Document Detail

Efficacy of very-low-dose betamethasone on neurological symptoms in ataxia-telangiectasia.
MedLine Citation:
PMID:  20840352     Owner:  NLM     Status:  In-Data-Review    
Background:  Ataxia-telangiectasia (A-T) is a non-curable neurodegenerative disorder, associated with progressive neurological dysfunction, oculocutaneous telangiectasia, immunodeficiency, predisposition to cancer and radiosensitivity. A recent study documented improvement in neurological symptoms after a short-term therapy with betamethasone in patients with A-T. Aim of this study was to evaluate the minimum therapeutically effective dosage of betamethasone on neurological symptoms of A-T. Methods:  Six responsive patients with A-T, received two 20-day cycles of oral betamethasone at 0.01 and 0.03 mg/kg/day (10% and 30% of the previously used full dosage), each followed by a 20-day washout period. Clinical and laboratory evaluations were carried out at T0 and at the end of each cycle. Neurological assessment was performed through the Scale for the Assessment and Rating of Ataxia (SARA). The glucocorticoid-induced leucine zipper (GILZ) and glucocorticoid receptor (GR) RNA expression were evaluated before and during the trial through real-time PCR. Results:  SARA scores significantly improved in all patients at the dosage of 0.03 mg/kg/day. In particular, three patients exhibited an improvement in 5/8 variables and two patients of 7 and 8 variables, respectively. Furthermore, the clinical improvement was already evident after the lower dosage. The basal GILZ and GR RNA expression were significantly lower in patients than in controls. GILZ expression increased in all patients after the beginning of the therapy, whereas no correlation between GR and the response was found. Conclusion:  Our data indicate that betamethasone is effective in A-T at a minimal dosage and that GILZ may be a useful biomarker of the clinical response. This study provides Class IIIA evidence that betamethasone at very low dosage is effective in improving neurological signs of patients affected with ataxia-telangiectasia.
T Broccoletti; E Del Giudice; E Cirillo; I Vigliano; G Giardino; V M Ginocchio; S Bruscoli; C Riccardi; C Pignata
Publication Detail:
Type:  Journal Article     Date:  2010-09-14
Journal Detail:
Title:  European journal of neurology : the official journal of the European Federation of Neurological Societies     Volume:  18     ISSN:  1468-1331     ISO Abbreviation:  Eur. J. Neurol.     Publication Date:  2011 Apr 
Date Detail:
Created Date:  2011-03-25     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  9506311     Medline TA:  Eur J Neurol     Country:  England    
Other Details:
Languages:  eng     Pagination:  564-70     Citation Subset:  IM    
Copyright Information:
© 2010 The Author(s). European Journal of Neurology © 2010 EFNS.
Department of Pediatrics, Federico II University, Naples Department of Clinical and Experimental Medicine, Section of Pharmacology, University of Perugia, Perugia, Italy.
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