Department of Pediatrics, Texas Tech University Health Sciences, Lubbock, Texas.
Pediatric Hematology, Oncology and Stem Cell Transplantation, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.
Pediatr Blood Cancer. 2020 Oct;67(10):e28390. doi: 10.1002/pbc.28390. Epub 2020 Jul 25.
The 1993 International Neuroblastoma Response Criteria (INRC) were revised in 2017 to include modern functional imaging studies and methods for quantifying disease in bone marrow. We hypothesized the 2017 INRC would enable more precise assessment of response to treatment and provide superior prognostic information compared with the 1993 criteria.
High-risk (HR) neuroblastoma patients from two institutions in Chicago diagnosed between 2006 and 2016 were identified. Patients were assessed post induction chemotherapy via the 1993 and 2017 INRC and classified as responder (≥ mixed response [MXR] or ≥ minor response [MR], respectively) or nonresponder (< MXR or < MR). Event-free survival (EFS) and overall survival (OS) for responders versus nonresponders were determined from end induction and stratified by Cox regression. Patients with progressive disease at end induction were eliminated from the EFS analyses but included in the OS analysis.
The 1993 criteria classified 52 of the 60 HR patients as responders, whereas 54 responders were identified using the 2017 criteria (Spearman correlation r = 0.82, P < 0.001). No statistically significant difference in EFS was observed for responders versus nonresponders using either criteria (P = 0.48 and P = 0.08). However, superior OS was observed for responders (P = 0.01) using either criteria. Both criteria were sensitive in identifying responders among those with good outcomes. The specificity to identify nonresponders among those with poor outcomes was poor.
In HR neuroblastoma, end-induction response defined by the 1993 or 2017 INRC is associated with survival. Larger cohorts are needed to determine if the 2017 INRC provides more precise prognostication.
1993 年国际神经母细胞瘤反应标准（INRC）于 2017 年进行了修订，纳入了现代功能成像研究和骨髓疾病定量方法。我们假设 2017 年 INRC 将能够更准确地评估治疗反应，并提供比 1993 年标准更好的预后信息。
在芝加哥的两个机构诊断出的 2006 年至 2016 年期间的高危（HR）神经母细胞瘤患者被确定。通过 1993 年和 2017 年 INRC 对患者进行诱导化疗后评估，并分别归类为应答者（≥混合反应[MXR]或≥次要反应[MR]）或非应答者（<MXR 或<MR）。从诱导结束时开始，通过 Cox 回归确定应答者与非应答者的无事件生存（EFS）和总生存（OS）。在诱导结束时进展性疾病的患者被排除在 EFS 分析之外，但纳入 OS 分析。
1993 年标准将 60 名 HR 患者中的 52 名归类为应答者，而 2017 年标准将 54 名应答者归类为应答者（Spearman 相关系数 r=0.82，P<0.001）。使用两种标准，应答者与非应答者之间的 EFS 均无统计学差异（P=0.48 和 P=0.08）。然而，使用任何标准，应答者的 OS 均较好（P=0.01）。两种标准均能在预后良好的患者中敏感地识别出应答者。在预后不良的患者中，识别非应答者的特异性较差。
在 HR 神经母细胞瘤中，通过 1993 年或 2017 年 INRC 定义的诱导结束时反应与生存相关。需要更大的队列来确定 2017 年 INRC 是否提供更准确的预后预测。
