Safety and efficacy of rivastigmine in children with Down syndrome: A double blind placebo controlled trial

Gail A. Spiridigliozzi, Sarah J. Hart, James H. Heller, Heather E. Schneider, Jane Ann Baker, Cathleen Weadon, George T. Capone, Priya S. Kishnani

Research output: Contribution to journalArticle

Abstract

Individuals with Down syndrome (DS) have decreased cholinergic function and an uneven profile of cognitive abilities, with more pronounced deficits in learning, memory, and expressive language. Cholinesterase inhibitors may improve cognitive function in adults and adolescents with DS, but studies in children with DS have been limited. This study aimed to: (i) investigate the safety and efficacy of rivastigmine treatment; (ii) build upon our open-label studies in children with DS in a double-blind, placebo-controlled clinical trial; and (iii) investigate specific cognitive domains that may respond to rivastigmine treatment. We conducted a 20-week double-blind, placebo-controlled trial to investigate the safety and efficacy of rivastigmine in 22 children and adolescents with DS aged 10-17 years. Safety measures included reports of adverse events, laboratory parameters, and electrocardiograms. Efficacy measures included parental assessments of adaptive behavior and executive function, and direct measures of language and memory. No group differences were found on safety measures and 22 of 24 participants that passed study screening completed the study. The results did not demonstrate evidence for significant improvement in aspects of cognition, language, or overall function in the children receiving rivastigmine. Our results suggest that rivastigmine is safe and well-tolerated for children and adolescents with DS, but may not be effective for improving performance on the selected measures in this study. However, larger samples and/or alternate measures could possibly reveal improvements in cognitive function with rivastigmine treatment. Further research is needed to define a battery of cognitive measures that is sensitive to treatment effects in DS.

Original languageEnglish (US)
JournalAmerican Journal of Medical Genetics, Part A
DOIs
StateAccepted/In press - 2016

Fingerprint

Down Syndrome
Placebos
Safety
Cholinesterase Inhibitors
Psychological Adaptation
Executive Function
Controlled Clinical Trials
Cognition
Cholinergic Agents
Electrocardiography
Learning

Keywords

  • Adaptive behavior
  • Cholinesterase inhibitors
  • Cognition
  • Down syndrome
  • Trisomy 21

ASJC Scopus subject areas

  • Genetics(clinical)
  • Genetics

Cite this

Spiridigliozzi, G. A., Hart, S. J., Heller, J. H., Schneider, H. E., Baker, J. A., Weadon, C., ... Kishnani, P. S. (2016). Safety and efficacy of rivastigmine in children with Down syndrome: A double blind placebo controlled trial. American Journal of Medical Genetics, Part A. DOI: 10.1002/ajmg.a.37650

Safety and efficacy of rivastigmine in children with Down syndrome : A double blind placebo controlled trial. / Spiridigliozzi, Gail A.; Hart, Sarah J.; Heller, James H.; Schneider, Heather E.; Baker, Jane Ann; Weadon, Cathleen; Capone, George T.; Kishnani, Priya S.

In: American Journal of Medical Genetics, Part A, 2016.

Research output: Contribution to journalArticle

Spiridigliozzi, Gail A.; Hart, Sarah J.; Heller, James H.; Schneider, Heather E.; Baker, Jane Ann; Weadon, Cathleen; Capone, George T.; Kishnani, Priya S. / Safety and efficacy of rivastigmine in children with Down syndrome : A double blind placebo controlled trial.

In: American Journal of Medical Genetics, Part A, 2016.

Research output: Contribution to journalArticle

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