Clinical and Genetic Characteristics of Paroxysmal Kinesigenic Dyskinesia: A Single‐Center Study and Literature Review

阵发性运动障碍 医学 遗传异质性 表型 遗传诊断 临床表型 医学遗传学 发病年龄 基因检测 儿科 单中心 生物信息学 遗传性疾病 运动障碍 内科学 梅德林 人类遗传学
作者
M Li,Dandan Tan,Yu Zhu,Ying Xiong,Min Zhu,Meihong Zhou,Daojun Hong,Yusen Qiu
出处
期刊:The FASEB Journal [Wiley]
卷期号:40 (2): e71479-e71479
标识
DOI:10.1096/fj.202502596r
摘要

ABSTRACT Paroxysmal kinesigenic dyskinesia (PKD) is a genetically heterogeneous movement disorder primarily associated with PRRT2 variants. Recently, TMEM151A and KCNJ10 have emerged as additional PKD‐associated genes. However, genotype–phenotype correlations remain poorly defined. In this study, we retrospectively analyzed 41 PKD patients from a single center in Southeastern China. All patients underwent comprehensive clinical evaluation and whole‐exome sequencing (WES), with variant classification based on ACMG guidelines. Additionally, we conducted a literature review of PKD cohorts published since 2021 to compare the clinical characteristics of patients carrying PRRT2 , TMEM151A , KCNJ10 variants, and those without identified mutations. A genetic diagnosis was achieved in 19/41 patients (46.3%), with PRRT2 being the most frequent. We identified five novel variants, including two in KCNJ10, two in TMEM151A , and one in PNKD . Compared to other groups, PRRT2 ‐positive patients had the earliest onset and highest treatment response. TMEM151A ‐positive patients tended to exhibit more frequent attacks and a lower response to carbamazepine. KCNJ10 ‐positive patients presented with later onset and ultra‐brief attacks. Genetically negative cases displayed distinct features, including fewer auras and more unilateral, ultra‐brief episodes, yet responded well to carbamazepine. PKD exhibits significant genotype‐dependent clinical heterogeneity. Novel variants in TMEM151A and KCNJ10 expand the mutational spectrum and suggest emerging genotype‐specific phenotypic trends. Systematic genetic and phenotypic profiling may guide more precise diagnosis and management of PKD.
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