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

2012; Wiley; Volume: 27; Issue: 10 Linguagem: Inglês

10.1002/mds.25126

1531-8257

Raffaella Zannolli, Sabrina Buoni, Gianni Betti, S Salvucci, Alessandro Plebani, Annarosa Soresina, Maria Cristina Pietrogrande, Silvana Martino, Vincenzo Leuzzi, Andrea Finocchi, Roberto Micheli, Livia N. Rossi, Alfredo Brusco, Filippo Misiani, A. Fois, Joussef Hayek, Colleen Kelly, Luciana Chessa,

Mitochondrial Function and Pathology

Abstract 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