Language

English

Publication Date

2-3-2025

Journal

Clinical Cancer Research

DOI

10.1158/1078-0432.CCR-24-2171

PMID

39601780

PMCID

PMC11790369

PubMedCentral® Posted Date

8-3-2025

PubMedCentral® Full Text Version

Author MSS

Abstract

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.

Keywords

Child, Humans, Early Detection of Cancer, Genetic Predisposition to Disease, Leiomyomatosis, Neoplastic Syndromes, Hereditary, Rare Diseases, Skin Neoplasms, Uterine Neoplasms, Ollier disease, Maffucci Syndrome, Hereditary multiple osteochondromas, hereditary leiomyomatosis and renal cell carcinoma syndrome, Schinzel Giedion syndrome, Rubinstein-Taybi Syndrome, cancer, surveillance, predispositions, genetics

Published Open-Access

yes

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