title: Patient-reported outcomes in neurofibromatosis and schwannomatosis clinical trials.
abstract: OBJECTIVE
  Neurofibromatosis (NF) is a genetic disease with multiple clinical manifestations that can significantly impact quality of life (QOL). Clinical trials should include patient-reported outcomes (PROs) as endpoints to assess treatment effects on various aspects of QOL, but there is no consensus on the selection and use of such measures in NF. This article describes the PRO Working Group of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) Collaboration, its main goals, methods for identifying appropriate PRO measures for NF clinical trials, and recommendations for assessing pain intensity.
  
  
  METHODS
  The REiNS PRO group selected core endpoint domains important to assess in NF. The members developed criteria to rate PRO measures, including patient characteristics, psychometric properties, and feasibility, and utilized a systematic process to evaluate PROs for NF clinical trials. Within the subdomain of pain intensity, the group reviewed the Numerical Rating Scale-11 (NRS-11), the Visual Analogue Scale, and the Faces Pain Scale-Revised using this process.
  
  
  RESULTS
  Based on the review criteria, each of these pain intensity scales is brief, reliable, valid, and widely used. However, the NRS-11 was given the highest rating for use in NF clinical trials due to recommendations from pain experts and other consensus groups, its extensive use in research, strong psychometric data including sensitivity to change, and excellent feasibility in ages ≥ 8 years.
  
  
  CONCLUSIONS
  The systematic review criteria and process are effective for identifying appropriate PRO measures and provide information utilized by the REiNS Collaboration to achieve consensus regarding PROs in NF clinical trials.
authors.affiliation: From the Pediatric Oncology Branch (P.L.W., S.M., A.B.), National Cancer Institute; Department of Neurology and Cancer Center (V.L.M.), Massachusetts General Hospital; Veterans Administration Pittsburgh Healthcare System and University of Pittsburgh (K.L.G.); Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University; University of Chicago Pritzker School of Medicine (J.H.T.); and Division of Human Genetics (E.K.S.), Cincinnati Children's Hospital.
authors.forename: Pamela L
authors.initials: PL
authors.lastname: Wolters
authors.forename: Staci
authors.initials: S
authors.lastname: Martin
authors.forename: Vanessa L
authors.initials: VL
authors.lastname: Merker
authors.forename: Kathy L
authors.initials: KL
authors.lastname: Gardner
authors.forename: Cynthia M
authors.initials: CM
authors.lastname: Hingtgen
authors.forename: James H
authors.initials: JH
authors.lastname: Tonsgard
authors.forename: Elizabeth K
authors.initials: EK
authors.lastname: Schorry
authors.forename: Andrea
authors.initials: A
authors.lastname: Baldwin
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods / Q000592:standards*; D032921:Consensus; D006801:Humans; D009442:Neurilemmoma / Q000628:therapy; D017253:Neurofibromatoses / Q000628:therapy*; D010147:Pain Measurement / Q000379:methods / Q000592:standards*; D063868:Patient Outcome Assessment; D012878:Skin Neoplasms / Q000628:therapy