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Long-term evaluation of low-dose betamethasone for ataxia telangiectasia

Pediatric Neurology Jul 05, 2019

Hasegawa S, et al. - In this investigation, researchers assessed the long-term impacts, advantages, and adverse effects of low-dose betamethasone in ataxia telangiectasia (an autosomal recessive disorder with cerebellar ataxia). Betamethasone was taken for 2 years by six patients with ataxia telangiectasia at 0.02 mg/kg/day. In five of the six patients, transient improvement of neurological symptoms was noted. However, only one of the six patients showed a slight improvement in the neurological score after two years of treatment with betamethasone, one patient exhibited no change, and the neurological scores of the remaining four patients worsened. Neurological symptoms in all patients worsened after the cessation of treatment with betamethasone. In all cases, transient adrenal dysfunction was noted as an adverse effect of betamethasone. Results of this study suggested that short-term betamethasone treatment transiently helps ataxia telangiectasia patients, but the long-term advantages and risks should be carefully considered.

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