Pui C H, Behm F G, Downing J R, Hancock M L, Shurtleff S A, Ribeiro R C, Head D R, Mahmoud H H, Sandlund J T, Furman W L
Department of Hematology-Oncology, St Jude Children's Research Hospital, Memphis, TN 38101-0318.
J Clin Oncol. 1994 May;12(5):909-15. doi: 10.1200/JCO.1994.12.5.909.
Leukemic cell characteristics were analyzed in infants less than 1 year of age with acute lymphoblastic leukemia (ALL) to determine adverse prognostic factors that might explain the poor prognosis of this group.
Treatment outcomes were analyzed according to the presenting clinical and laboratory features of 30 infants treated between May 1979 and April 1993. A stepwise multivariate regression model was used to identify the most important prognostic indicator with respect to event-free survival.
Infant ALL cases were characterized by high presenting leukocyte count (median, 87 x 10(9)/L), increased frequency of CNS leukemia (50%), and blast cells with a CD10- phenotype (67%), myeloid-associated antigen expression (48%), and 11q23/MLL rearrangement (68%). The 11q23/MLL involvement was correlated with age less than 6 months, CD10- phenotype, myeloid-associated antigen expression, and high leukocyte count. Although 11q23/MLL involvement, age less than 6 months, myeloid-associated antigen expression, and female sex were each significantly associated with an inferior treatment outcome, only rearranged 11q23/MLL emerged as an independent predictor of prognosis in multivariate analysis (P = .01). Infants with this genetic abnormality had a 4.7-fold (95% confidence interval, 1.3- to 17.0-fold) increased risk in adverse events compared to other infants.
The 11q23/MLL involvement of blast cells identifies a major subgroup of infant ALL cases that require an innovative treatment approach. Infants who lack this genetic abnormality have an intermediate prognosis and could be treated accordingly on risk-directed protocols.
分析1岁以下急性淋巴细胞白血病（ALL）婴儿的白血病细胞特征，以确定可能解释该组预后不良的不良预后因素。
根据1979年5月至1993年4月期间接受治疗的30例婴儿的临床和实验室特征分析治疗结果。采用逐步多因素回归模型确定无事件生存最重要的预后指标。
婴儿ALL病例的特征为就诊时白细胞计数高（中位数，87×10⁹/L）、中枢神经系统白血病发生率增加（50%），以及具有CD10⁻表型（67%）、髓系相关抗原表达（48%）和11q23/MLL重排（68%）的原始细胞。11q23/MLL受累与6个月以下年龄、CD10⁻表型、髓系相关抗原表达和高白细胞计数相关。尽管11q23/MLL受累、6个月以下年龄、髓系相关抗原表达和女性性别均与较差的治疗结果显著相关，但在多因素分析中，只有重排的11q23/MLL是独立的预后预测指标（P = 0.01）。与其他婴儿相比，具有这种基因异常的婴儿发生不良事件的风险增加4.7倍（95%置信区间，1.3至17.0倍）。
原始细胞的11q23/MLL受累确定了婴儿ALL病例的一个主要亚组，需要创新的治疗方法。缺乏这种基因异常的婴儿预后中等，可根据风险导向方案进行相应治疗。
