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
A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia / R. Zannolli, S. Buoni, G. Betti, S. Salvucci, A. Plebani, A. Soresina, M.C. Pietrogrande, S. Martino, V. Leuzzi, A. Finocchi, R. Micheli, L.N. Rossi, A. Brusco, F. Misiani, A. Fois, J. Hayek, C. Kelly, L. Chessa. - In: MOVEMENT DISORDERS. - ISSN 0885-3185. - 27:10(2012 Sep 01), pp. 1312-1316.
Titolo: | A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia |
Autori: | |
Parole Chiave: | Administration, Oral ; Adolescent ; Anti-Inflammatory Agents ; Ataxia Telangiectasia ; Betamethasone ; Child ; Female ; Humans ; Male ; Treatment Outcome |
Settore Scientifico Disciplinare: | Settore MED/38 - Pediatria Generale e Specialistica Settore MED/39 - Neuropsichiatria Infantile |
Data di pubblicazione: | 1-set-2012 |
Rivista: | |
Tipologia: | Article (author) |
Digital Object Identifier (DOI): | http://dx.doi.org/10.1002/mds.25126 |
Appare nelle tipologie: | 01 - Articolo su periodico |