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青少年型亨廷顿舞蹈症患者无早期发育迟缓迹象。

No Evidence of Early Developmental Delay in Juvenile-Onset Huntington's Disease Patients.

作者信息

Olson Lucy, Dickens Sarah, Schultz Jordan L, Neema Mohit, Nopoulos Peggy C

机构信息

Department of Psychiatry, Carver College of Medicine at the University of Iowa, Iowa City, Iowa, USA.

Stead Family Children's Hospital at the University of Iowa, Iowa City, Iowa, USA.

出版信息

Mov Disord Clin Pract. 2025 Mar;12(3):346-352. doi: 10.1002/mdc3.14287. Epub 2024 Dec 7.

Abstract

BACKGROUND

Previous studies suggest that early developmental delay is a common feature of Juvenile-Onset Huntington's disease (JOHD), with highest incidence in those with very high CAG repeats (> 80). However, all reports of developmental delay in JOHD are exclusively based on retrospective review of medical charts. Comprehensive assessment of birth history metrics may provide better insight into the question of early life development in JOHD.

OBJECTIVE

To explore the prevalence of prematurity, birth complications, low birth weight and developmental delay in patients with JOHD in comparison to control participants.

METHODS

Parents of patients with JOHD and gene-non-expanded (GNE) control participants from Kids-HD (n = 104) and Kids-JOHD (n = 34, 24% with CAG > 80) studies completed a comprehensive birth history questionnaire. Answers focused on prematurity, birth complications, and birth weight, and along with reports of early developmental milestones, were compared between groups.

RESULTS

There were no statistically significant differences in prematurity, birth weights, birth complications, or motor and verbal developmental milestones between JOHD patients and GNE controls (all P values > 0.1). Furthermore, stratifying JOHD patients by CAG expansion (low vs. high) also showed no significant differences (GNE vs. low or GNE vs. high).

CONCLUSIONS

These findings support the notion that JOHD does not manifest as developmental delay before motor symptom onset and highlight a new framework to understand the course and nature of the disease.

摘要

背景

先前的研究表明,早期发育迟缓是青少年型亨廷顿舞蹈病(JOHD)的一个常见特征,在CAG重复序列非常高(> 80)的患者中发病率最高。然而,所有关于JOHD发育迟缓的报告均完全基于对病历的回顾性审查。对出生史指标进行全面评估可能有助于更好地了解JOHD患者的早期发育问题。

目的

与对照参与者相比,探讨JOHD患者中早产、出生并发症、低出生体重和发育迟缓的患病率。

方法

来自儿童亨廷顿舞蹈病研究(Kids-HD,n = 104)和儿童青少年型亨廷顿舞蹈病研究(Kids-JOHD,n = 34,24%的患者CAG> 80)的JOHD患者的父母和基因未扩增(GNE)对照参与者完成了一份全面的出生史问卷。问卷答案聚焦于早产、出生并发症和出生体重,并结合早期发育里程碑的报告,在两组之间进行比较。

结果

JOHD患者与GNE对照在早产、出生体重、出生并发症或运动及语言发育里程碑方面无统计学显著差异(所有P值> 0.1)。此外,按CAG扩增情况(低 vs. 高)对JOHD患者进行分层,也未显示出显著差异(GNE vs. 低或GNE vs. 高)。

结论

这些发现支持了JOHD在运动症状出现前不会表现为发育迟缓的观点,并突出了一个理解该疾病病程和本质的新框架。

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