当代以风险为导向的治疗已经改善了患有急性淋巴细胞白血病（ALL）和TCF3 :: PBX1融合基因的患者的预后。在这项研究中，作者试图确定可以用于进一步改善预后的预后因素。作者研究了384例在2015年1月1日至2019年12月31日期间接受中国儿童癌症集团ALL-2015方案治疗的这种基因型的患者。所有患者临时接受中危致密度增强化疗，无需预防性颅内放疗；那些在缓解诱导期第46天（结束时）的最低残留病（MRD）≥1％的患者是造血细胞移植的候选人。总体5年无事件生存率为84.4％（95％置信区间[CI]，80.6-88.3），5年总体生存率为88.9％（95％CI，85.5-92.4）。与较低的5年无事件生存率相关的独立因素包括男性（80.4％[95％CI，74.8-86.4]，比女性中的88.9％[95％CI，84.1-93.9]低，p = .03）和阳性第46天MRD（≥0.01 ％）（62.1％[95％CI，44.2-87.4]，比MRD阴性患者中的87.1％[95％CI，83.4-90.9]低，p <.001）。诊断时睾丸白血病的存在（n = 10）与特别惨淡的5年无事件生存率相关（33.3％[95％CI，11.6-96.1]，比其他192名男性患者中的83.0％[95％CI，77.5-88.9]低，p <.001），并且是一个独立的风险因素（危险比[HR]，5.7;[95％CI，2.2-14.5]，p <.001）。这些数据表明，强化缓解诱导后MRD阳性和诊断时的睾丸白血病是TCF3 :: PBX1 ALL患者新型分子治疗或免疫治疗的指标。©2023年作者。《癌症》由Wiley Periodicals LLC代表美国癌症学会出版。

Contemporary risk-directed treatment has improved the outcome of patients with acute lymphoblastic leukemia (ALL) and TCF3::PBX1 fusion. In this study, the authors seek to identify prognostic factors that can be used to further improve outcome.The authors studied 384 patients with this genotype treated on Chinese Children's Cancer Group ALL-2015 protocol between January 1, 2015 and December 31, 2019. All patients provisionally received intensified chemotherapy in the intermediate-risk arm without prophylactic cranial irradiation; those with high minimal residual disease (MRD) ≥1% at day 46 (end) of remission induction were candidates for hematopoietic cell transplantation.The overall 5-year event-free survival was 84.4% (95% confidence interval [CI], 80.6-88.3) and 5-year overall survival 88.9% (95% CI, 85.5-92.4). Independent factors associated with lower 5-year event-free survival were male sex (80.4%, [95% CI, 74.8-86.4] vs. 88.9%, [95% CI, 84.1-93.9] in female, p = .03) and positive day 46 MRD (≥0.01%) (62.1%, [95% CI, 44.2-87.4] vs. 87.1%, [95% CI, 83.4-90.9] in patients with negative MRD, p < .001). The presence of testicular leukemia at diagnosis (n = 10) was associated with particularly dismal 5-year event-free survival (33.3% [95% CI, 11.6-96.1] vs. 83.0% [95% CI, 77.5-88.9] in the other 192 male patients, p < .001) and was an independent risk factor (hazard ratio [HR], 5.7; [95% CI, 2.2-14.5], p < .001).These data suggest that the presence of positive MRD after intensive remission induction and testicular leukemia at diagnosis are indicators for new molecular therapeutics or immunotherapy in patients with TCF3::PBX1 ALL.© 2023 The Authors. Cancer published by Wiley Periodicals LLC on behalf of American Cancer Society.