Division of Oncology, Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 3012, 3501 Civic Center Boulevard, Philadelphia, PA 19104, USA.
Eur J Pediatr. 2011 Mar;170(3):285-94. doi: 10.1007/s00431-010-1377-2. Epub 2011 Jan 6.
Improvements in our understanding of the genetic basis of human disease and increased utilization of genetic testing have identified a variety of heritable disorders associated with the onset of benign or malignant neoplasms during childhood. In many cases, the optimal management of affected children is dependent upon the early detection and treatment of tumors. Surveillance strategies based on the natural history of these lesions are often complex, requiring clinical examinations and radiologic and laboratory studies that evolve over a patient's lifetime. A general pediatrician may be the first to suspect one of these disorders in a patient, or may be faced with questions regarding genetic testing, cancer risk, and cancer screening. The pediatrician may also coordinate and interpret the results of specific surveillance studies. In this review, we present the genetic etiology, presentation, natural history, and surveillance recommendations for four disparate hereditary tumor predisposing syndromes, including Beckwith-Wiedemann syndrome/idiopathic hemihyperplasia, von Hippel-Lindau disease, Li-Fraumeni syndrome, and rhabdoid tumor/schwannomatosis. These examples are meant to offer the clinician practical recommendations as well as a framework upon which to base the understanding and management of other conditions associated with an increased risk to develop tumors in childhood.
我们对人类疾病遗传基础的理解不断提高,基因检测的应用也越来越广泛,这使得人们发现了多种遗传性疾病,这些疾病可导致儿童期良性或恶性肿瘤的发生。在许多情况下,儿童肿瘤的最佳治疗方案取决于肿瘤的早期发现和治疗。基于这些病变自然史的监测策略通常较为复杂,需要进行临床检查以及影像学和实验室研究,这些检查和研究贯穿患者的整个生命周期。一般儿科医生可能是第一个怀疑患者存在这些疾病的人,或者可能需要面对有关基因检测、癌症风险和癌症筛查的问题。儿科医生还可以协调和解释特定监测研究的结果。在这篇综述中,我们介绍了四种不同的遗传性肿瘤易感综合征(包括 Beckwith-Wiedemann 综合征/特发性半身肥大、von Hippel-Lindau 病、Li-Fraumeni 综合征和横纹肌瘤/许旺细胞瘤)的遗传病因、表现、自然史和监测建议。这些例子旨在为临床医生提供实用的建议,并为理解和管理与儿童期肿瘤发生风险增加相关的其他疾病提供一个框架。