Schneider Meike, Dinkelborg Katja, Xiao Xiuli, Chan-Smutko Gayun, Hruska Kathleen, Huang Dongli, Sagar Pallavi, Harisinghani Mukesh, Iliopoulos Othon
Massachusetts General Hospital Cancer Center, Boston, MA, 02114, USA.
Harvard Medical School, Boston, MA, 02115, USA.
Fam Cancer. 2018 Jan;17(1):135-139. doi: 10.1007/s10689-017-0008-8.
Birt-Hogg-Dube (BHD) disease is an autosomal dominant cancer syndrome characterized by benign skin tumors, renal cancer and spontaneous pneumothorax and is caused by mutations in the Folliculin (FLCN) gene. Benign skin tumors and pneumothorax occur in the majority of patients affected by BHD syndrome, but only 30-45% of them develop renal cell carcinoma (RCC) with a median age of diagnosis at 48. The earliest onset of RCC in a BHD patient has been reported at age 20. Here we report a case of a 14 year-old patient with germline FLCN mutation leading to an early-onset bulky RCC that could not be classified strictly according to existing histological types. Germline genetic testing revealed a deletion at FLCN exon 5. The father of the patient was identified as the asymptomatic carrier. We report the youngest patient with BHD-related RCC. This early onset presentation supports genetic testing of at-risk patients and initiation of imaging surveillance for RCC in early adolescence. In addition, future studies are necessary to understand the determinants of reduced penetrance in BHD disease.
Birt-Hogg-Dube(BHD)综合征是一种常染色体显性遗传癌症综合征,其特征为良性皮肤肿瘤、肾癌和自发性气胸,由卵泡抑素(FLCN)基因突变引起。大多数受BHD综合征影响的患者会出现良性皮肤肿瘤和气胸,但其中只有30%-45%会发展为肾细胞癌(RCC),诊断的中位年龄为48岁。据报道,BHD患者中最早发生RCC的年龄为20岁。在此,我们报告一例14岁的患者,其种系FLCN基因突变导致早期发生体积较大的RCC,无法严格按照现有的组织学类型进行分类。种系基因检测显示FLCN外显子5缺失。该患者的父亲被确定为无症状携带者。我们报告了最年轻的与BHD相关的RCC患者。这种早期发病表现支持对高危患者进行基因检测,并在青春期早期开始对RCC进行影像学监测。此外,有必要开展进一步研究以了解BHD综合征中基因外显率降低的决定因素。