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Recommendations for Social Skills End Points for Clinical Trials in Neurofibromatosis Type 1

Jennifer A Janusz1, Bonita P Klein-Tasman2, Jonathan M Payne2

  • 1From the Children's Hospital Colorado and University of Colorado School of Medicine (J.A.J.), Aurora; Department of Psychology (B.P.K.-T.), University of Wisconsin-Milwaukee; Murdoch Children's Research Institute and Department of Pediatrics (J.M.P., K.H.), University of Melbourne, Australia; Pediatric Oncology Branch (P.L.W., S.M., M.A.T.-T.), National Cancer Institute, Bethesda, MD; Department of Communication Sciences and Disorders (H.L.T.), California State University, Sacramento; University of Cincinnati Medical Center (P.d.B.), OH; Boston Children's Hospital (N.U.), MA; Children's National Hospital, Gilbert NF Institute (A.d.C., K.K.H., K.S.W.), Washington, DC; Children's Tumor Foundation (M.H.), New York, NY; The George Washington School of Medicine (K.K.H., K.S.W.), Washington, DC; Children's Hospital Los Angeles (T.R.), CA; and Leidos Biomedical Research, Inc. (M.A.T.-T.), Frederick, MD. jennifer.janusz@childrenscolorado.org.

Neurology
|July 7, 2021
PubMed

Insights

This study recommends the Social Responsiveness Scale-2 (SRS-2) and the Social Skills Improvement System-Rating Scale (SSIS-RS) for assessing social deficits in children with neurofibromatosis type 1 (NF1). These tools aid in clinical trials for social skills development.

Area of Science:

  • Neuroscience
  • Pediatric Psychology
  • Clinical Trial Methodology

Background:

  • Social deficits are a significant concern in neurofibromatosis type 1 (NF1) affecting children and adolescents.
  • Standardized, reliable measures are crucial for evaluating interventions in clinical trials for NF1.
  • Existing social skills measures require evaluation for suitability in the NF1 population.

Purpose of the Study:

  • To review parent-report social skills measures for children and adolescents (ages 6-18) with NF1.
  • To identify and recommend consensus outcome measures for use in NF1 clinical trials.
  • To guide the selection of appropriate social functioning assessments.

Main Methods:

  • Conducted literature searches in PubMed and ClinicalTrials.gov for social skills measures.
  • Included measures used in clinical trials for populations with social deficits (ADHD, ASD) within the last 5 years.
  • Evaluated measures based on patient characteristics, study usage, domains, scores, psychometrics, and feasibility by the REiNS Neurocognitive Committee.

Main Results:

  • The Social Responsiveness Scale-2 (SRS-2) and the Social Skills Improvement System-Rating Scale (SSIS-RS) were recommended.
  • These two measures were identified as appropriate for clinical trials involving NF1.
  • Each measure captures different facets of social functioning.

Conclusions:

  • The SSIS-RS is suitable for studies on broader social functioning.
  • The SRS-2 is optimal for research targeting ASD-associated social behaviors.
  • Researchers should select measures based on specific study objectives for NF1 social deficit trials.
Abstract

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