Outcome of treatment in childhood acute lymphoblastic leukaemia with rearrangements of the 11q23 chromosomal region
Outcome of treatment in childhood acute lymphoblastic leukaemia with rearrangements of the 11q23 chromosomal region
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DOI：
10.1016/s0140-6736(02)08782-2
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发表时间：
2002-06-01
期刊：
影响因子：
168.9
通讯作者：
Camitta, B
中科院分区：
文献类型：
--
作者：
Pui, CH;Gaynon, PS;Camitta, B
背景：儿童急性淋巴细胞白血病（ALL）伴染色体11 q23带异常的预后和最佳治疗存在争议。我们的目的是确定可能有助于计划未来治疗的预后因素，并评估t（4;11）易位患者的造血干细胞移植的有效性，这与一个特别差的outcome.Methods我们回顾了497例儿童和年轻成人ALL患者的数据，他们患有各种11 q23异常，包括易位t（4;11），t（9; 11），和t（11;19）。1983年至1995年，11个研究组和单一机构对所有患者进行了强化化疗，首次完全缓解时有或无造血干细胞移植。在按11 q23异常分层的考克斯比例风险模型中，小于1岁的婴儿比1岁或1岁以上的患者表现明显更差（无事件生存的风险比为1.84 [95%CI 1.38-2.47]，p=0.0001）。在婴儿中，11 q23异常的任何类别都赋予了令人沮丧的结果，而在老年患者中，t（4;11）和t（9;11）与其他11 q23变化相比，与更差的结果相关。在最大的亚组-256例t（4;11）患者中，与仅化疗相比，任何类型的移植与无病生存率（1.61 [1.10-2.35]，p=0.014）和总生存率（1.76 [1.08-2.45]，p=0.004）显著更差相关。即使移植与干细胞从HLA匹配的相关或HLA匹配无关的捐助者往往与一个更糟糕的结果比化疗单独。解释急性淋巴细胞白血病与11 q23异常的预后是特别令人沮丧的婴儿。用HLA匹配的亲属供者的造血干细胞进行异基因移植似乎不能改善t（4：11）阳性白血病患者的临床结局。
Background The prognosis and optimum treatment of childhood acute lymphoblastic leukaemia (ALL) with abnormalities of chromosomal band 11q23 are controversial. We aimed to identify prognostic factors that might help in planning future therapy, and to assess the effectiveness of haemopoietic stem-cell transplantation in patients with the t(4;11) translocation, which is associated with a particularly poor outcome.Methods We reviewed data on 497 children and young adults who had ALL with various 11q23 abnormalities, including the translocations t(4;11), t(9;11), and t(11;19). All patients were treated with intensive chemotherapy, with or without haemopoietic stem-cell transplantation in first complete remission, by 11 study groups and single institutions from 1983 to 1995.Findings Age was the most important prognostic factor. In a Cox's proportional-hazard model stratified by 11q23 abnormalities, infants younger than 1 year fared significantly worse than patients 1 year of age or older (hazard ratio for event-free survival 1.84 [95% CI 1.38-2.47], p=0.0001). Among infants, any category of 11q23 abnormality conferred a dismal outcome, whereas in older patients, t(4;11) and t(9;11) were associated with a worse outcome than were other 11q23 changes. In the largest subgroup-256 patients with t(4;11)-any type of transplantation was associated with significantly worse disease-free survival (1.61 [1.10-2.35], p=0.014) and overall survival (1.76 [1.08-2.45], p=0.004) compared with chemotherapy only. Even transplantation with stem cells from HLA-matched related or HLA-matched unrelated donors tended to be associated with a worse outcome than chemotherapy alone.Interpretation The prognosis of acute lymphoblastic leukaemia with an 11q23 abnormality is particularly dismal in infants. Allogeneic transplantation with haemopoietic stem cells from an HLA-matched related donor does not seem to improve the clinical outcome in patients with t(4:11)-positive leukaemia.