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, REiNS International Collaboration, Shivani Ahlawat, Srivandana Akshintala, Jeffrey Allen, Simone Ardern-Holmes, Robert Avery, Amedeo Azizi, Dusica Babovic-Vuksanovic, Annette Bakker, Andrea Baldwin, Fred Barker, Amanda Bergner, Chetan Bettegowda, Sucharita Bhaumik, Larissa Bilaniuk, Kim Bischoff, Jaishri Blakely, Diana Bradford, Miriam Bredella, Wenli Cai, John Carino, Avneesh Chabra, Patricia Ciavarelli, Wade Clapp, Stephen Connor, Albert Cornelius, Tambra Dahlheimer, Stephanie Davis, Peter de Blank, Vidya Dhote, Joni Doherty, Eva Dombi, William Dudley, Rachel Ershler, D Gareth Evans, Laura Fayad, Cristina Fernandez-Valle, Rosalie Ferner, Michael Fisher, Barbara Franklin, Giulia Fulci, Tracy Galloway, Kathy Gardner, Richard Gedrich, Marco Giovannini, Amy Goldstein, Anne Goodwin, Stephane Goutagny, David Gutmann, Theresa Hadlock, Chris Halpin, C Oliver Hanemann, Kristina Hardy, Gordon Harris, Desiree Headley, Gena Heidary, Jonathan Heller, Cynthia Hingtgen, Trent Hummel, Susan Huson, Michael Jacobs, Jennifer Janusz, Diego Jaramillo, Justin Jordan, Allen Julian, Michel Kalamarides, Matthias Karajannis, Bonnie Klein-Tasman, Pamela Knight, Bruce Korf, Shannon Langmead, Theresa LaVallee, Fawn Leigh, Donita Lightner, Carol Lin, Robert Listernick, Grant Liu, Mevo Marco, Carole Marcus, Gabriella Mariani, Staci Martin, Victor Mautner, Vanessa Merker, Michael Ferguson, Chris Moertel, Jill Morris, Katrina Morris, Kathryn North, Fabio Nunes, Roger Packer, Laura Papi, Allyson Parry, Neha Patel, Jonathan Payne, Karen Peluso, Sebastian Perreault, Scott Plotkin, Tina Poussaint, Nancy Ratner, Karlyne Reilly, Vincent Riccardi, Kent Robertson, Claas Rohl, Deborah Rukin Gold, La Rosa Salvatore, Laura Schaffner Gray, Elizabeth Schorry, Claire Semerjian, Monica Sheridan, Chie-Schin Shih, Carolyn Sidor, William Slattery 3rd, Miriam Smith, Kathy Sommer, Marigo Stathis, Matt Steensma, Anat Stemmer-Rachamimov, David Stevenson, Kari Struemph, Lara Sullivan, Mary Anne Tamula, Mary Thomas, Heather Thompson, James Tonsgard, Nicole Ullrich, Sharad Verma, David Viskochil, Ana-Maria Vranceanu, Ute Wahllander, Karin Walsh, Bradley Welling, Ralph Wenzel, Trish Whitcomb, Brigitte Widemann, Victoria Williams, David Wolf, Pamela Wolters, Kaleb Yohay, 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, REiNS International Collaboration, Shivani Ahlawat, Srivandana Akshintala, Jeffrey Allen, Simone Ardern-Holmes, Robert Avery, Amedeo Azizi, Dusica Babovic-Vuksanovic, Annette Bakker, Andrea Baldwin, Fred Barker, Amanda Bergner, Chetan Bettegowda, Sucharita Bhaumik, Larissa Bilaniuk, Kim Bischoff, Jaishri Blakely, Diana Bradford, Miriam Bredella, Wenli Cai, John Carino, Avneesh Chabra, Patricia Ciavarelli, Wade Clapp, Stephen Connor, Albert Cornelius, Tambra Dahlheimer, Stephanie Davis, Peter de Blank, Vidya Dhote, Joni Doherty, Eva Dombi, William Dudley, Rachel Ershler, D Gareth Evans, Laura Fayad, Cristina Fernandez-Valle, Rosalie Ferner, Michael Fisher, Barbara Franklin, Giulia Fulci, Tracy Galloway, Kathy Gardner, Richard Gedrich, Marco Giovannini, Amy Goldstein, Anne Goodwin, Stephane Goutagny, David Gutmann, Theresa Hadlock, Chris Halpin, C Oliver Hanemann, Kristina Hardy, Gordon Harris, Desiree Headley, Gena Heidary, Jonathan Heller, Cynthia Hingtgen, Trent Hummel, Susan Huson, Michael Jacobs, Jennifer Janusz, Diego Jaramillo, Justin Jordan, Allen Julian, Michel Kalamarides, Matthias Karajannis, Bonnie Klein-Tasman, Pamela Knight, Bruce Korf, Shannon Langmead, Theresa LaVallee, Fawn Leigh, Donita Lightner, Carol Lin, Robert Listernick, Grant Liu, Mevo Marco, Carole Marcus, Gabriella Mariani, Staci Martin, Victor Mautner, Vanessa Merker, Michael Ferguson, Chris Moertel, Jill Morris, Katrina Morris, Kathryn North, Fabio Nunes, Roger Packer, Laura Papi, Allyson Parry, Neha Patel, Jonathan Payne, Karen Peluso, Sebastian Perreault, Scott Plotkin, Tina Poussaint, Nancy Ratner, Karlyne Reilly, Vincent Riccardi, Kent Robertson, Claas Rohl, Deborah Rukin Gold, La Rosa Salvatore, Laura Schaffner Gray, Elizabeth Schorry, Claire Semerjian, Monica Sheridan, Chie-Schin Shih, Carolyn Sidor, William Slattery 3rd, Miriam Smith, Kathy Sommer, Marigo Stathis, Matt Steensma, Anat Stemmer-Rachamimov, David Stevenson, Kari Struemph, Lara Sullivan, Mary Anne Tamula, Mary Thomas, Heather Thompson, James Tonsgard, Nicole Ullrich, Sharad Verma, David Viskochil, Ana-Maria Vranceanu, Ute Wahllander, Karin Walsh, Bradley Welling, Ralph Wenzel, Trish Whitcomb, Brigitte Widemann, Victoria Williams, David Wolf, Pamela Wolters, Kaleb Yohay
Abstract
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.
© 2016 American Academy of Neurology.
Source: PubMed