Yehia Lamis, Heald Brandie, Eng Charis
Genomic Medicine Institute, Lerner Research Institute, Cleveland Clinic, Cleveland, Ohio.
Invitae, San Francisco, California.
Gastroenterology. 2023 Apr;164(5):800-811. doi: 10.1053/j.gastro.2023.01.026. Epub 2023 Jan 28.
The hamartomatous polyposis syndromes are a set of clinically distinct disorders characterized by the occurrence of hamartomatous polyps in the gastrointestinal tract. These syndromes include juvenile polyposis syndrome, Peutz-Jeghers syndrome, and PTEN hamartoma tumor syndrome. Although each of the syndromes has distinct phenotypes, the hamartomatous polyps can be challenging to differentiate histologically. Additionally, each of these syndromes is associated with increased lifetime risks of gene-specific and organ-specific cancers, including those outside of the gastrointestinal tract. Germline pathogenic variants can be identified in a subset of individuals with these syndromes, which facilitates molecular diagnosis and subsequent gene-enabled management in the setting of genetic counseling. Although the malignant potential of hamartomatous polyps remains elusive, timely recognition of these syndromes is important and enables presymptomatic cancer surveillance and management before symptom exacerbation. Presently, there are no standard agents to prevent the development of polyps and cancers in the hamartomatous polyposis syndromes.
错构瘤性息肉综合征是一组临床特征各异的疾病,其特点是胃肠道出现错构瘤性息肉。这些综合征包括幼年性息肉病综合征、黑斑息肉综合征和PTEN错构瘤肿瘤综合征。尽管每种综合征都有独特的表型,但错构瘤性息肉在组织学上可能难以鉴别。此外,这些综合征中的每一种都与特定基因和特定器官癌症的终生风险增加相关,包括胃肠道以外的癌症。在这些综合征的一部分个体中可以识别出种系致病性变异,这有助于在遗传咨询的背景下进行分子诊断和随后基于基因的管理。尽管错构瘤性息肉的恶性潜能仍不明确,但及时识别这些综合征很重要,能够在症状加重之前进行症状前癌症监测和管理。目前,尚无标准药物可预防错构瘤性息肉综合征中息肉和癌症的发生。