Improved outcome for children with acute lymphoblastic leukemia: results of Total Therapy Study XIIIB at St Jude Children's Research Hospital
Improved outcome for children with acute lymphoblastic leukemia: results of Total Therapy Study XIIIB at St Jude Children's Research Hospital
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DOI：
10.1182/blood-2004-04-1616
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发表时间：
2004-11-01
期刊：
影响因子：
20.3
通讯作者：
Evans, WE
中科院分区：
文献类型：
--
作者：
Pui, CH;Sandlund, JT;Evans, WE
圣犹达针对儿童急性淋巴细胞白血病（ALL）的全面治疗研究XIIIB纳入了更严格的风险分类、早期强化鞘内化疗、再诱导治疗以及在缓解后治疗中添加地塞米松，以增加无事件生存者的比例，而不影响其生活质量。对于12%的T细胞ALL患者，如果白细胞计数≥ 100 x 10（9）/L，或CNS-3（5个或更多白细胞/穆尔，无创伤样本中有可识别的原始细胞或存在颅神经麻痹）状态，则保留头颅照射。在入组研究的247例连续患者中，117例被归类为低风险白血病，主要接受以抗代谢药物为基础的持续治疗; 130例高风险白血病患者接受更强化的持续化疗，每周轮换给予多种药物对。5年无事件生存率估计值为80.8% +/- 2.6%（SE）; 8年率为78.6% +/-5.8%。单独中枢神经系统（CNS）复发的5年累积风险为1.7% +/-0.8%，单独加合并CNS复发的5年累积风险为3.0% +/-1.1%。在接受累积剂量为1.2 g/m2的低风险患者中，依托泊苷相关骨髓恶性肿瘤的5年累积风险为1.8% ± 1.3%，在接受累积剂量高达14.4 g/m2的高风险患者中，为5.0% ± 2.0%（p = 0.18）。独立的不良预后特征包括MLL-AF 4或BCR-ABL融合基因的存在以及在6周缓解诱导期结束时0.01%或更多的微小残留白血病。我们的研究结果提示了早期强化鞘内化疗的有效性，并为完全省略颅照射的研究提供了基础。(C)2004年，美国血液学会。
St Jude Total Therapy Study XIIIB for childhood acute lymphoblastic leukemia (ALL) incorporated more stringent risk classification, early intensification of intrathecal chemotherapy, reinduction treatment, and the addition of dexamethasone to postremission therapy to increase the proportion of event-free survivors without jeopardizing their quality of life. Cranial irradiation was reserved for the 12% of patients who had T-cell ALL and a presenting leukocyte count of 100 x 10(9)/L or more, or CNS-3 (5 or more leukocytes/muL with identifiable blast cells in an atraumatic sample or the presence of cranial nerve palsy) status. Among the 247 consecutive patients enrolled in the study, 117 were classified as having lower-risk leukemia and received mainly antimetabolite-based continuation therapy; the 130 cases with higher-risk leukemia received more intensive continuation chemotherapy with multiple drug pairs administered in weekly rotation. The 5-year event-free survival estimate was 80.8% +/- 2.6% (SE); the 8-year rate was 78.6% +/- 5.8%. The 5-year cumulative risk of an isolated central nervous system (CNS) relapse was 1.7% +/- 0.8%, and that of isolated plus combined CNS relapse was 3.0% +/- 1.1%. The 5-year cumulative risks of etoposide-related myeloid malignancies were 1.8% +/- 1.3% in the lower-risk patients who received a cumulative dose of 1.2 g/m(2) and 5.0% +/- 2.0% in the higher-risk patients who received a cumulative dose of up to 14.4 g/m(2) (p = .18). Independent adverse prognostic features included the presence of MLL-AF4 or BCR-ABL fusion gene and minimal residual leukemia of 0.01% or more at the end of the 6-week remission induction phase. Our results suggest the efficacy of early intensification of intrathecal chemotherapy and provide the basis for studies omitting cranial irradiation altogether. (C) 2004 by The American Society of Hematology.