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

Jennifer A Janusz; Bonita P Klein-Tasman; Jonathan M Payne; Pamela L Wolters; Heather L Thompson; Staci Martin; Peter de Blank; Nicole Ullrich; Allison Del Castillo; Maureen Hussey; Kristina K Hardy; Kristina Haebich; Tena Rosser; Mary Anne Toledo-Tamula; Karin S Walsh; REiNS International Collaboration

doi:10.1212/WNL.0000000000012422

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

Neurology. 2021 Aug 17;97(7 Suppl 1):S73-S80. doi: 10.1212/WNL.0000000000012422. Epub 2021 Jul 6.

Authors

Jennifer A Janusz¹, Bonita P Klein-Tasman², Jonathan M Payne², Pamela L Wolters², Heather L Thompson², Staci Martin², Peter de Blank², Nicole Ullrich², Allison Del Castillo², Maureen Hussey², Kristina K Hardy², Kristina Haebich², Tena Rosser², Mary Anne Toledo-Tamula², Karin S Walsh²; REiNS International Collaboration

Affiliations

¹ From 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.
² From 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.

Abstract

Objective: To review parent-report social skills measures to identify and recommend consensus outcomes for use in clinical trials of social deficit in children and adolescents (ages 6-18 years) with neurofibromatosis type 1 (NF1).

Methods: Searches were conducted via PubMed and ClinicalTrials.gov to identity social skills outcome measures with English language versions used in clinical trials in the past 5 years with populations with known social skills deficits, including attention-deficit/hyperactivity disorder and autism spectrum disorder (ASD). Measures were rated by the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) Neurocognitive Committee on patient characteristics, use in published studies, domains assessed, availability of standard scores, psychometric properties, and feasibility to determine their appropriateness for use in NF1 clinical trials.

Results: Two measures were ultimately recommended by the committee: the Social Responsiveness Scale-2 (SRS-2) and the Social Skills Improvement System-Rating Scale (SSIS-RS).

Conclusions: Each of the 2 measures assesses different aspects of social functioning. The SSIS-RS is appropriate for studies focused on broader social functioning; the SRS-2 is best for studies targeting problematic social behaviors associated with ASD. Researchers will need to consider the goals of their study when choosing a measure, and specific recommendations for their use are provided.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Aged
Attention Deficit Disorder with Hyperactivity / psychology
Attention Deficit Disorder with Hyperactivity / therapy
Autism Spectrum Disorder / psychology*
Autism Spectrum Disorder / therapy
Female
Humans
Language
Male
Neurilemmoma / psychology
Neurofibromatoses / complications
Neurofibromatoses / psychology
Neurofibromatosis 1 / complications
Neurofibromatosis 1 / psychology*
Neurofibromatosis 1 / therapy
Skin Neoplasms / psychology
Social Behavior*
Social Skills*

Supplementary concepts

Schwannomatosis

Grants and funding

P50 HD105351/HD/NICHD NIH HHS/United States