Shuster J J, Falletta J M, Pullen D J, Crist W M, Humphrey G B, Dowell B L, Wharam M D, Borowitz M
University of Florida, POG Statistical Office, Gainesville.
Blood. 1990 Jan 1;75(1):166-73.
Two hundred fifty-three children with newly diagnosed T-cell acute lymphoblastic leukemia (ALL), who were treated uniformly with modified LSA2L2 therapy, were evaluated using univariate and recursive partition analyses to define clinical or biologic features associated with risk of treatment failure. Overall event-free survival (EFS) at 4 years was 43% (SE = 4%). Factors examined included white blood cell (WBC) level, age, gender, race (black v other), presence of a mediastinal mass, hepatomegaly, splenomegaly, marked lymphadenopathy, hemoglobin level, platelet count, blast cell expression of antigens such as the common acute lymphoblastic leukemia antigen (CALLA, CD10), HLA-DR, and T-cell-associated antigens (CD3, CD4, CD8, CD7, CD5, and THY). Univariate analysis showed that age less than or equal to 5 or less than or equal to 7 years, WBC level less than 10, less than 25, less than 50 or less than 100 x 10(3)/microL, and blast cell expression of CD4, CD8, or CALLA were associated with significantly better EFS, while hepatomegaly and splenomegaly were associated with worse EFS. Recursive partitioning analysis showed that the most important single favorable prognostic factor was a WBC level less than 50 x 10(3)/microL and, for patients with WBC counts below this level, the most important predictor of EFS was blast cell expression of the pan-T antigen defined by the monoclonal antibody (MoAb), L17F12 (CD5). For patients with higher WBC levels, the most important predictor of EFS was blast cell expression of THY antigen. The recursive partitioning analysis defined three groups of patients with widely varied prognoses identified as follows: (1) those with a WBC count less than 50 x 10(3)/microL who lacked massive splenomegaly and had blasts expressing CD5 had the best prognosis (66%, SE = 7%, EFS 4 years, n = 84); (2) those with (b1) WBC counts less than 50 x 10(3)/microL with either massive splenomegaly or who had blasts lacking CD5 expression, or (b2) WBC counts greater than 50 x 10(3)/microL with expression of the THY antigen had an intermediate prognosis (39%, SE = 7% EFS at 4 years, n = 94); (3) those with WBC counts greater than 50 x 10(3)/microL and whose blasts lacked expression of THY antigen had the poorest outcome (EFS = 19% at 4 years, SE = 8%, n = 63). A three-way comparison of EFS according to these groupings showed significant differences among the three patient groups (P less than .001). The recursive partitioning was able to classify 241 (95%) of the patients.(ABSTRACT TRUNCATED AT 400 WORDS)
253例新诊断的T细胞急性淋巴细胞白血病（ALL）患儿接受了改良LSA2L2疗法的统一治疗，采用单因素分析和递归划分分析来确定与治疗失败风险相关的临床或生物学特征。4年时的总体无事件生存率（EFS）为43%（标准误=4%）。所检查的因素包括白细胞（WBC）水平、年龄、性别、种族（黑人与其他种族）、纵隔肿块的存在、肝肿大、脾肿大、显著淋巴结病、血红蛋白水平、血小板计数、原始细胞对抗原的表达，如常见急性淋巴细胞白血病抗原（CALLA，CD10）、HLA-DR以及T细胞相关抗原（CD3、CD4、CD8、CD7、CD5和THY）。单因素分析显示，年龄小于或等于5岁或小于或等于7岁、WBC水平小于10、小于25、小于50或小于100×10³/μL，以及原始细胞表达CD4、CD8或CALLA与显著更好的EFS相关，而肝肿大和脾肿大与更差的EFS相关。递归划分分析表明，最重要的单一有利预后因素是WBC水平小于50×10³/μL，对于WBC计数低于该水平的患者，EFS的最重要预测因素是由单克隆抗体（MoAb）L17F12（CD5）定义的全T抗原在原始细胞中的表达。对于WBC水平较高的患者，EFS的最重要预测因素是THY抗原在原始细胞中的表达。递归划分分析确定了三组预后差异很大的患者，如下：（1）WBC计数小于50×10³/μL且无巨大脾肿大且原始细胞表达CD5的患者预后最佳（66%，标准误=7%，4年EFS，n=84）；（2）（b1）WBC计数小于50×10³/μL但有巨大脾肿大或原始细胞缺乏CD5表达的患者，或（b2）WBC计数大于50×10³/μL且表达THY抗原的患者预后中等（4年EFS为39%，标准误=7%，n=94）；（3）WBC计数大于50×10³/μL且原始细胞缺乏THY抗原表达的患者预后最差（4年EFS=19%，标准误=8%，n=63）。根据这些分组进行的EFS的三方比较显示，三组患者之间存在显著差异（P＜0.001）。递归划分能够对241例（95%）患者进行分类。（摘要截于400字）
