TY - JOUR
T1 - Neurocognitive outcomes in neurofibromatosis clinical trials
T2 - Recommendations for the domain of attention
AU - Walsh, Karin S
AU - Janusz, Jennifer
AU - Wolters, Pamela L
AU - Martin, Staci
AU - Klein-Tasman, Bonita P
AU - Toledo-Tamula, Mary Anne
AU - Thompson, Heather L
AU - Payne, Jonathan M
AU - Hardy, Kristina K
AU - de Blank, Peter
AU - Semerjian, Claire
AU - Gray, Laura Schaffner
AU - Solomon, Sondra E
AU - Ullrich, Nicole
AU - REiNS International Collaboration
N1 - © 2016 American Academy of Neurology.
PY - 2016/8/16
Y1 - 2016/8/16
N2 - 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.
AB - 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.
U2 - 10.1212/WNL.0000000000002928
DO - 10.1212/WNL.0000000000002928
M3 - Article
C2 - 27527646
SN - 0028-3878
VL - 87
SP - S21-30
JO - Neurology
JF - Neurology
IS - 7 Suppl 1
ER -