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.
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