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极低龄婴儿的肾上腺肿块:观察等待是否安全?SIOPEN观察性研究结果报告

Suprarenal Masses in Very Young Infants: Is It Safe to Watch and Wait? Report of a SIOPEN Observational Study Results.

作者信息

Papadakis Vassilios, Segura Vanessa, Conte Massimo, Plantaz Dominique, Di Cataldo Andrea, Schleiermacher Gudrun, Wheeler Kate, Bermúdez Jose D, Ash Shifra, Brichard Bénédicte, Ladenstein Ruth, Combaret Valérie, Sarnacki Sabine, Fagnani Anna Maria, Granata Claudio, Cañete Adela

机构信息

Department of Pediatric Hematology-Oncology, Aghia Sophia Children's Hospital, Levadias Street 8, 11527 Athens, Greece.

Instituto de Investigación Sanitaria La Fe, Fernando Abril Martorell 106, 46026 Valencia, Spain.

出版信息

Cancers (Basel). 2022 Aug 19;14(16):4007. doi: 10.3390/cancers14164007.

DOI:10.3390/cancers14164007
PMID:36011005
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9406882/
Abstract

To assess whether expectant observation of infants ≤ 90 days old with small suprarenal masses (sSRMs) could avoid unnecessary surgery without impacting outcome. Infants ≤ 90 days with a ≤ 5 cm mass, without midline extension or lymph node or distant spread were registered (ClinicalTrials.org:NCT01728155). Once staging was completed, they were followed with ultrasound, MRI and urinary catecholamines. Surgical resection was only planned if there was a ≥40% mass volume increase or for a mass persisting after 48 weeks of the planned observation. Over a 5-year period, 128 infants were registered. No infant had detectable MYCN amplification in the peripheral blood. Surgery was performed in 39 (30.5%) patients, in 18 during and in 21 after the planned 48-week observation, and 74% were confirmed to be neuroblastomas. Non-life-threatening surgical complications occurred in two cases. The 3-year overall survival and event-free survival were 100% and 87.1%, respectively. The 16 events observed were volume increase (N = 11) and progression to neuroblastoma stage MS (N = 5). Patients with solid masses or MIBG-positive masses had lower EFS. : Expectant observation for infants with sSRMs with clinical follow-up and timely imaging (including MRI scan) is safe and effective, allowing surgery to be avoided in the majority of them.

摘要

评估对90日龄及以下患有小肾上腺肿块(sSRM)的婴儿进行观察等待是否可以避免不必要的手术且不影响预后。登记了肿块≤5 cm、无中线延伸、无淋巴结或远处转移的90日龄及以下婴儿(ClinicalTrials.org:NCT01728155)。一旦完成分期,就通过超声、MRI和尿儿茶酚胺对他们进行随访。仅在肿块体积增加≥40%或在计划观察48周后肿块仍持续存在时才计划进行手术切除。在5年期间,登记了128名婴儿。外周血中未检测到婴儿有MYCN扩增。39例(30.5%)患者接受了手术,其中18例在计划的48周观察期间接受手术,21例在观察期后接受手术,74%被确诊为神经母细胞瘤。2例发生了不危及生命的手术并发症。3年总生存率和无事件生存率分别为100%和87.1%。观察到的16个事件为体积增加(N = 11)和进展为神经母细胞瘤MS期(N = 5)。实性肿块或MIBG阳性肿块的患者无事件生存率较低。对于患有sSRM的婴儿,通过临床随访和及时成像(包括MRI扫描)进行观察等待是安全有效的,可使大多数婴儿避免手术。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5798/9406882/c23856fbcf45/cancers-14-04007-g004a.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5798/9406882/7b362f89a0a4/cancers-14-04007-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5798/9406882/9b7746973e99/cancers-14-04007-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5798/9406882/c24d5ac5440c/cancers-14-04007-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5798/9406882/c23856fbcf45/cancers-14-04007-g004a.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5798/9406882/7b362f89a0a4/cancers-14-04007-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5798/9406882/9b7746973e99/cancers-14-04007-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5798/9406882/c24d5ac5440c/cancers-14-04007-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5798/9406882/c23856fbcf45/cancers-14-04007-g004a.jpg

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本文引用的文献

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N Engl J Med. 2017 Mar 9;376(10):905-907. doi: 10.1056/NEJMp1700196. Epub 2017 Feb 8.
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Genomic Copy Number Profiling Using Circulating Free Tumor DNA Highlights Heterogeneity in Neuroblastoma.利用循环游离肿瘤 DNA 进行基因组拷贝数分析突显神经母细胞瘤的异质性。
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A prospective study of expectant observation as primary therapy for neuroblastoma in young infants: a Children's Oncology Group study.一项针对小婴儿神经母细胞瘤期待观察作为一线治疗的前瞻性研究:一项儿童肿瘤协作组研究。
Ann Surg. 2012 Oct;256(4):573-80. doi: 10.1097/SLA.0b013e31826cbbbd.
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Circulating MYCN DNA predicts MYCN-amplification in neuroblastoma.循环中的MYCN DNA可预测神经母细胞瘤中的MYCN扩增。
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Favorable prognosis for patients 12 to 18 months of age with stage 4 nonamplified MYCN neuroblastoma: a Children's Cancer Group Study.12至18个月大的4期非扩增型MYCN神经母细胞瘤患者的预后良好:一项儿童癌症研究组的研究。
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