Atypical Neuroblastoma With Absent Urinary Catecholamine Excretion and 123ImIBG Avidity Are of Favorable Outcome: A Retrospective French Single-Center Study

IF 2.3 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer Pub Date : 2025-09-03 DOI:10.1002/pbc.32025

Anna Borovkov, Juliette Assy, Isabelle Aerts, Franck Bourdeaut, Camille Cordero, Valérie Laurence, Amaury Leruste, Sarah Winter, Jean Michon, Daniel Orbach, Hélène Pacquement, Pascale Philippe-Chomette, Gaelle Pierron, Julien Masliah-Planchon, Sylvie Helfre, Isabelle Janoueix-Lerosey, Anne-Sophie Defachelles, Claudia Pasqualini, Nina Jehanno, Joanna Cyrta, Arnaud Gauthier, Véronique Mosseri, François Doz, Sabine Sarnacki, Liesbeth Cardoen, Hervé J. Brisse, Marie Luporsi, Gudrun Schleiermacher

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引用次数: 0

Abstract

Background

In neuroblastoma (NB), urinary catecholamine excretion and ¹²³ImIBG avidity—depending on tumor enzymatic activity and norepinephrine transporter expression, respectively—are diagnostic standards. The prognostic impact of atypical NB, without urinary catecholamine excretion and/or ¹²³ImIBG avidity, remains to be determined. We sought to determine the frequency and prognosis of atypical NB and investigate the significance of catecholamine profiles and ¹²³ImIBG avidity at diagnosis.

Methods

From 2000 to 2020, 275 children with NB, aged 0–20 years at diagnosis, treated at Institut Curie, France, were retrospectively analyzed.

Results

Overall, 24% of NB had atypical features (n = 67/275). Lower INRG stages L1/L2 were more frequent in atypical NB, 66% versus 28% (n = 44/67 vs. 59/208), with less INRG Stage M than in typical NB, 25% versus 61% (n = 17/67 vs. 126/208), p < 0.001. Atypical tumors more frequently harbored favorable molecular features with less frequent MYCN amplification, 12% (n = 8/64) versus 29% (n = 58/201), p < 0.01, and fewer cases with segmental chromosomal alterations, 30% (n = 13/44) versus 60% (n = 69/115), p < 0.05. Event-free survival (EFS) and overall survival (OS) were better in atypical than typical NB (5-year EFS: 77% ± 5% vs. 50% ± 4% and OS 87% ± 4% vs. 65% ± 4%, p < 0.001). However, in multivariate analysis, atypical features in NB were not significant independent markers of prognosis.

Conclusions

Atypical NB constitute a subgroup of interest for biomolecular analyses, including transcriptomics, which might provide further insights into disease-associated molecular features and our understanding of NB development.

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不典型神经母细胞瘤伴尿儿茶酚胺排泄缺失和123ImIBG发生率是有利的结果：一项回顾性法国单中心研究。

背景：在神经母细胞瘤（NB）中，尿儿茶酚胺排泄量和123ImIBG浓度——分别取决于肿瘤酶活性和去甲肾上腺素转运蛋白的表达——是诊断标准。不典型NB的预后影响，没有尿儿茶酚胺排泄和/或123ImIBG，仍有待确定。我们试图确定非典型NB的频率和预后，并探讨儿茶酚胺谱和123ImIBG在诊断中的意义。方法：回顾性分析2000年至2020年在法国居里研究所治疗的0 ~ 20岁NB患儿275例。结果：总体而言，24%的NB具有不典型特征（n = 67/275）。INRG L1/L2期较低在非典型NB中更为常见，分别为66%和28% (n = 44/67和59/208)，INRG M期较典型NB少，分别为25%和61% (n = 17/67和126/208),p < 0.001。非典型肿瘤更常具有有利的分子特征，MYCN扩增频率较低，分别为12% （n = 8/64）和29% (n = 58/201), p < 0.01；段性染色体改变较少，分别为30% （n = 13/44）和60% (n = 69/115), p < 0.05。非典型NB的无事件生存期（EFS）和总生存期（OS）优于典型NB（5年EFS: 77%±5% vs 50%±4%，OS: 87%±4% vs 65%±4%,p < 0.001）。然而，在多变量分析中，NB的非典型特征并不是预后的重要独立标志。结论：非典型NB构成了一个生物分子分析感兴趣的亚群，包括转录组学，这可能为疾病相关的分子特征和我们对NB发展的理解提供进一步的见解。

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来源期刊

Pediatric Blood & Cancer 医学-小儿科

CiteScore

4.90

自引率

9.40%

发文量

546

审稿时长

1.5 months

期刊介绍： Pediatric Blood & Cancer publishes the highest quality manuscripts describing basic and clinical investigations of blood disorders and malignant diseases of childhood including diagnosis, treatment, epidemiology, etiology, biology, and molecular and clinical genetics of these diseases as they affect children, adolescents, and young adults. Pediatric Blood & Cancer will also include studies on such treatment options as hematopoietic stem cell transplantation, immunology, and gene therapy.