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
{"title":"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。","authors":"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","doi":"10.1002/pbc.32025","DOIUrl":null,"url":null,"abstract":"<div>\n \n \n <section>\n \n <h3> Background</h3>\n \n <p>In neuroblastoma (NB), urinary catecholamine excretion and <sup>123</sup>ImIBG 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 <sup>123</sup>ImIBG avidity, remains to be determined. We sought to determine the frequency and prognosis of atypical NB and investigate the significance of catecholamine profiles and <sup>123</sup>ImIBG avidity at diagnosis.</p>\n </section>\n \n <section>\n \n <h3> Methods</h3>\n \n <p>From 2000 to 2020, 275 children with NB, aged 0–20 years at diagnosis, treated at Institut Curie, France, were retrospectively analyzed.</p>\n </section>\n \n <section>\n \n <h3> Results</h3>\n \n <p>Overall, 24% of NB had atypical features (<i>n</i> = 67/275). Lower INRG stages L1/L2 were more frequent in atypical NB, 66% versus 28% (<i>n</i> = 44/67 vs. 59/208), with less INRG Stage M than in typical NB, 25% versus 61% (<i>n</i> = 17/67 vs. 126/208), <i>p</i> < 0.001. Atypical tumors more frequently harbored favorable molecular features with less frequent <i>MYCN</i> amplification, 12% (<i>n</i> = 8/64) versus 29% (<i>n</i> = 58/201), <i>p</i> < 0.01, and fewer cases with segmental chromosomal alterations, 30% (<i>n</i> = 13/44) versus 60% (<i>n</i> = 69/115), <i>p</i> < 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%, <i>p</i> < 0.001). However, in multivariate analysis, atypical features in NB were not significant independent markers of prognosis.</p>\n </section>\n \n <section>\n \n <h3> Conclusions</h3>\n \n <p>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.</p>\n </section>\n </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3000,"publicationDate":"2025-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32025","citationCount":"0","resultStr":"{\"title\":\"Atypical Neuroblastoma With Absent Urinary Catecholamine Excretion and 123ImIBG Avidity Are of Favorable Outcome: A Retrospective French Single-Center Study\",\"authors\":\"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\",\"doi\":\"10.1002/pbc.32025\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div>\\n \\n \\n <section>\\n \\n <h3> Background</h3>\\n \\n <p>In neuroblastoma (NB), urinary catecholamine excretion and <sup>123</sup>ImIBG 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 <sup>123</sup>ImIBG avidity, remains to be determined. We sought to determine the frequency and prognosis of atypical NB and investigate the significance of catecholamine profiles and <sup>123</sup>ImIBG avidity at diagnosis.</p>\\n </section>\\n \\n <section>\\n \\n <h3> Methods</h3>\\n \\n <p>From 2000 to 2020, 275 children with NB, aged 0–20 years at diagnosis, treated at Institut Curie, France, were retrospectively analyzed.</p>\\n </section>\\n \\n <section>\\n \\n <h3> Results</h3>\\n \\n <p>Overall, 24% of NB had atypical features (<i>n</i> = 67/275). Lower INRG stages L1/L2 were more frequent in atypical NB, 66% versus 28% (<i>n</i> = 44/67 vs. 59/208), with less INRG Stage M than in typical NB, 25% versus 61% (<i>n</i> = 17/67 vs. 126/208), <i>p</i> < 0.001. Atypical tumors more frequently harbored favorable molecular features with less frequent <i>MYCN</i> amplification, 12% (<i>n</i> = 8/64) versus 29% (<i>n</i> = 58/201), <i>p</i> < 0.01, and fewer cases with segmental chromosomal alterations, 30% (<i>n</i> = 13/44) versus 60% (<i>n</i> = 69/115), <i>p</i> < 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%, <i>p</i> < 0.001). However, in multivariate analysis, atypical features in NB were not significant independent markers of prognosis.</p>\\n </section>\\n \\n <section>\\n \\n <h3> Conclusions</h3>\\n \\n <p>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.</p>\\n </section>\\n </div>\",\"PeriodicalId\":19822,\"journal\":{\"name\":\"Pediatric Blood & Cancer\",\"volume\":\"72 11\",\"pages\":\"\"},\"PeriodicalIF\":2.3000,\"publicationDate\":\"2025-09-03\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32025\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Pediatric Blood & Cancer\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1002/pbc.32025\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric Blood & Cancer","FirstCategoryId":"3","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1002/pbc.32025","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
Atypical Neuroblastoma With Absent Urinary Catecholamine Excretion and 123ImIBG Avidity Are of Favorable Outcome: A Retrospective French Single-Center Study
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.
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.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
