%（24/34） and 38%（18/48） （χ2=8.682, P=0.003）. There were statistically significant differences between groups. The 8-year OS rates of patients who achieved CR after 1 course and other patients were 68% （36/53） and 46% （12/26） （χ2=9.606, P=0.002）, while the 8-year EFS rates were 66% （35/53） and 27% （7/26） （χ2=19.471, P=0.000）, the differences were all statistically significant. COX multivariate analysis showed that CBF-AML or non-CBF-AML and whether achieved CR after 1 course were independent prognostic factors of OS rates （relative risk： 2.538, 2.561） and EFS rates （relative risk： 3.050, 3.686） （P 〈0.05）.ConclusionsThe efficacy of the CAMS-2005 regimen in the treatment of AML patients was well. CBF-AML or non-CBF-AML and whether achieved CR after 1 course were independent prognostic factors for pediatric AML patients.