Department of Urology, Peking University First Hospital, Institute of Urology, Peking University, National Urological Cancer Center, Beijing, China.
Clin Genet. 2013 Dec;84(6):581-4. doi: 10.1111/cge.12092. Epub 2013 Feb 5.
von Hippel-Lindau (VHL) disease is an inheritable multisystem tumor syndrome characterized by multiple benign and malignant tumors affecting multiple organs. VHL is the result of a germline mutation in the VHL tumor suppressor gene. Molecular genomic analysis routinely confirms the clinical diagnosis. However, the use of molecular diagnostic methods can often be insufficient for the detection of mosaic germline VHL mutations, making the diagnosis of some cases of VHL difficult. Here, we report the case of a VHL mosaic patient with bilateral renal lesions in the absence of other VHL-associated lesions. A VHL mutation was not originally detected by routine molecular testing. Nonetheless, the detection of a heterozygous c.194C>G (p.Ser65Trp) VHL mutation in the patient's daughter prompted further genetic assessment and eventually resulted in the finding of a mosaic c.194C>G (p.Ser65Trp) VHL mutation in the patient. The mutation rate was 18.8 ± 3.84% in peripheral leukocytes. As the frequency of VHL mosaicism remains underdetermined, the possibility of a diagnosis of mosaic VHL should be considered in patients with both typical and atypical VHL-associated manifestations.
希佩尔-林道(VHL)病是一种可遗传的多系统肿瘤综合征,其特征是多种良性和恶性肿瘤影响多个器官。VHL 是 VHL 肿瘤抑制基因种系突变的结果。分子基因组分析通常可确认临床诊断。然而,分子诊断方法的使用通常不足以检测嵌合种系 VHL 突变,从而使某些 VHL 病例的诊断变得困难。在这里,我们报告了一例 VHL 嵌合体患者,其双侧肾脏存在病变,但无其他 VHL 相关病变。最初通过常规分子检测未检测到 VHL 突变。然而,在患者的女儿中检测到杂合 c.194C>G(p.Ser65Trp)VHL 突变,促使进一步进行遗传评估,最终发现患者存在嵌合 c.194C>G(p.Ser65Trp)VHL 突变。在外周血白细胞中的突变率为 18.8±3.84%。由于 VHL 嵌合体的频率仍不确定,因此对于具有典型和非典型 VHL 相关表现的患者,应考虑诊断为 VHL 嵌合体。