Office of the Clinical Director, Division of Intramural Research at the National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland.
Division of Intramural Research at the National Institute of Mental Health, National Institutes of Health, Bethesda, Maryland.
Am J Med Genet A. 2019 Feb;179(2):224-236. doi: 10.1002/ajmg.a.61003.
Smith-Magenis syndrome (SMS) is a contiguous gene syndrome linked to interstitial microdeletion, or mutation of RAI1, within chromosome 17p11.2. Key behavioral features of SMS include intellectual disability, sleep-disturbances, maladaptive, aggressive and self-injurious behaviors, hyperactivity, and sudden changes in mood. A distinguishing feature of this syndrome is an inverted pattern of melatonin characterized by elevated daytime and low nighttime melatonin levels. As the central circadian clock controls the 24-hr rhythm of melatonin, we hypothesized that the clock itself may contribute to the disrupted pattern of melatonin and sleep. In this report, 24-hr patterns of body temperature, a surrogate marker of clock-timing, and continuous wrist activity were collected to examine the links between body temperature, sleep behavior, and the circadian clock. In addition, age-dependent changes in sleep behavior were explored. Actigraphy-estimated sleep time for SMS was 1 hr less than expected across all ages studied. The timing of the 24-hr body temperature (Tb-24) rhythm was phase advanced, but not inverted. Compared to sibling (SIB) controls, the SMS group had less total night sleep, lower sleep efficiency, earlier sleep onset, earlier final awake times, increased waking after sleep onset (WASO), and increased daytime nap duration. The timing of wake onset varied with age, providing evidence of ongoing developmental sleep changes from childhood through adolescence. Clarification of the circadian and developmental factors that contribute to the disrupted and variable sleep patterns in this syndrome will be helpful in identifying more effective individualized treatments.
史密斯-马根尼斯综合征(SMS)是一种连续基因综合征,与染色体 17p11.2 内的 RAI1 基因的间质微缺失或突变有关。SMS 的关键行为特征包括智力障碍、睡眠障碍、适应不良、攻击性行为和自残行为、多动和情绪突然变化。该综合征的一个显著特征是褪黑素的倒置模式,表现为白天褪黑素水平升高,夜间褪黑素水平降低。由于中央生物钟控制着褪黑素的 24 小时节律,我们假设生物钟本身可能导致褪黑素和睡眠模式的紊乱。在本报告中,我们收集了 24 小时体温(生物钟计时的替代标志物)和连续手腕活动的模式,以研究体温、睡眠行为和生物钟之间的联系。此外,还探讨了睡眠行为随年龄的变化。在所有研究的年龄中,SMS 患者的活动记录仪估计睡眠时间比预期少 1 小时。24 小时体温(Tb-24)节律的时间提前,但没有倒置。与兄弟姐妹(SIB)对照组相比,SMS 组的总夜间睡眠时间更少,睡眠效率更低,入睡时间更早,最后一次清醒时间更早,睡眠后觉醒时间增加(WASO),白天小睡时间增加。清醒起始时间随年龄变化,为正在进行的儿童期到青春期睡眠发育变化提供了证据。阐明导致该综合征睡眠模式紊乱和多变的昼夜节律和发育因素,将有助于确定更有效的个体化治疗方法。