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Recommendations for Measurement of Attention Outcomes in Preschoolers With Neurofibromatosis
Bonita P Klein-Tasman1, Kristin Lee2, Heather L Thompson2
1From the Department of Psychology (B.P.K.-T., K.L., S.P.), University of Wisconsin-Milwaukee; Department of Communication Sciences and Disorders (H.L.T.), California State University, Sacramento; University of Colorado School of Medicine (J.J.), Aurora; Murdoch Children's Research Institute and Department of Pediatrics (J.M.P.), University of Melbourne, Australia; University of Cincinnati Medical Center (P.d.B.), OH; Children's National Hospital (T.K., A.d.C., K.S.W.), Gilbert NF Institute, Washington, DC; and Division of Oncology (K.M.J.), Children's Hospital of Philadelphia, PA. bklein@uwm.edu.
Insights
Children with neurofibromatosis type 1 (NF1) experience attention issues. This study recommends specific assessment tools for preschoolers with NF1 to enable early intervention and improve clinical trial outcomes.
Area of Science:
- Neuroscience
- Developmental Psychology
- Genetics
Background:
- Children with neurofibromatosis type 1 (NF1) have a higher risk of attention problems.
- Preschool-aged children represent a critical developmental stage for early intervention in NF1.
- Previous research primarily focused on school-aged children, necessitating tailored approaches for younger cohorts.
Purpose of the Study:
- To recommend validated assessment measures for attention in preschool-aged children with NF1.
- To facilitate the inclusion of preschoolers in clinical trials for NF1.
- To inform early intervention strategies for attention difficulties in young children with NF1.
Main Methods:
- Review of existing literature and recommendations from the REiNS Neurocognitive Subcommittee.
- Identification of observer-rated and performance-based measures suitable for preschoolers.
- Consideration of measures for use in clinical trials targeting both physical and psychological aspects of NF1.
Main Results:
- The Attention-Deficit/Hyperactivity Disorder Rating Scale-Preschool version is recommended as a primary outcome measure.
- Performance-based measures including NIH Toolbox tasks (Flanker, Dimensional Change Card Sort, List Sort Working Memory) and Digits Forward (DAS-2) are recommended as secondary outcomes.
- Methodological recommendations for including preschoolers in clinical trials research were provided.
Conclusions:
- Standardized assessment tools are crucial for evaluating attention in preschoolers with NF1.
- Early identification and intervention using recommended measures can potentially mitigate later attention difficulties.
- These recommendations support the development of more inclusive and effective clinical trials for NF1 in young children.
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 report, 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 Attention-Deficit/Hyperactivity Disorder 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-2nd Edition (performance-based measures) are recommended as secondary outcome measures. Specific methodologic recommendations for inclusion of preschoolers in clinical trials research are also offered.
