Uderzo C, Valsecchi M G, Balduzzi A, Dini G, Miniero R, Locatelli F, Rondelli R, Pession A, Arcese W, Bacigalupo A, Polchi P, Andolina M, Messina C, Conter V, Aricó M, Galimberti S, Masera G
Paediatrics Department, University of Milan, Monza, Italy.
Br J Haematol. 1997 Feb;96(2):387-94. doi: 10.1046/j.1365-2141.1997.d01-2033.x.
We compared the outcome of children with high-risk acute lymphoblastic leukaemia (HR-ALL) in first complete remission (first CR) treated with chemotherapy (CHEMO) or with allogeneic bone marrow transplantation (BMT) in a multicentre study. All children treated by the Italian Paediatric Haematology Oncology Association for HR-ALL in first CR between 1986 and 1994 were eligible for the study. 30 children were given BMT at a median of 4 months from first CR, with preparative regimens including total-body irradiation (n = 25/30). 130 matched controls for BMT patients were identified among 397 HR-ALL CHEMO patients. Matching on main prognostic factors and duration of first CR was adopted to control the selection and time-to-transplant biases. The comparative analysis was based on the results of a stratified Cox model. The estimated hazard ratios of BMT versus CHEMO at 6 months, 1 year and 2 years after CR were 1.38 (CI 0.59-3.24), 0.69 (CI 0.27-1.77) and 0.35 (CI 0.06-1.91), with an overall non-significant difference between the two groups (P = 0.34). With a median follow-up of 4 years, the disease-free survival was 58.5% (SE 9.3) in the BMT group and 47.7% (SE 4.8) in the CHEMO group, at 4 years from CR. Non-leukaemic death occurred in 4% of CHEMO and 10% of BMT patients. In the BMT group the estimated cumulative incidence of relapse at 1.5 years from CR was 31.5% (SE 8.8) and did not change thereafter, whereas in the CHEMO group the corresponding figure was 29.2% (SE 4.1) and the incidence continued to increase thereafter (48.2% (SE 4.8) at 4 years from CR). The results of this study suggest that, with respect to the CHEMO group, the higher risk of early failure in the BMT group is outweighed by the lower risk of relapse after 1 year. Results prompt the need for a prospective study, in order to demonstrate the likely advantage of BMT in HR childhood ALL in first CR.
在一项多中心研究中，我们比较了高危急性淋巴细胞白血病（HR-ALL）首次完全缓解（首次CR）的儿童接受化疗（CHEMO）或异基因骨髓移植（BMT）的治疗结果。1986年至1994年间，所有由意大利儿科血液肿瘤协会治疗的处于首次CR的HR-ALL儿童均符合该研究的条件。30名儿童在首次CR后中位4个月接受了BMT，预处理方案包括全身照射（30例中的25例）。在397例接受化疗的HR-ALL患者中确定了130名与BMT患者匹配的对照。采用主要预后因素和首次CR持续时间进行匹配，以控制选择和移植时间偏差。比较分析基于分层Cox模型的结果。CR后6个月、1年和2年时，BMT与CHEMO相比的估计风险比分别为1.38（CI 0.59 - 3.24）、0.69（CI 0.27 - 1.77）和0.35（CI 0.06 - 1.91），两组之间总体差异无统计学意义（P = 0.34）。中位随访4年时，BMT组的无病生存率为58.5%（SE 9.3），CHEMO组为47.7%（SE 4.8），自CR起4年。非白血病死亡发生在4%的化疗患者和10%的BMT患者中。在BMT组中，自CR起1.5年时估计的复发累积发生率为31.5%（SE 8.8），此后未改变，而在CHEMO组中相应数字为29.2%（SE 4.），此后发生率持续上升（自CR起4年时为48.2%（SE 4.8））。本研究结果表明，相对于化疗组，BMT组早期失败的较高风险被1年后较低的复发风险所抵消。结果提示需要进行前瞻性研究，以证明BMT在处于首次CR的儿童HR-ALL中的可能优势。
