ArticleNeurology2021
Current Recommendations for Patient-Reported Outcome Measures Assessing Domains of Quality of Life in Neurofibromatosis Clinical Trials.
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Article in Neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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0cells of the map it votes in
22citing papers in PubMed
1.7field-weighted citation impact, top 13% of its field
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
22 citing papers in PubMed, 25 citations in OpenAlex.
- ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2025Trial
- Imaging as an early biomarker to predict sensitivity to everolimus for progressive NF2-related vestibular schwannoma.Journal of neuro-oncology · 2024Trial
- Effect of Mind-Body Skills Training on Quality of Life for Geographically Diverse Adults With Neurofibromatosis: A Fully Remote Randomized Clinical Trial.JAMA network open · 2023Trial
- Development and Initial Validation of the Quality of life Evaluation inmedRxiv : the preprint server for health sciences · 2026Article
- Determinants of the quality of life of patients with NF2-related schwannomatosis and validation of the Dutch NFTI-QOL questionnaire.Familial cancer · 2026Article
- Neurofibromatosis type 1-plexiform neurofibromas: Integrating treatment across pediatric and adult populations.Neuro-oncology · 2026Review
- Article
- Beyond the optic disc: Investigating gender-based differences in optic neuritis.World journal of clinical cases · 2025Review
- Psychosocial functioning and determinants of the health-related quality of life in children with neurofibromatosis type 1 and cognitive impairments.Journal of neuro-oncology · 2025Article
- How does a preference-based generic health-related quality of life measure perform in patients with a rare disease? Measurement properties of the EQ-5D-Y proxy version among underage patients with neurofibromatosis type 1.Orphanet journal of rare diseases · 2025Article
- Quality of life assessment in schwannomatosis - A systematic review.Brain & spine · 2025Article
- The impact of cutaneous neurofibromas on quality of life and mental health in neurofibromatosis type 1.The Journal of dermatology · 2024Article
- Quality of life in individuals with neurofibromatosis type 1 associated cutaneous neurofibromas: validation of the Dutch cNF-Skindex.Journal of patient-reported outcomes · 2024Article
- Minimal clinically important difference in the World Health Organization Quality of Life Brief (WHOQOL-BREF) for adults with neurofibromatosis.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2024Article
- The PlexiQoL, a patient-reported outcome measure on quality of life in neurofibromatosis type 1-associated plexiform neurofibroma: translation, cultural adaptation and validation into the Dutch language for the Netherlands.Journal of patient-reported outcomes · 2024Article
- Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in neurofibromatosis 1 clinical trials.Clinical trials (London, England) · 2024Article
- Long-term distress throughout one's life: health-related quality of life, economic and caregiver burden of patients with neurofibromatosis type 1 in China.Frontiers in public health · 2024Article
- Ten-Year Follow-up of Internal Neurofibroma Growth Behavior in Adult Patients With Neurofibromatosis Type 1 Using Whole-Body MRI.Neurology · 2023Article
- Early feasibility testing of a web-based mind-body resiliency program for adults with neurofibromatosis: The NF-Web study.PEC innovation · 2022Article
- A cross-sectional study of gender differences in quality of life domains in patients with neurofibromatosis type 1.Orphanet journal of rare diseases · 2022Article
Corrections and comments
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Authors and funding
17 authors at 1 institution in 3 countries.
Pamela L WoltersFrom 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.ORCID 0000-0001-9319-6281
Ana-Maria VranceanuFrom 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.).
Heather L ThompsonFrom 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.).
Staci MartinFrom 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.).
Vanessa L MerkerFrom 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.).ORCID 0000-0002-4542-5227
Andrea BaldwinFrom 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.).
Carolina BarnettFrom 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.).ORCID 0000-0001-5546-0221
Kimberley S KoetsierFrom 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.).ORCID 0000-0002-2918-4750
Cynthia M HingtgenFrom 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.).
Christopher J FunesFrom 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.).
James H TonsgardFrom 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.).
Elizabeth K SchorryFrom 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.).
Taryn AllenFrom 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.).
Taylor SmithFrom 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.).
Barbara FranklinFrom 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.).
Stephanie ReeveFrom 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.).
REiNS International Collaboration
Cincinnati Children's Hospital Medical Center · US
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveTo 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.
methodsThe 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.
resultsThe 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.
conclusionsThe 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.
Indexed as
Quality of LifeSelf ReportAdultChildHumansMaleNeurilemmomaNeurofibromatosesPatient Reported Outcome MeasuresPsychometricsSkin Neoplasms
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