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Patient-reported outcomes of pain and physical functioning in neurofibromatosis clinical trials

2016; Lippincott Williams & Wilkins; Volume: 87; Issue: 7_Supplement_1 Linguagem: Inglês

10.1212/wnl.0000000000002927

1526-632X

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

Nerve Injury and Rehabilitation

Objective: Tumors and other disease complications of neurofibromatosis (NF) can cause pain and negatively affect physical functioning. To document the clinical benefit of treatment in NF trials targeting these manifestations, patient-reported outcomes (PROs) assessing pain and physical functioning should be included as study endpoints. Currently, there is no consensus on the selection and use of such measures in the NF population. This article presents the recommendations of the PRO group of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration for assessing the domains of pain and physical functioning for NF clinical trials. Methods: The REiNS PRO group reviewed and rated existing PRO measures assessing pain intensity, pain interference, and physical functioning using their systematic method. Final recommendations are based primarily on 4 main criteria: patient characteristics, item content, psychometric properties, and feasibility for clinical trials. Results: The REiNS PRO group chose the Numeric Rating Scale–11 (≥8 years) to assess pain intensity, the Pain Interference Index (6–24 years) and the Patient-Reported Outcome Measurement Information System (PROMIS) Pain Interference Scale (≥18 years) to evaluate pain interference, and the PROMIS Physical Functioning Scale to measure upper extremity function and mobility (≥5 years) for NF clinical trials. Conclusions: The REiNS Collaboration currently recommends these PRO measures to assess the domains of pain and physical functioning for NF clinical trials; however, further research is needed to evaluate their use in individuals with NF. A final consensus recommendation for the pain interference measure will be disseminated in a future publication based on findings from additional published research.