Betamethasone Trials
Reference:
Zannolli, R., Sabrina, B., Betti, G., Salvucci, S., Plebani, A., Soresina, A., Pietrogrande, M.C., Martino, S., Leuzzi, V., Finocchi, A., et al. (2012). A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia. Mov Disord 27, 1312-1316.
Investigator:
Claudio Pignata, MD
Location:
Italy
Purpose:
This small open label (non-placebo controlled) trial was performed to evaluate the minimum therapeutically effective dosage of bethamethasone, a corticosteroid, on the neurological symptoms of A-T.
Conclusions
Betamethasone at very low dosage (0.03 mg/kg/day) is effective at improving neurological signs in patients affected with ataxia-telangiectasia. However, as previously shown, a worsening of the neurological signs was noted after the washout periods. In the near future, it will be important to define whether a prolongation of the treatment will lead to the persistence of the neurological improvement. A long-term study with low dosage would also help define the benefit to risk ratio of such therapeutic intervention to address the concern of the immunosuppressive effect on infection susceptibility in these patients.
Reference:
Broccoletti, T., Del Giudice, E., Cirillo, E., Vigliano, I., Giardino, Gl, Ginocchio, V.M., Bruscoli, S., Riccardi, C., and Pignata, C. (2011). Efficacy of very-low-dose betamethasone on neurological symptoms of ataxia-telangiectasia. Eur Neurol 18, 564-570.