Michel G, Gluckman E, Esperou-Bourdeau H, Reiffers J, Pico J L, Bordigoni P, Thuret I, Blaise D, Bernaudin F, Jouet J P
Service d'Hématologie Pédiatrique, Hôpital d'enfants La Timone, Marseille, France.
J Clin Oncol. 1994 Jun;12(6):1217-22. doi: 10.1200/JCO.1994.12.6.1217.
To analyze the French experience of chemotherapeutic preparation before human leukocyte antigen (HLA)-identical bone marrow transplantation (BMT) in children with acute myeloblastic leukemia (AML) in first complete remission (CR).
The data base used for this study was a French BMT registry for childhood AML. Twenty-three children were conditioned with busulfan and 120 mg/kg cyclophosphamide (Bu-Cy 120 group). Nineteen received busulfan and 200 mg/kg cyclophosphamide (Bu-Cy200 group). During the same time period, 32 patients were prepared with total-body irradiation (TBI group) most often in combination with 120 mg/kg of cyclophosphamide.
The probability of relapse was 54%, 13%, and 10% for the Bu-Cy120, Bu-Cy200, and TBI groups, respectively (P < .05 in the univariate analysis, log-rank test, 2 df). In the multivariate analysis, a conditioning regimen with Bu-Cy120 was significantly associated with a higher risk of relapse (P = .02; relative risk, 3.62). The probability of transplant-related mortality (TRM) was 0% for Bu-Cy120, 5% for Bu-Cy200, and 10% for TBI. Kaplan-Meier estimations of event-free survival (EFS) were 46% +/- 24%, 82% +/- 18%, and 80% +/- 14%, respectively, for the three groups, with median follow-up durations of 28 months (range, 3 to 78), 31 months (4 to 68), and 48 months (2 to 73). In the multivariate analysis, two factors adversely affected EFS: a conditioning regimen with Bu-Cy120 (P = .07) and a long interval from diagnosis to BMT (> or = 120 days, P = .08).
Bu-Cy120 is a well-tolerated preparation, but results in a high risk of relapse for children with AML in first CR. This high risk of relapse is not observed when the dose of cyclophosphamide is increased to 200 mg/kg.
分析法国在人类白细胞抗原(HLA)配型相合的骨髓移植(BMT)前对首次完全缓解(CR)的急性髓细胞白血病(AML)患儿进行化疗预处理的经验。
本研究使用的数据库是法国儿童AML的BMT登记处。23例患儿采用白消安和120mg/kg环磷酰胺进行预处理(白消安-环磷酰胺120组)。19例接受白消安和200mg/kg环磷酰胺(白消安-环磷酰胺200组)。在同一时期,32例患者采用全身照射进行预处理(全身照射组),多数联合120mg/kg环磷酰胺。
白消安-环磷酰胺120组、白消安-环磷酰胺200组和全身照射组的复发概率分别为54%、13%和10%(单因素分析,对数秩检验,P<.05,2个自由度)。多因素分析显示,白消安-环磷酰胺120预处理方案与较高的复发风险显著相关(P =.02;相对风险,3.62)。白消安-环磷酰胺120组的移植相关死亡率(TRM)为0%,白消安-环磷酰胺200组为5%,全身照射组为10%。三组的无事件生存期(EFS)的Kaplan-Meier估计值分别为46%±24%、82%±18%和80%±14%,中位随访时间分别为28个月(范围3至78个月)、31个月(4至68个月)和48个月(2至73个月)。多因素分析中,两个因素对EFS有不利影响:白消安-环磷酰胺120预处理方案(P =.07)和从诊断到BMT的间隔时间长(≥120天,P =.08)。
白消安-环磷酰胺120是一种耐受性良好的预处理方案,但会导致首次CR的AML患儿有较高的复发风险。当环磷酰胺剂量增加到200mg/kg时,未观察到这种高复发风险。