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.

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

PLEBANI, Alessandro;Martino, Silvana;LEUZZI, Vincenzo;Brusco, Alfredo;
2012-01-01

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.
2012
27
10
1312
1316
http://onlinelibrary.wiley.com/doi/10.1002/mds.25126/abstract
Cerebellar ataxia; Cerebellar degeneration; Clinical neurology; Movement disorders; Neuropharmacology.
Zannolli, Raffaella; Buoni, Sabrina; Betti, Gianni; Salvucci, Sara; Plebani, Alessandro; Soresina, Annarosa; Pietrogrande, Maria C; Martino, Silvana; Leuzzi, Vincenzo; Finocchi, Andrea; Micheli, Roberto; Rossi, Livia Nicoletta; Brusco, Alfredo; Misiani, Filippo; Fois, Alberto; Hayek, Joseph; Kelly, Colleen; Chessa, Luciana
File in questo prodotto:
File Dimensione Formato  
76. A Randomized Trial of Oral betametasone_mov dis 2012.pdf

Accesso riservato

Tipo di file: PDF EDITORIALE
Dimensione 344.29 kB
Formato Adobe PDF
344.29 kB Adobe PDF   Visualizza/Apri   Richiedi una copia

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2318/118841
Citazioni
  • ???jsp.display-item.citation.pmc??? 22
  • Scopus 67
  • ???jsp.display-item.citation.isi??? 63
social impact