Michaeli Orli, Kim Sun Young, Mitchell Sarah G, Jongmans Marjolijn C J, Wasserman Jonathan D, Perrino Melissa R, Das Anirban, MacFarland Suzanne P, Scollon Sarah R, Greer Mary-Louise C, Sobreira Nara, Gallinger Bailey, Lupo Philip J, Malkin David, Schneider Kami Wolfe, Schultz Kris Ann P, Foulkes William D, Woodward Emma R, Stewart Douglas R
Division of Hematology and Oncology, Schneider Children's Medical Center of Israel, School of Medicine, Tel Aviv University, Tel Aviv, Israel.
Division of Human Genetics, Department of Pediatrics, Cincinnati Children's Hospital, University of Cincinnati, Cincinnati, Ohio.
Clin Cancer Res. 2025 Feb 3;31(3):457-465. doi: 10.1158/1078-0432.CCR-24-2171.
The management of children with syndromes associated with an increased risk of benign and malignant neoplasms is a complex challenge for health care professionals. The 2023 American Association for Cancer Research Childhood Cancer Predisposition Workshop provided updated consensus guidelines on cancer surveillance in these syndromes, aiming to improve early detection and intervention and reduce morbidity associated with such neoplasms. In this article, we review several of the rare conditions discussed in this workshop. Ollier disease and Maffucci syndrome are enchondromatoses (disorders featuring benign bone lesions) with up to 50% risk of malignancy, including chondrosarcoma. These patients require surveillance with baseline whole-body MRI and routine monitoring of potential malignant transformation of bony lesions. Hereditary multiple osteochondromas carry a lower risk of chondrosarcoma (<6%) but still require lifelong surveillance and baseline imaging. Related syndromes of benign bone lesions are also described. Hereditary leiomyomatosis and renal cell carcinoma syndrome, associated with fumarate hydratase pathogenic variants, is discussed in detail. Surveillance for renal cell carcinoma in pediatric age is recommended, as well as prompt intervention when a lesion is detected. Schinzel-Giedion syndrome and Rubinstein-Taybi syndrome are described for their associated malignancies and other complications, as well as expert consensus on the need for childhood cancer surveillance. Clinical recommendations, including imaging modalities and frequency of screenings, are proposed and are tailored to each syndrome's age-specific tumor risk profile. In all syndromes, patients and their families should be educated about the potential malignancy risk and advised to seek medical care for rapid growth of a mass, persistent pain, or other unexplained symptoms.
对患有与良性和恶性肿瘤风险增加相关综合征的儿童进行管理,对医疗保健专业人员来说是一项复杂的挑战。2023年美国癌症研究协会儿童癌症易感性研讨会提供了关于这些综合征癌症监测的最新共识指南,旨在改善早期发现和干预,并降低与此类肿瘤相关的发病率。在本文中,我们回顾了本次研讨会上讨论的几种罕见病症。骨软骨瘤病(以良性骨病变为特征的疾病)和马富西综合征发生恶性肿瘤(包括软骨肉瘤)的风险高达50%。这些患者需要通过基线全身MRI进行监测,并对骨病变的潜在恶性转化进行常规监测。遗传性多发性骨软骨瘤发生软骨肉瘤的风险较低(<6%),但仍需要终身监测和基线影像学检查。还描述了良性骨病变的相关综合征。详细讨论了与延胡索酸水合酶致病性变异相关的遗传性平滑肌瘤病和肾细胞癌综合征。建议对儿童期肾细胞癌进行监测,以及在检测到病变时及时进行干预。描述了申策尔-吉迪恩综合征和鲁宾斯坦-泰比综合征的相关恶性肿瘤和其他并发症,以及关于儿童癌症监测必要性的专家共识。提出了临床建议,包括成像方式和筛查频率,并针对每种综合征特定年龄的肿瘤风险特征进行了调整。在所有综合征中,都应告知患者及其家属潜在的恶性肿瘤风险,并建议他们在出现肿块快速生长、持续疼痛或其他无法解释的症状时寻求医疗护理。