A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia

Title

A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia

Creator

Zannolli R; Buoni S; Betti G; Salvucci S; Plebani A; Soresina A; Pietrogrande M C; Martino S; Leuzzi V; Finocchi A; Micheli R; Rossi L N; Brusco A; Misiani F; Fois A; Hayek J; Kelly C; Chessa L

Identifier

Publisher

Movement Disorders

Date

2012

Subject

tone and motor problems; ataxia; ataxia telangectasia; pharmacologic intervention; betamethasone;

Description

No controlled studies exist regarding the pharmaceutical reduction of ataxia symptoms in ataxia telangiectasia (A-T). In a multicenter, double-blind, randomized, placebo-controlled crossover trial, oral betamethasone (BETA) and placebo were compared in terms of their reduction of ataxia symptoms as assessed with the International Cooperative Ataxia Rating Scale (ICARS). In this study of 13 A-T children, betamethasone reduced the ICARS total score by a median of 13 points in the intent-to-treat population and 16 points in the per-protocol population (ie, median percent decreases of ataxia symptoms of 28% and 31%, respectively). In conclusion, Oral betamethasone could be a promising therapy to relieve ataxia symptoms in A-T patients; however, long-term effectiveness and safety must be established. (Current Controlled Trials, number ISRCTN08774933). © 2012 Movement Disorder Society.

Rights

Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).

Citation

Zannolli R; Buoni S; Betti G; Salvucci S; Plebani A; Soresina A; Pietrogrande M C; Martino S; Leuzzi V; Finocchi A; Micheli R; Rossi L N; Brusco A; Misiani F; Fois A; Hayek J; Kelly C; Chessa L, “A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia,” Pediatric Palliative Care Library, accessed October 17, 2021, https://pedpalascnetlibrary.omeka.net/items/show/16853.

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