Agrawal Gautam, Agarwal Bhawna, Chandrasekhara Pillai Anjana, Kuriakose Kiran
Nephrology, Independence Health System, Greensburg, USA.
Internal Medicine, University of Pittsburgh Medical Center McKeesport Hospital, McKeesport, USA.
Cureus. 2025 Feb 23;17(2):e79498. doi: 10.7759/cureus.79498. eCollection 2025 Feb.
Autosomal dominant polycystic kidney disease (ADPKD) is a hereditary disorder, characterized by the formation of multiple cysts in the kidneys, leading to progressive kidney enlargement and, eventually, renal failure. It is most frequently associated with PKD1 or PKD2 mutations, although rare variants, such as the GANAB gene, are also associated, but they present as a milder renal phenotype. ADPKD patients often present with renal manifestations, such as hypertension, abdominal pain, hematuria, or urinary tract infections, along with extrarenal manifestations, such as liver cysts, heart valve disease, and cerebral aneurysms. ADPKD is usually diagnosed in the fourth or fifth decade of life. This case report discusses the clinical presentation, diagnostic approach, and management of an 18-year-old female patient with no known first-degree family history of ADPKD, who presented with hypertension and bilateral renal cysts on ultrasound. The diagnosis was confirmed by imaging studies and genetic testing. The GANAB gene mutation found in this patient is typically associated with mild kidney disease; however, according to the Mayo Clinic Imaging Classification (MIC) for ADPKD, our patient falls under Classification 1E, which is predictive of rapid progression to end-stage renal disease (ESRD). It highlights the challenges in treating young patients with ADPKD, given the limited studies available for managing this progressive disease in the young population. This case questions the assumption that GANAB-associated ADPKD progresses in a mild manner. Clinicians should prioritize vigilant monitoring and a multidisciplinary approach for young patients with high-risk imaging characteristics, regardless of their genetic findings.
常染色体显性多囊肾病(ADPKD)是一种遗传性疾病,其特征是在肾脏中形成多个囊肿,导致肾脏逐渐增大,并最终发展为肾衰竭。虽然罕见的变异基因,如GANAB基因,也与该病有关,但它最常与PKD1或PKD2基因突变相关,不过由GANAB基因导致的患者肾脏表型较轻。ADPKD患者常出现肾脏表现,如高血压、腹痛、血尿或尿路感染,同时伴有肾外表现,如肝囊肿、心脏瓣膜病和脑动脉瘤。ADPKD通常在40或50岁时被诊断出来。本病例报告讨论了一名18岁女性患者的临床表现、诊断方法和治疗情况,该患者无ADPKD的一级家族病史,超声检查发现患有高血压和双侧肾囊肿。通过影像学研究和基因检测确诊。在该患者中发现的GANAB基因突变通常与轻度肾脏疾病相关;然而,根据梅奥诊所ADPKD影像分类(MIC),我们的患者属于1E类,这预示着将快速进展至终末期肾病(ESRD)。鉴于针对年轻人群中这种进行性疾病的治疗研究有限,这凸显了治疗年轻ADPKD患者的挑战。该病例对GANAB相关的ADPKD以轻度方式进展这一假设提出了质疑。临床医生应优先对具有高风险影像特征的年轻患者进行密切监测,并采取多学科方法进行治疗,无论其基因检测结果如何。