Ghrelin improves dystonia and tremor in patients with Rett syndrome: A pilot study.

ABSTRACT

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.