[82例急性髓系白血病患儿的长期随访研究]
[A long-term follow-up study of 82 children with acute myeloid leukemia].
作者信息
Ruan M, Qi B Q, Liu F, Liu T F, Liu X M, Chen X J, Yang W Y, Guo Y, Zhang L, Zou Y, Chen Y M, Zhu X F
机构信息
Institute of Hematology, Blood Disease Hospital, Chinese Academy of Medical Sciences, Tianjin 300020, China.
出版信息
Zhonghua Er Ke Za Zhi. 2018 Oct 2;56(10):730-734. doi: 10.3760/cma.j.issn.0578-1310.2018.10.003.
To 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). Eighty-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. A 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) (χ(2)=5.410, 0.020) , while the relapse rates were 29% (9/31) and 38% (12/32) (χ(2)=0.508, 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) (χ(2)=5.812, 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) (χ(2)=8.682, 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) (χ(2)=9.606, 0.002), while the 8-year EFS rates were 66% (35/53) and 27% (7/26) (χ(2)=19.471, 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) ( <0.05). The 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.
探讨中国医学科学院2005方案（CAMS-2005）治疗儿童急性髓系白血病（AML）的疗效及预后因素。本病例观察性研究纳入了2005年4月至2009年7月期间接受CAMS-2005方案治疗的88例新诊断AML患者。对其临床特征、长期预后及预后因素进行回顾性分析。采用Kaplan-Meier法估计总生存（OS）率和无事件生存（EFS）率。采用Log-rank检验比较组间生存率。通过COX回归分析评估预后因素。本研究共纳入82例患者，其中核心结合因子（CBF）-AML患者34例，非CBF-AML患者48例。男性45例，女性37例。诊断时的中位年龄为8.0（0.7 - 16.0）岁。诱导治疗期间，3例患者（4%）发生治疗相关早期死亡，63例患者（77%）获得完全缓解（CR），53例患者（65%）在1个疗程后获得CR。21例患者（33%）疾病复发。CBF-AML患者和非CBF-AML患者的CR率分别为91%（31/34）和67%（32/48）（χ² = 5.410，P = 0.用Log-rank检验比较组间生存率。通过COX回归分析评估预后因素。本研究共纳入82例患者，其中核心结合因子（CBF）-AML患者34例，非CBF-AML患者48例。男性45例，女性37例。诊断时的中位年龄为8.0（0.7 - 16.0）岁。诱导治疗期间，3例患者（4%）发生治疗相关早期死亡，63例患者（77%）获得完全缓解（CR），53例患者（65%）在1个疗程后获得CR。21例患者（33%）疾病复发。CBF-AML患者和非CBF-AML患者的CR率分别为91%（31/34）和67%（32/48）（χ² = 5.410，P = 0.020），而复发率分别为29%（9/31）和38%（12/32）（χ² = 0.508，P = 0.476）。82例患者的8年OS率和EFS率分别为59%（48/82）和51%（42/82）。CBF-AML患者和非CBF-AML患者的8年OS率分别为74%（25/34）和48%（23/48）（χ² = 5.812，P = 0.016），而CBF-AML患者和非CBF-AML患者的8年EFS率分别为71%（24/34）和38%（18/48）（χ² = 8.682，P = 0.003）。组间差异有统计学意义。1个疗程后获得CR的患者与其他患者的8年OS率分别为68%（36/53）和46%（12/26）（χ² = 9.606，P = 0.002），而8年EFS率分别为66%（35/53）和27%（7/26）（χ² = 19.471，P = 0.000），差异均有统计学意义。COX多因素分析显示，CBF-AML或非CBF-AML以及是否在1个疗程后获得CR是OS率（相对危险度：2.538，2.561）和EFS率（相对危险度：3.050，3.686）的独立预后因素（P < 0.05）。CAMS-2-AML患者的疗效良好。CBF-AML或非CBF-AML以及是否在1个疗程后获得CR是儿童AML患者的独立预后因素。 020），而复发率分别为29%（9/31）和38%（12/32）（χ² = 0.508，P = 0.476）。82例患者的8年OS率和EFS率分别为59%（48/82）和51%（42/82）。CBF-AML患者和非CBF-AML患者的8年OS率分别为74%（25/34）和48%（23/48）（χ² = 5.812，P = 0.016），而CBF-AML患者和非CBF-AML患者的8年EFS率分别为71%（24/34）和38%（18/48）（χ² = 8.682，P = 0.003）。组间差异有统计学意义。1个疗程后获得CR的患者与其他患者的8年OS率分别为68%（36/53）和46%（12/26）（χ² = 9.606，P = 0.002），而8年EFS率分别为66%（35/53）和27%（7/26）（χ² = 19.471，P = 0.000），差异均有统计学意义。COX多因素分析显示，CBF-AML或非CBF-AML以及是否在1个疗程后获得CR是OS率（相对危险度：2.538，2.561）和EFS率（相对危险度：3.050，3.686）的独立预后因素（P < 0.05）。CAMS-2005方案治疗AML患者的疗效良好。CBF-AML或非CBF-AML以及是否在1个疗程后获得CR是儿童AML患者的独立预后因素。
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