Outcome of risk-based therapy for infant acute lymphoblastic leukemia with or without an MLL gene rearrangement, with emphasis on late effects: A final report of two consecutive studies, MLL96 and MLL98, of the Japan Infant Leukemia Study Group
Outcome of risk-based therapy for infant acute lymphoblastic leukemia with or without an MLL gene rearrangement, with emphasis on late effects: A final report of two consecutive studies, MLL96 and MLL98, of the Japan Infant Leukemia Study Group
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DOI：
10.1038/sj.leu.2404903
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发表时间：
2007-11-01
期刊：
影响因子：
11.4
通讯作者：
Ishii, E.
中科院分区：
文献类型：
--
作者：
Tomizawa, D.;Koh, K.;Ishii, E.
我们评估了一种治疗策略的疗效，在这种治疗策略中，急性淋巴细胞白血病（ALL）婴儿根据他们的MLL基因状态分层，然后分配到不同的基于风险的治疗方法。在1995年至2001年期间，共有102名患者注册参加了两个连续的多中心试验，指定为MLL96和MLL98。MLL基因重排患者（MLL- r, n = 80）接受强化化疗，然后进行造血干细胞移植（HSCT），而种系MLL患者（MLL- g, n = 22）接受单独化疗。102名婴儿的5年无事件生存率（EFS）为50.9%（95%可信区间为41.0-60.8%）。最突出的晚期效应是生长障碍，在MLL-R组中58.9%的可评估患者中观察到。与先前报道的结果相比，这种基于风险的治疗方案似乎改善了ALL患儿的总体预后。然而，MLL重排患者中超过一半的事件发生在HSCT启动之前，HSCT相关的毒性事件占移植后事件的36.3%(8/22)，这表明在MLL- r组中进一步分层和开发更有效的早期强化化疗将需要在充分发挥该策略的潜力之前实现。
We evaluated the efficacy of a treatment strategy in which infants with acute lymphoblastic leukemia (ALL) were stratified by their MLL gene status and then assigned to different risk-based therapies. A total of 102 patients were registered on two consecutive multicenter trials, designated MLL96 and MLL98, between 1995 and 2001. Those with a rearranged MLL gene (MLL-R, n = 80) were assigned to receive intensive chemotherapy followed by hematopoietic stem cell transplantation (HSCT), while those with germline MLL (MLL-G, n = 22) were treated with chemotherapy alone. The 5-year event-free survival (EFS) rate for all 102 infants was 50.9% (95% confidence interval, 41.0-60.8%). The most prominent late effect was growth impairment, observed in 58.9% of all evaluable patients in the MLL-R group. This plan of risk-based therapy appears to have improved the overall prognosis for infants with ALL, compared with previously reported results. However, over half the events in patients with MLL rearrangement occurred before the instigation of HSCT, and that HSCT-related toxic events comprised 36.3% (8/22) of post-transplantation events, suggesting that further stratification within the MLL-R group and the development of more effective early-phase intensification chemotherapy will be needed before the full potential of this strategy is realized.