A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia
@article{Zannolli2012ART, title={A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia}, author={Raffaella Zannolli and Sabrina Buoni and Gianni Betti and Sara Salvucci and Alessandro Plebani and Annarosa Soresina and Maria Cristina Pietrogrande and S. Martino and Vincenzo Leuzzi and Andrea Finocchi and Roberto Micheli and Livia N. Rossi and Alfredo Brusco and Filippo Misiani and Alberto Fois and Joseph Hayek and Colleen Kelly and Luciana Chessa}, journal={Movement Disorders}, year={2012}, volume={27} }
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…
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Title : Minimum effective betamethasone dosage on the neurological phenotype in patients with Ataxia-Telangiectasia : a multicenter observer-blind study Running Title : Betamethasone treatment in Ataxia-Telangiectasia
- Medicine, Psychology
- 2018
These data suggest that a short-term betamethasone oral treatment, at a daily dosage of 0.005 mg/kg/day, is effective in some patients, and pre-existing risk factors for sideeffects should be taken into account before therapy.
Minimum effective betamethasone dosage on the neurological phenotype in patients with ataxia‐telangiectasia: a multicenter observer‐blind study
- Medicine, BiologyEuropean journal of neurology
- 2018
The aim of this multicenter study was to define the minimal effective dosage of oral betamethasone, thus preventing the occurrence of side effects, in patients with ataxia‐telangiectasia.
Long-Term Evaluation of Low-Dose Betamethasone for Ataxia Telangiectasia.
- Medicine, PsychologyPediatric neurology
- 2019
Positive effect of erythrocyte-delivered dexamethasone in ataxia-telangiectasia
- MedicineNeurology: Neuroimmunology & Neuroinflammation
- 2015
Patients in the extended study experienced a continuous neurologic improvement with respect to their pretreatment status, whereas controls showed a progressive neurologic deterioration (according to the natural history of the disease) after the discontinuation of the treatment.
Betamethasone therapy in ataxia telangiectasia: unraveling the rationale of this serendipitous observation on the basis of the pathogenesis
- Biology, MedicineEuropean journal of neurology
- 2013
A better understanding of the mechanisms of action of GCs in the brain is needed, because in A‐T during the initial phase of cell loss the neurological impairment may be rescued by interfering in the biochemical pathways.
Intra-Erythrocyte Infusion of Dexamethasone Reduces Neurological Symptoms in Ataxia Teleangiectasia Patients: Results of a Phase 2 Trial
- Medicine, PsychologyOrphanet Journal of Rare Diseases
- 2014
EryDex treatment led to a significant improvement in neurological symptoms, without association with the typical steroid side effects, and a more marked improvement was found in less neurologically impaired patients.
Placebo response in degenerative cerebellar ataxias: a descriptive review of randomized, placebo-controlled trials
- Psychology, MedicineJournal of Neurology
- 2020
The placebo response in patients with CAs on various objective ataxia scales is demonstrated and it is emphasized that the placebo response should be considered when designing, analyzing, and interpreting clinical trials and in clinical practice in CA patients.
Ibuprofen prevents progression of ataxia telangiectasia symptoms in ATM-deficient mice
- Biology, PsychologyJournal of Neuroinflammation
- 2018
While the behavioral symptoms could not be reversed once they were established in adult Atm−/− animals, administration of ibuprofen to young mutant pups prevented their symptoms from appearing, implying that modulation of the immune system can have therapeutic benefit for both the behavioral and cellular symptoms of this neurodegenerative disease.
Current and future therapeutic strategies to treat ataxia telangiectasia
- Medicine, Biology
- 2014
Efficacy of steroids in improving ataxia of AT patients is proven and encapsulation of the drug into erythrocytes followed by slow release appears promising, and extrapyramidal symptoms may be ameliorated by dopaminergic drugs.
Focusing New Ataxia Telangiectasia Therapeutic Approaches
- Biology, Medicine
- 2016
The classical and the new therapeutic approaches in ataxia Telangiectasia are discussed in the light of the most recent reports in the literature.
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