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

Pamela L Wolters; Staci Martin; Vanessa L Merker; James H Tonsgard; Sondra E Solomon; Andrea Baldwin; Amanda L Bergner; Karin Walsh; Heather L Thompson; Kathy L Gardner; Cynthia M Hingtgen; Elizabeth Schorry; William N Dudley; Barbara Franklin

doi:10.1212/WNL.0000000000002927

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

Journal article

Open access

Peer reviewed

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

Pamela L Wolters, Staci Martin, Vanessa L Merker, James H Tonsgard, Sondra E Solomon, Andrea Baldwin, Amanda L Bergner, Karin Walsh, Heather L Thompson, Kathy L Gardner, …

Neurology, Vol.87(7 Suppl 1), pp.S4-S12

08/16/2016

DOI: https://doi.org/10.1212/WNL.0000000000002927

Handle:

https://hdl.handle.net/20.500.12741/rep:5194

PMCID: PMC5578357

PMID: 27527648

Abstract

Disability Evaluation

Pain Measurement - methods

Pain - physiopathology

Humans

Self Report

Neurofibromatoses - physiopathology

Clinical Trials as Topic - methods

Neurofibromatoses - therapy

Patient Reported Outcome Measures

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. 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. 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. 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.

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Title: Patient-reported outcomes of pain and physical functioning in neurofibromatosis clinical trials
Creators: Pamela L Wolters - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA. woltersp@mail.nih.gov
Staci Martin - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
Vanessa L Merker - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
James H Tonsgard - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
Sondra E Solomon - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
Andrea Baldwin - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
Amanda L Bergner - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
Karin Walsh - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
Heather L Thompson - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
Kathy L Gardner - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
Cynthia M Hingtgen - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
Elizabeth Schorry - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
William N Dudley - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
Barbara Franklin - From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute, National Institutes of Health, Bethesda, MD; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital, Boston; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Department of Psychological Sciences (S.E.S.), University of Vermont, Burlington; Departments of Neurology and Genetics (A.L.B.), Johns Hopkins University, Baltimore, MD; Children's National Health System & The George Washington School of Medicine (K.W.), Washington, DC; Department of Speech Pathology & Audiology (H.L.T.), California State University, Sacramento; Veteran's Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.), PA; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; Division of Human Genetics (E.S.), Cincinnati Children's Hospital, OH; Department of Public Health Education (W.N.D.), School of Health and Human Sciences, University of North Carolina at Greensboro; and Advocure NF2 Inc. (B.F.), Los Angeles, CA
Academic Unit: Comm Sciences & Disorders
Publisher: United States
Publication Details: 08/16/2016
Grant note: U54 HD090255 / NICHD NIH HHS
Identifiers: 99257847427401671; https://hdl.handle.net/20.500.12741/rep:5194; https://doi.org/10.1212/WNL.0000000000002927
Language: English