Neurocognitive outcomes in neurofibromatosis clinical trials: Recommendations for the domain of attention

Karin S Walsh; Jennifer Janusz; Pamela L Wolters; Staci Martin; Bonita P Klein-Tasman; Mary Anne Toledo-Tamula; Heather L Thompson; Jonathan M Payne; Kristina K Hardy; Peter de Blank; Claire Semerjian; Laura Schaffner Gray; Sondra E Solomon; Nicole Ullrich

doi:10.1212/WNL.0000000000002928

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Neurocognitive outcomes in neurofibromatosis clinical trials: Recommendations for the domain of attention

Journal article

Open access

Peer reviewed

Neurocognitive outcomes in neurofibromatosis clinical trials: Recommendations for the domain of attention

Karin S Walsh, Jennifer Janusz, Pamela L Wolters, Staci Martin, Bonita P Klein-Tasman, Mary Anne Toledo-Tamula, Heather L Thompson, Jonathan M Payne, Kristina K Hardy, Peter de Blank, …

Neurology, Vol.87(7 Suppl 1), pp.S21-S30

08/16/2016

DOI: https://doi.org/10.1212/WNL.0000000000002928

Handle:

https://hdl.handle.net/20.500.12741/rep:4451

PMCID: PMC5578356

PMID: 27527646

Abstract

Neuropsychological Tests

Humans

Attention

Treatment Outcome

Clinical Trials as Topic - methods

Neurofibromatosis 1 - therapy

Neurofibromatosis 1 - psychology

Neurofibromatosis type 1 (NF1) is associated with neurocognitive deficits that can impact everyday functioning of children, adolescents, and adults with this disease. However, there is little agreement regarding measures to use as cognitive endpoints in clinical trials. This article describes the work of the Neurocognitive Committee of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration. The goal of this committee is to identify standardized and specific cognitive assessment tools for use in NF clinical trials. The committee first identified cognitive domains relevant to NF1 and prioritized attention as the first domain of focus given prior and current trends in NF1 cognitive clinical trials. Performance measures and behavioral rating questionnaires of attention were reviewed by the group using established criteria to assess patient characteristics, psychometric properties, and feasibility. The highest rated tests underwent side-by-side comparison. The Digit Span subtest from the Wechsler scales was given the highest ratings of the performance measures due to its good psychometrics, feasibility, utility across a wide age range, and extensive use in previous research. The Conners scales achieved the highest ratings of the behavioral questionnaires for similar reasons. Future articles will focus on other cognitive domains, with the ultimate goal of achieving agreement for cognitive endpoints that can be used across NF clinical trials.

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Title: Neurocognitive outcomes in neurofibromatosis clinical trials: Recommendations for the domain of attention
Creators: Karin S Walsh - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington. kwalsh@childrensnational.org
Jennifer Janusz - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Pamela L Wolters - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Staci Martin - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Bonita P Klein-Tasman - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Mary Anne Toledo-Tamula - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Heather L Thompson - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Jonathan M Payne - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Kristina K Hardy - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Peter de Blank - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Claire Semerjian - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Laura Schaffner Gray - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Sondra E Solomon - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Nicole Ullrich - From the Children's National Health System (K.S.W., K.K.H.), The George Washington School of Medicine, Washington, DC; Children's Hospital Colorado (J.J.), University of Colorado School of Medicine, Aurora; Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, Bethesda, MD; University of Wisconsin-Milwaukee (B.P.K.-T.); Clinical Research Directorate/Clinical Monitoring Research Program (M.A.T.-T.), Leidos Biomedical Research, Inc., NCI at Frederick, MD; California State University (H.L.T.), Sacramento; Murdoch Children's Research Institute (J.M.P.), Victoria, Australia; Rainbow Babies & Children's Hospital (P.d.B.), Cleveland, OH; Roosevelt University (C.S.), Chicago, IL; Boston Children's Hospital (L.S.G., N.U.), MA; and University of Vermont (S.E.S.), Burlington
Academic Unit: Comm Sciences & Disorders
Publisher: United States
Publication Details: 08/16/2016
Grant note: U54 HD090255 / NICHD NIH HHS
Identifiers: 99257847089201671; https://hdl.handle.net/20.500.12741/rep:4451; https://doi.org/10.1212/WNL.0000000000002928
Language: English