Pediatric Adrenocortical Carcinoma: The Nuts and Bolts of Diagnosis and Treatment and Avenues for Future Discovery

IF 2.5 4区医学 Q3 ONCOLOGY

Cancer Management and Research Pub Date : 2024-09-07 DOI:10.2147/cmar.s348725

Allison F O'Neill, Raul C Ribeiro, Emilia M Pinto, Michael R Clay, Gerard P Zambetti, Brent A Orr, Christopher B Weldon, Carlos Rodriguez-Galindo

{"title":"Pediatric Adrenocortical Carcinoma: The Nuts and Bolts of Diagnosis and Treatment and Avenues for Future Discovery","authors":"Allison F O'Neill, Raul C Ribeiro, Emilia M Pinto, Michael R Clay, Gerard P Zambetti, Brent A Orr, Christopher B Weldon, Carlos Rodriguez-Galindo","doi":"10.2147/cmar.s348725","DOIUrl":null,"url":null,"abstract":"Abstract: Adrenocortical tumors (ACTs) are infrequent neoplasms in children and adolescents and are typically associated with clinical symptoms reflective of androgen overproduction. Pediatric ACTs typically occur in the context of a germline TP53 mutation, can be cured when diagnosed at an early stage, but are difficult to treat when advanced or associated with concurrent TP53 and ATRX alterations. Recent work has demonstrated DNA methylation patterns suggestive of prognostic significance. While current treatment standards rely heavily upon surgical resection, chemotherapy, and hormonal modulation, small cohort studies suggest promise for multi-tyrosine kinases targeting anti-angiogenic pathways or immunomodulatory therapies. Future work will focus on novel risk stratification algorithms and combination therapies intended to mitigate toxicity for patients with perceived low-risk disease while intensifying therapy or accelerating discoveries aimed at improving survival for patients with difficult-to-treat disease. Keywords: adrenocortical, pediatric, management, discovery, carcinoma, tumors ","PeriodicalId":9479,"journal":{"name":"Cancer Management and Research","volume":"55 1","pages":""},"PeriodicalIF":2.5000,"publicationDate":"2024-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cancer Management and Research","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.2147/cmar.s348725","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"ONCOLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

Abstract: Adrenocortical tumors (ACTs) are infrequent neoplasms in children and adolescents and are typically associated with clinical symptoms reflective of androgen overproduction. Pediatric ACTs typically occur in the context of a germline TP53 mutation, can be cured when diagnosed at an early stage, but are difficult to treat when advanced or associated with concurrent TP53 and ATRX alterations. Recent work has demonstrated DNA methylation patterns suggestive of prognostic significance. While current treatment standards rely heavily upon surgical resection, chemotherapy, and hormonal modulation, small cohort studies suggest promise for multi-tyrosine kinases targeting anti-angiogenic pathways or immunomodulatory therapies. Future work will focus on novel risk stratification algorithms and combination therapies intended to mitigate toxicity for patients with perceived low-risk disease while intensifying therapy or accelerating discoveries aimed at improving survival for patients with difficult-to-treat disease.

Keywords: adrenocortical, pediatric, management, discovery, carcinoma, tumors

查看原文本刊更多论文

小儿肾上腺皮质癌：诊断和治疗的要点以及未来探索的途径

摘要：肾上腺皮质肿瘤（ACTs）是儿童和青少年中不常见的肿瘤，通常伴有反映雄激素过度分泌的临床症状。小儿肾上腺皮质肿瘤通常发生在TP53基因突变的情况下，早期诊断可以治愈，但晚期或同时伴有TP53和ATRX基因改变时则难以治疗。最近的研究表明，DNA 甲基化模式提示了预后意义。虽然目前的治疗标准主要依赖于手术切除、化疗和激素调节，但小规模队列研究表明，针对抗血管生成途径或免疫调节疗法的多酪氨酸激酶疗法大有可为。未来的工作将集中在新的风险分层算法和联合疗法上，旨在减轻低风险疾病患者的毒性，同时加强治疗或加速发现旨在改善难治性疾病患者生存率的疗法。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Cancer Management and Research Medicine-Oncology

CiteScore

7.40

自引率

0.00%

发文量

448

审稿时长

16 weeks

期刊介绍： Cancer Management and Research is an international, peer reviewed, open access journal focusing on cancer research and the optimal use of preventative and integrated treatment interventions to achieve improved outcomes, enhanced survival, and quality of life for cancer patients. Specific topics covered in the journal include: ◦Epidemiology, detection and screening ◦Cellular research and biomarkers ◦Identification of biotargets and agents with novel mechanisms of action ◦Optimal clinical use of existing anticancer agents, including combination therapies ◦Radiation and surgery ◦Palliative care ◦Patient adherence, quality of life, satisfaction The journal welcomes submitted papers covering original research, basic science, clinical & epidemiological studies, reviews & evaluations, guidelines, expert opinion and commentary, and case series that shed novel insights on a disease or disease subtype.