title: A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia.
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.)
authors.affiliation: Child Neurology and Psychiatry Unit, Azienda Ospedaliera Universitaria Senese/University of Siena, Policlinico Le Scotte, Siena, Italy. zannolli@unisi.it
authors.forename: Raffaella
authors.initials: R
authors.lastname: Zannolli
authors.forename: Sabrina
authors.initials: S
authors.lastname: Buoni
authors.forename: Gianni
authors.initials: G
authors.lastname: Betti
authors.forename: Sara
authors.initials: S
authors.lastname: Salvucci
authors.forename: Alessandro
authors.initials: A
authors.lastname: Plebani
authors.forename: Annarosa
authors.initials: A
authors.lastname: Soresina
authors.forename: Maria C
authors.initials: MC
authors.lastname: Pietrogrande
authors.forename: Silvana
authors.initials: S
authors.lastname: Martino
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Leuzzi
authors.forename: Andrea
authors.initials: A
authors.lastname: Finocchi
authors.forename: Roberto
authors.initials: R
authors.lastname: Micheli
authors.forename: Livia Nicoletta
authors.initials: LN
authors.lastname: Rossi
authors.forename: Alfredo
authors.initials: A
authors.lastname: Brusco
authors.forename: Filippo
authors.initials: F
authors.lastname: Misiani
authors.forename: Alberto
authors.initials: A
authors.lastname: Fois
authors.forename: Joseph
authors.initials: J
authors.lastname: Hayek
authors.forename: Colleen
authors.initials: C
authors.lastname: Kelly
authors.forename: Luciana
authors.initials: L
authors.lastname: Chessa
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000893:Anti-Inflammatory Agents / Q000008:administration & dosage*; D001260:Ataxia Telangiectasia / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology*; D001623:Betamethasone / Q000008:administration & dosage*; D002648:Child; D005260:Female; D006801:Humans; D008297:Male; D016896:Treatment Outcome
chemical_list: D000893:Anti-Inflammatory Agents; D001623:Betamethasone