胃饥饿素改善瑞特综合征患者的肌张力障碍和震颤：一项初步研究。
Ghrelin improves dystonia and tremor in patients with Rett syndrome: A pilot study.
作者信息
Yuge Kotaro, Hara Munetsugu, Okabe Rumiko, Nakamura Yuki, Okamura Hisayoshi, Nagamitsu Shinichiro, Yamashita Yushiro, Orimoto Kenji, Kojima Masayasu, Matsuishi Toyojiro
机构信息
Department of Pediatrics and Child Health, Kurume University School of Medicine, 67 Asahi-machi, Kurume, Fukuoka 830-0011, Japan.
Cognitive and Molecular Institute of Brain Diseases, Kurume University School of Medicine, 67 Asahi-machi, Kurume, Fukuoka 830-0011, Japan.
出版信息
J Neurol Sci. 2017 Jun 15;377:219-223. doi: 10.1016/j.jns.2017.04.022. Epub 2017 Apr 12.
BACKGROUND
Dystonia occurs in approximately 60% of patients with Rett syndrome (RTT) and severely impairs their quality of life. However, an effective standard therapy has not been established. In a previous study, ghrelin levels were significantly decreased in patients with RTT, in particular, among patients over 10years old. This prompted speculation that ghrelin may play an important role in RTT.
OBJECTIVES
Four patients, including two adults, with severe dystonia and tremor, were recruited.
METHODS
Ghrelin was intravenously administered at a dose of 3μg/kg, once-daily for 3days, followed by once every 3weeks. Objective evaluation was performed, including scoring for different clinical features (SDCF), the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) and the Visual Analog Scale (VAS).
RESULTS
The SDCF, BFMDRS, autonomic dysfunction and VAS scores were markedly improved in two patients with severe dystonia and head tremor.
CONCLUSION
Ghrelin may improve extrapyramidal symptoms in patients with RTT.
背景
肌张力障碍发生在约60%的雷特综合征（RTT）患者中，严重损害他们的生活质量。然而，尚未确立有效的标准治疗方法。在先前的一项研究中，RTT患者的胃饥饿素水平显著降低，尤其是在10岁以上的患者中。这引发了胃饥饿素可能在RTT中起重要作用的推测。
目的
招募了4例包括2名成年人在内的患有严重肌张力障碍和震颤的患者。
方法
以3μg/kg的剂量静脉注射胃饥饿素，每日1次，共3天，随后每3周1次。进行了客观评估，包括不同临床特征评分（SDCF）、伯克-法恩-马斯登肌张力障碍评定量表（BFMDRS）和视觉模拟量表（VAS）。
结果
2例患有严重肌张力障碍和头部震颤的患者的SDCF、BFMDRS、自主神经功能障碍和VAS评分显著改善。
结论
胃饥饿素可能改善RTT患者的锥体外系症状。
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