Department of Clinical Genetics, Maastricht University Medical Center, Maastricht, The Netherlands.
Clin Genet. 2013 Sep;84(3):223-9. doi: 10.1111/cge.12063. Epub 2012 Dec 7.
Little is known about the aging process of people with specific syndromes, like Rett syndrome (RTT). Recognition of the clinical and behavioral characteristics of the adult RTT is needed in order to improve future management of the RTT girl and counseling of parents. In association with the Dutch RTT parent association, a 5-year longitudinal study was carried out. The study population consisted of 53 adult women with a clinical diagnosis of RTT. Postal questionnaires were sent, including demographic features, skills, physical and psychiatric morbidity. At the time of the second measurement seven women had died. In 2012, 80% of the questionnaires (37/46) were returned. Mean age of the women was 31.4 years. Molecular confirmation was possible for 83% of the women for whom analyses were carried out. The adult RTT woman has a more or less stable condition. The general disorder profile is that of a slow on-going deterioration of gross motor functioning in contrast to a better preserved cognitive functioning, less autonomic and epileptic features and good general health. This is the first longitudinal cohort study about aging in RTT. Continuing longitudinal studies are needed to gain more insight into the aging process in RTT.
对于特定综合征患者(如雷特综合征)的衰老过程,人们知之甚少。为了改善 RTT 女孩的未来管理和家长咨询,需要识别成人 RTT 的临床和行为特征。我们与荷兰 RTT 家长协会合作,开展了一项为期 5 年的纵向研究。研究人群包括 53 名临床诊断为 RTT 的成年女性。我们通过邮寄问卷的方式收集了人口统计学特征、技能、身体和精神疾病发病率等信息。在第二次测量时,有 7 名女性已经去世。2012 年,我们收到了 80%的问卷(37/46)。这些女性的平均年龄为 31.4 岁。对于接受分析的女性,83%的人可以进行分子确认。成年 RTT 女性的状况基本稳定。一般疾病特征是粗大运动功能的缓慢持续恶化,而认知功能较好,自主和癫痫特征较少,整体健康状况良好。这是关于 RTT 衰老的第一项纵向队列研究。需要进行持续的纵向研究,以更深入地了解 RTT 的衰老过程。