针对神经纤维瘤病 1 型、努南综合征、CBL 综合征、科斯特洛综合征及相关 RAS 病症患者的儿科癌症监测建议更新。

IF 10.2 1区 医学 Q1 ONCOLOGY Clinical Cancer Research Pub Date : 2024-11-01 DOI:10.1158/1078-0432.CCR-24-1611
Melissa R Perrino, Anirban Das, Sarah R Scollon, Sarah G Mitchell, Mary-Louise C Greer, Marielle E Yohe, Jordan R Hansford, Jennifer M Kalish, Kris Ann P Schultz, Suzanne P MacFarland, Wendy K Kohlmann, Philip J Lupo, Kara N Maxwell, Stefan M Pfister, Rosanna Weksberg, Orli Michaeli, Marjolijn C J Jongmans, Gail E Tomlinson, Jack Brzezinski, Uri Tabori, Gina M Ney, Karen W Gripp, Andrea M Gross, Brigitte C Widemann, Douglas R Stewart, Emma R Woodward, Christian P Kratz
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引用次数: 0

摘要

神经纤维瘤病 1 型(NF1)、努南综合征和相关综合征被归类为 RAS 病,它们是 RAS-MAPK 通路失调的结果,并表现出不同的多系统临床表型。RAS综合征是最常见的遗传性癌症易感综合征之一,需要细致入微的临床管理。与普通人群相比,患有 RAS 病的儿童罹患良性和恶性肿瘤的风险明显增加。近十年来,临床试验表明,靶向治疗可以改善低度良性肿瘤病变的预后,但也存在自身的挑战,这凸显了此类患者(尤其是 NF1 患者)所需的多学科护理。本观点源于 2023 年 AACR 儿童癌症易感性研讨会,旨在更新儿科肿瘤学家、神经学家、遗传学家、咨询师和其他医疗保健专业人士对修订后诊断标准的认识,回顾之前发布的监测指南,并协调针对 NF1 或 RAS 病变患者的最新监测建议。
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Update on Pediatric Cancer Surveillance Recommendations for Patients with Neurofibromatosis Type 1, Noonan Syndrome, CBL Syndrome, Costello Syndrome, and Related RASopathies.

Neurofibromatosis type 1 (NF1), Noonan syndrome, and related syndromes, grouped as RASopathies, result from dysregulation of the RAS-MAPK pathway and demonstrate varied multisystemic clinical phenotypes. Together, RASopathies are among the more prevalent genetic cancer predisposition syndromes and require nuanced clinical management. When compared with the general population, children with RASopathies are at significantly increased risk of benign and malignant neoplasms. In the past decade, clinical trials have shown that targeted therapies can improve outcomes for low-grade and benign neoplastic lesions but have their own challenges, highlighting the multidisciplinary care needed for such individuals, specifically those with NF1. This perspective, which originated from the 2023 American Association for Cancer Research Childhood Cancer Predisposition Workshop, serves to update pediatric oncologists, neurologists, geneticists, counselors, and other health care professionals on revised diagnostic criteria, review previously published surveillance guidelines, and harmonize updated surveillance recommendations for patients with NF1 or RASopathies.

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来源期刊
Clinical Cancer Research
Clinical Cancer Research 医学-肿瘤学
CiteScore
20.10
自引率
1.70%
发文量
1207
审稿时长
2.1 months
期刊介绍: Clinical Cancer Research is a journal focusing on groundbreaking research in cancer, specifically in the areas where the laboratory and the clinic intersect. Our primary interest lies in clinical trials that investigate novel treatments, accompanied by research on pharmacology, molecular alterations, and biomarkers that can predict response or resistance to these treatments. Furthermore, we prioritize laboratory and animal studies that explore new drugs and targeted agents with the potential to advance to clinical trials. We also encourage research on targetable mechanisms of cancer development, progression, and metastasis.
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