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

Raffaella Zannolli, Buoni Sabrina Buoni, Gianni Betti, Sara Salvucci, Alessandro Plebani, Annarosa Soresina, Maria C. Pietrogrande, Silvana Martino, Vincenzo Leuzzi, Andrea Finocchi, Roberto Micheli, Livia Nicoletta Rossi, Alfredo Brusco, Filippo Misiani, Alberto Fois, Joseph Hayek, Colleen Kelly, Luciana Chessa

Research output: Contribution to journalArticlepeer-review

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).

Original languageEnglish
Pages (from-to)1312-1316
Number of pages5
JournalMovement Disorders
Volume27
Issue number10
DOIs
Publication statusPublished - Sep 1 2012

Keywords

  • Cerebellar ataxia
  • Cerebellar degeneration
  • Clinical neurology
  • Movement disorders
  • Neuropharmacology

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology

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