Martins Carlos, Lacerda João F, Lourenço Fernanda, Carmo José A, Lacerda J M F
Unidade de Transplante de Medula Ossea, Hospital de Santa Maria, Faculdade de Medicina de Lisboa, Lisboa, Portugal.
Acta Med Port. 2005 Sep-Oct;18(5):329-37. Epub 2005 Oct 16.
We report our results of autologous stem cell transplantation (SCT) in patients with AML during the last 13 years. Between August 1990 and December 2003, 42 patients with acute myeloid leukemia (AML) received an autologous SCT. Patients were classified as standard risk if first complete remission (CR) was induced after one or two chemotherapy regimens and the white blood cell count at presentation was below 50,000/mL (n=12), while patients requiring more than two induction regimens to attain first CR and with CR2 ou more advanced disease and/or had a higher white blood cell count at presentation were defined as high risk (n=30). Twenty one patients were transplanted in first CR. The median patient age was 24 years (range, 2-56 years), and the median time interval from diagnosis to autologous SCT was 9 months (range 3-87 months). The conditioning regimen for SCT consisted of busulfan (BU) 16 mg/kg and melfalan (MEL) 180 mg/m2 (BUMEL) in 17 (40%) patients and busulfan 16 mg/kg and VP-16 60 mg/kg (BUVP16) in 22 (52%) patients. Three patients received a different conditioning regimen with BCNU 300 mg/m2, VP16 2 g/m2 and melphalan 160 mg/m2 (BEM). Twenty five (60%) patients received bone marrow (BM), 11 (26%) patients received peripheral blood stem cells (PBSC) and 6 patients (14%) received BM plus PBSC. With a median follow-up of 7 years, the 13 year overall survival (OS) and diseasefree survival (DFS) of all patients is 52% and 40%, respectively. In univariate analysis, males had a significantly superior DFS than females (55% vs 22%, p=0.003), and patients younger than 15 years of age had significantly superior OS and DFS than older patients (50% vs 35%, p=0.05; and 50% vs 28%, p=0.03, respectively). Patients with FAB M3 subtype also had a superior OS than the other FAB subtypes (100% vs 44%, p=0.05). There was a strong statistical correlation between risk group and survival. In fact, the patients with standard risk had a superior OS and DFS than those with high risk disease (67% vs 23%, p=0.0004; and 50% vs 27%, p=0.01, respectively). When patients with FAB M3 disease were excluded from the analysis, the group with standard risk continue to have a superior OS and DFS (67% vs 13%, p=0.008; and 50% vs 14%, p=0.02, respectively). We conclude that autologous SCT is an effective treatment in AML with the possibility of long survivorship, particularly in patients with standard risk disease.
我们报告了过去13年中急性髓系白血病（AML）患者自体干细胞移植（SCT）的结果。1990年8月至2003年12月期间，42例急性髓系白血病患者接受了自体SCT。如果患者在接受一或两个化疗方案后诱导出首次完全缓解（CR）且就诊时白细胞计数低于50,000/mL，则被分类为标准风险（n = 12），而需要超过两个诱导方案才能达到首次CR、处于CR2或更晚期疾病阶段和/或就诊时白细胞计数较高的患者被定义为高风险（n = 30）。21例患者在首次CR时接受了移植。患者的中位年龄为24岁（范围为2 - 56岁），从诊断到自体SCT的中位时间间隔为9个月（范围为3 - 87个月）。17例（40%）患者的SCT预处理方案为白消安（BU）16 mg/kg和美法仑（MEL）180 mg/m²（BUMEL），22例（52%）患者的预处理方案为白消安16 mg/kg和依托泊苷（VP - 16）60 mg/kg（BUVP16）。3例患者接受了不同的预处理方案，即卡莫司汀（BCNU）300 mg/m²、VP16 2 g/m²和美法仑160 mg/m²（BEM）。25例（60%）患者接受了骨髓（BM）移植，11例（26%）患者接受了外周血干细胞（PBSC）移植，6例（14%）患者接受了BM加PBSC移植。中位随访7年，所有患者的13年总生存率（OS）和无病生存率（DFS）分别为52%和40%。单因素分析显示，男性的DFS显著优于女性（55%对22%，p = 0.003），15岁以下患者的OS和DFS显著优于年龄较大的患者（分别为50%对35%，p = 0.05；50%对28%，p = 0.03）。FAB M3亚型患者的OS也优于其他FAB亚型（100%对44%，p = 0.05）。风险组与生存率之间存在很强的统计学相关性。实际上，标准风险患者的OS和DFS优于高风险疾病患者（分别为67%对23%，p = 0.0004；50%对27%，p = 0.01）。当将FAB M3疾病患者排除在分析之外时，标准风险组的OS和DFS仍然更高（分别为67%对13%，p = 0.008；50%对14%，p = 0.02）。我们得出结论，自体SCT是AML的一种有效治疗方法，有可能实现长期生存，特别是对于标准风险疾病患者。
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