Recommendations for Measurement of Attention Outcomes in Preschoolers With Neurofibromatosis

Abstract

Children with neurofibromatosis type 1 (NF1) are at increased risk for attention problems. While most research has been conducted with school-aged cohorts, preschool-aged children offer a novel developmental window for clinical studies, with the promise that treatments implemented earlier in the developmental trajectory may most effectively modify risk for later difficulties. Designing research studies around the youngest children with NF1 can result in intervention earlier in the developmental cascade associated with NF1 gene abnormalities. Furthermore, clinical trials for medications targeting physical and psychological aspects of NF1 often include individuals spanning a wide age range, including preschool-aged children. In a prior paper, the REiNS Neurocognitive Subcommittee made recommendations regarding performance-based and observer-rated measures of attention for use in clinical trials and highlighted the need for separate consideration of assessment methods for young children. The observer-rated ADHD Rating Scale – Preschool version is recommended as a primary outcome measure. The NIH Toolbox Flanker, Dimensional Change Card Sort, and List Sort Working Memory tasks and Digits Forward from the Differential Ability Scales – Second Edition (performance-based measures) are recommended as secondary outcome measures. Specific methodological recommendations for inclusion of preschoolers in clinical trials research are also offered.

Neurofibromatosis Clinical Trials—REiNS Collaboration 2020 Recommendations

“…8 This supplement also includes assessment and recommendations for the use of patient-reported outcome measures for other NF-related conditions such as disease-specific quality of life measures 9 and measures of hearing-related quality of life for patients with NF2. 10 In addition, validation of objective assessments of strength (handheld dynamometry), 11 attention, 12 and social skills 13 in patients with NF will provide key guidance across a broad spectrum of NF-related clinical trials. The REiNS biomarker working group presents an overview of current NF genotype-phenotype correlations and how these might be used in future clinical trials.…”