Taltirelin Hydrate in Patients with Ataxia Due to Spinocerebellar Degeneration.

Autor:	Cho JW; Department of Neurology, Samsung Seoul Hospital, Sungkyunkwan University, School of Medicine, Seoul, Korea., Lee JY; Department of Neurology, Seoul Metropolitan Government-Seoul National University Boramae Medical Center., Kim HJ; Department of Neurology, Seoul National University College of Medicine, Seoul, Korea., Kim JS; Department of Neurology, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea., Park KW; Department of Neurology, Korea University Anam Hospital, Korea University College of medicine, Seoul, Korea., Choi SM; Department of Neurology, Chonnam National University Hospital, Chonnam National University Medical School, Gwangju, Korea., Lyoo CH; Department of Neurology, Gangnam Severance Hospital, Yonsei University College of Medicine, Seoul, Korea., Koh SB; Department of Neurology, Korea University Guro Hospital, Korea University College of medicine, Seoul, Korea.
Jazyk:	angličtina
Zdroj:	Journal of movement disorders [J Mov Disord] 2024 Oct 21. Date of Electronic Publication: 2024 Oct 21.
DOI:	10.14802/jmd.24127
Abstrakt:	Objective: We conducted this study to assess the efficacy and safety of taltirelin hydrate (TH) in spinocerebellar degeneration (SCD). Methods: Patients were randomly assigned to either the taltirelin group (5 mg orally, twice daily) or the control group. The primary endpoint was changes in the Korean version of Scale for the Assessment and Rating of Ataxia (K-SARA) scores at 24 weeks. The secondary endpoints include changes in the K-SARA scores at 4 and 12 weeks, the Clinical Global Impression, Five-level version of the EuroQol five-dimensional questionnaire, Tinetti balance test and gait analysis at 4, 12 and 24 weeks. Results: A total of 149 patients (hereditary:non-hereditary = 86:63) were enrolled. There were significant differences in changes in K-SARA scores at 24 weeks from baseline between the taltirelin group and the control group (-0.51 ± 2.79 versus 0.36 ± 2.62, respectively; p = 0.0321). Of the K-SARA items, both 'Stance' and 'Speech disturbance' had significantly lower subscores in the taltirelin group as compared with the control group (-0.04 ± 0.89 versus 0.23 ± 0.79 and -0.07 ± 0.74 versus 0.18 ± 0.67; p = 0.0270 and 0.0130, respectively). But there were no significant differences in changes in other secondary efficacy outcome measures at 24 weeks from baseline between the two treatment arms (p > 0.05). Conclusion: Clinicians might consider using TH in the treatment of ataxia due to SCD.
Databáze:	MEDLINE
Externí odkaz:	Zobrazit plný text záznamu