UCSF Benioff Children's Hospital and Departments of Pediatrics, University of California, San Francisco School of Medicine, San Francisco, California, USA.
Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston, Massachusetts, USA.
Pediatr Blood Cancer. 2022 Sep;69(9):e29616. doi: 10.1002/pbc.29616. Epub 2022 Feb 21.
We sought to analyze biologic, clinical, and prognostic differences according to pattern of failure at the time of first relapse in neuroblastoma.
Children <21 years diagnosed with neuroblastoma between 1989 and 2017 with known site of first relapse (isolated local vs. distant only vs. combined local and distant sites) were identified from the International Neuroblastoma Risk Group (INRG) database. Data were compared between sites of relapse according to clinical features, biologic features, initial treatment, time to first relapse, and overall survival (OS) from time of first relapse.
Pattern of first relapse among 1833 children was 19% isolated local; 65% distant only; and 16% combined sites. All evaluated clinical and biologic variables with exception of tumor diagnosis differed statistically by relapse pattern, with patients with isolated local failure having more favorable prognostic features. Patients with stage 3 disease were more likely to have isolated local failure compared to all other stages (49% vs. 16%; p < .001). OS significantly differed by relapse pattern (5-year OS ± SE): isolated local: 64% ± 3%; distant only: 23% ± 2%; and combined: 26% ± 4% (p < .001). After controlling for age, stage, and MYCN status, patients with isolated local failure (adjusted hazard ratio [HR] = 0.46; 95% confidence interval [CI]: 0.33-0.62; p < .001) and distant-only failure (adjusted HR = 0.57; 95% CI: 0.45-0.71; p < .001) remained at decreased risk for death as compared to patients with combined failure.
Patients with distant-only and combined failures have a higher proportion of unfavorable clinical and biological features, and a lower survival than those with isolated local relapse.
我们旨在根据神经母细胞瘤首次复发时的失败模式，分析生物学、临床和预后差异。
从国际神经母细胞瘤风险组（INRG）数据库中确定了 1989 年至 2017 年间诊断为神经母细胞瘤且已知首次复发部位（孤立局部 vs. 仅远处转移 vs. 局部和远处联合部位）的<21 岁儿童。根据复发部位比较了临床特征、生物学特征、初始治疗、首次复发时间和从首次复发开始的总生存期（OS）。
1833 例儿童中，首次复发模式为 19%孤立局部；65%仅远处转移；16%为联合部位。除肿瘤诊断外，所有评估的临床和生物学变量均因复发模式而存在统计学差异，孤立局部失败患者具有更有利的预后特征。与所有其他分期相比，3 期疾病患者更有可能出现孤立局部失败（49% vs. 16%；p<.001）。复发模式的 OS 显著不同（5 年 OS ± SE）：孤立局部：64% ± 3%；仅远处转移：23% ± 2%；联合部位：26% ± 4%（p<.001）。在控制年龄、分期和 MYCN 状态后，孤立局部失败（调整后的危险比[HR] = 0.46；95%置信区间[CI]：0.33-0.62；p<.001）和仅远处转移失败（调整后的 HR = 0.57；95% CI：0.45-0.71；p<.001）的患者死亡风险仍低于联合失败的患者。
与孤立局部复发的患者相比，仅远处转移和联合失败的患者具有更多不良的临床和生物学特征，生存率更低。
