Long-Term Evaluation of Low-Dose Betamethasone for Ataxia Telangiectasia

Pediatr Neurol. 2019 Nov;100:60-66. doi: 10.1016/j.pediatrneurol.2019.05.006. Epub 2019 May 13.


Background: Ataxia telangiectasia is an autosomal recessive disorder characterized by cerebellar ataxia, telangiectases, immune defects, and a predisposition to malignancy. Quality of life is severely impaired by neurological symptoms. However, curative options for the neurological symptoms are limited. Recent studies have demonstrated short-term improvement in neurological symptoms with betamethasone therapy. However, the long-term and adverse effects of betamethasone are unclear. The aim of this study was to evaluate the long-term effects, benefits, and adverse effects of low-dose betamethasone in ataxia telangiectasia.

Methods: Six patients with ataxia telangiectasia received betamethasone at 0.02 mg/kg/day for two years. After cessation of betamethasone, the patients were observed for two additional years. Neurological assessments were performed, and adverse effects were monitored every three months throughout the four-year study period.

Results: Transient improvement of neurological symptom was observed in five of the six patients. However, after two years betamethasone treatment, only one of the six patients showed a slight improvement in the neurological score, one patient showed no change, and the neurological scores of the remaining four patients deteriorated. After the cessation of betamethasone treatment, neurological symptoms worsened in all patients. As an adverse effect of betamethasone, transient adrenal dysfunction was observed in all cases.

Conclusions: Although these findings are in agreement with previous studies suggesting that short-term betamethasone treatment transiently benefits patients with ataxia telangiectasia, the long-term benefits and risks should be carefully considered.

Keywords: Adverse effect; Ataxia telangiectasia; Betamethasone; Long-term effect.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adrenal Gland Diseases / chemically induced
  • Ataxia Telangiectasia / drug therapy*
  • Betamethasone / administration & dosage
  • Betamethasone / adverse effects
  • Betamethasone / pharmacology*
  • Child
  • Drug-Related Side Effects and Adverse Reactions*
  • Female
  • Glucocorticoids / administration & dosage
  • Glucocorticoids / adverse effects
  • Glucocorticoids / pharmacology*
  • Humans
  • Longitudinal Studies
  • Male
  • Outcome Assessment, Health Care*
  • Peripheral Nervous System Diseases / chemically induced


  • Glucocorticoids
  • Betamethasone