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Current Recommendations for Patient-Reported Outcome Measures Assessing Domains of Quality of Life in
Pamela L Wolters1, Ana-Maria Vranceanu2, Heather L Thompson2
1From the Pediatric Oncology Branch (P.L.W., S.M.), National Cancer Institute, NIH, Bethesda, MD; Integrated Brain Health Clinical and Research Program (A.-M.V., C.J.F.), Department of Psychiatry, Massachusetts General Hospital, Harvard Medical School, Boston; Department of Communication Sciences and Disorders (H.L.T.), California State University, Sacramento; Center for Healthcare Organization and Implementation Research (V.L.M.), Edith Nourse Rogers Memorial Veterans Hospital, Bedford; Clinical Monitoring Research Program Directorate (A.B., T.A.), Frederick National Laboratory for Cancer Research, National Cancer Institute, Frederick, MD; Division of Neurology (C.B.), Department of Medicine, University Health Network and University of Toronto, Ontario, Canada; Department of Otolaryngology/Head and Neck Surgery (K.S.K.), Leiden University Medical Center, the Netherlands; Department of Clinical Neurosciences (C.M.H.), Spectrum Health Medical Group and College of Human Medicine, Michigan State University, East Lansing; University of Chicago Pritzker School of Medicine (J.H.T.), IL; Division of Human Genetics (E.K.S.), Cincinnati Children's Hospital, OH; Department of Psychology and Child Development (T.S.), California Polytechnic State University, San Luis Obispo; and REiNS International Collaboration Patient Representative (B.F., S.R.). woltersp@mail.nih.gov.
Objective:
To review and recommend patient-reported outcome (PRO) measures assessing multidimensional domains of quality of life (QoL) to use as clinical endpoints in medical and psychosocial trials for children and adults with neurofibromatosis (NF) type 1, NF2, and schwannomatosis.
Methods:
The PRO working group of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration used systematic methods to review, rate, and recommend existing self-report and parent-report PRO measures of generic and disease-specific QoL for NF clinical trials. Recommendations were based on 4 main criteria: patient characteristics, item content, psychometric properties, and feasibility.
Results:
The highest-rated generic measures were (1) the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scales for NF clinical trials for children or for children through adults, (2) the Functional Assessment of Cancer Therapy-General for adult medical trials, and (3) the World Health Organization Quality of Life-BREF for adult psychosocial trials. The highest-rated disease-specific measures were (1) the PedsQL NF1 Module for NF1 trials, (2) the NF2 Impact on Quality of Life Scale for NF2 trials, and (3) the Penn Acoustic Neuroma Quality of Life Scale for NF2 trials targeting vestibular schwannomas. To date, there are no disease-specific tools assessing multidimensional domains of QoL for schwannomatosis.
Conclusions:
The REiNS Collaboration currently recommends these generic and disease-specific PRO measures to assess multidimensional domains of QoL for NF clinical trials. Additional research is needed to further evaluate the use of these measures in both medical and psychosocial trials.
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