Behavioral and cognitive outcomes for clinical trials in children with neurofibromatosis type 1.

ABSTRACT:

Objective

To evaluate the appropriateness of cognitive and behavioral outcome measures in clinical trials in neurofibromatosis type 1 (NF1) by analyzing the degree of deficits compared to reference groups, test-retest reliability, and how scores correlate between outcome measures.

Methods

Data were analyzed from the Simvastatin for cognitive deficits and behavioral problems in patients with neurofibromatosis type 1 (NF1-SIMCODA) trial, a randomized placebo-controlled trial of simvastatin for cognitive deficits and behavioral problems in children with NF1. Outcome measures were compared with age-specific reference groups to identify domains of dysfunction. Pearson r was computed for before and after measurements within the placebo group to assess test-retest reliability. Prin

SUBMITTER: van der Vaart T 

PROVIDER: S-EPMC4731686 | biostudies-literature | 2016 Jan

REPOSITORIES: biostudies-literature