title: Safety and efficacy of rivastigmine in adolescents with Down syndrome: long-term follow-up.
abstract: Following the completion of a 20-week, open-label study of the safety and efficacy of liquid rivastigmine for adolescents with Down syndrome, 5 of the 10 adolescents in the clinical trial continued long-term rivastigmine therapy and 5 did not. After an average period of 38 months, all 10 subjects returned for a follow-up assessment to determine the safety and efficacy of long-term rivastigmine use. Rivastigmine was well tolerated and overall health appeared to be unaffected by long-term rivastigmine use. Performance change on cognitive and language measures administered at the termination of the open-label clinical trial was compared between the two groups. No between-group difference in median performance change across the long-term period was found, suggesting that the long-term use of rivastigmine does not improve cognitive and language performance. However, two subjects demonstrated remarkable improvement in adaptive function over the long-term period. Both subjects had received long-term rivastigmine therapy. The discussion addresses the challenge of assessing cognitive change in clinical trials using adolescents with Down syndrome as subjects and the use of group versus individual data to evaluate the relevance of medication effects.
authors.affiliation: Department of Surgery, Duke University Medical Center, Durham, North Carolina 27710, USA. hell001@mc.duke.edu
authors.forename: James H
authors.initials: JH
authors.lastname: Heller
authors.forename: Gail A
authors.initials: GA
authors.lastname: Spiridigliozzi
authors.forename: Blythe G
authors.initials: BG
authors.lastname: Crissman
authors.forename: Jane Anne
authors.initials: JA
authors.lastname: McKillop
authors.forename: Haru
authors.initials: H
authors.lastname: Yamamoto
authors.forename: Priya S
authors.initials: PS
authors.lastname: Kishnani
mesh_terms: D000293:Adolescent; D003071:Cognition / Q000187:drug effects*; D004314:Down Syndrome / Q000188:drug therapy*; D005500:Follow-Up Studies; D006801:Humans; D007806:Language Disorders / Q000188:drug therapy / Q000209:etiology; D007807:Language Tests; D008297:Male; D018696:Neuroprotective Agents / Q000009:adverse effects / Q000627:therapeutic use*; D048448:Phenylcarbamates / Q000009:adverse effects / Q000627:therapeutic use*; D000068836:Rivastigmine; D013997:Time Factors
chemical_list: D018696:Neuroprotective Agents; D048448:Phenylcarbamates; D000068836:Rivastigmine