赫尔曼斯基-普德拉克综合征
医学
眼白化病
出血素质
白化病
病因学
疾病
无义突变
胡说
皮肤病科
病理
内科学
突变
遗传学
基因
血小板
生物
错义突变
纤维化
肺纤维化
出处
期刊:Cureus
[Cureus, Inc.]
日期:2023-10-30
卷期号:15 (10): e47970-e47970
被引量:2
摘要
Hermansky-Pudlak syndrome (HPS) is a group of 10 autosomal recessive inherited diseases. Most patients exhibit albinism with nystagmus, visual acuity loss, and a platelet storage pool deficiency with bleeding diathesis. The severity and variety of other clinical features depend on the HPS subtype. We report a 24-year-old male with end-stage renal disease (ESRD) of unknown etiology and a history of oculocutaneous albinism and bleeding diathesis. Two of his siblings also had oculocutaneous albinism. The diagnostic workup for renal impairment was unremarkable. Further genetic testing revealed a homozygous novel nonsense mutation in the HPS6 gene. Additionally, a heterozygous variant of uncertain significance was identified in the HPS5 gene. Renal failure is an uncommon clinical feature of HPS. To our knowledge, this is the first case that describes the association of HPS types 5 and 6 with renal failure.
科研通智能强力驱动
Strongly Powered by AbleSci AI