ArticleNeurology2021
Perspective of Adults With Neurofibromatosis 1 and Cutaneous Neurofibromas: Implications for Clinical Trials.
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In one paragraph
Article in Neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
0numbers the graph read from it
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9citing papers in PubMed
0.9field-weighted citation impact, top 23% of its field
What it found
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The trial behind it
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Who cites it
9 citing papers in PubMed, 10 citations in OpenAlex.
- Effect of NFX-179 MEK inhibitor on cutaneous neurofibromas in persons with neurofibromatosis type 1.Science advances · 2024Trial
- Clinical Phenotypic Spectrum and Multisystem Burden in Neurofibromatosis Type 1: A Retrospective Regional Cohort Study.Biomedicines · 2026Article
- Quantitative measurement of cutaneous neurofibromas in neurofibromatosis type 1 using a structured-light scanner.Quantitative imaging in medicine and surgery · 2026Article
- Assessment and Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1: A Scoping Review.Neurology. Genetics · 2026Review
- Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1 With MEK Inhibitor Selumetinib: A Nonrandomized Clinical Trial.JAMA dermatology · 2025Article
- snRNA-seq of human cutaneous neurofibromas before and after selumetinib treatment implicates role of altered Schwann cell states, inter-cellular signaling, and extracellular matrix in treatment response.Acta neuropathologica communications · 2024Article
- Perspectives of adolescents with neurofibromatosis 1 and cutaneous neurofibromas: Implications for clinical trials.Clinical trials (London, England) · 2024Article
- Characterization of health concerns in people with neurofibromatosis type 1.Molecular genetics & genomic medicine · 2023Article
- Neurofibromatosis Clinical Trials-REiNS Collaboration 2020 Recommendations: Looking Back and Moving Ahead.Neurology · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors at 1 institution in 1 country.
Ashley CannonFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston. ashleycannon@uabmc.edu.
Dominique C PichardFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.
Pamela L WoltersFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.
Sarah AdsitFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.
Gregg EricksonFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.
Andrés J LessingFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.ORCID 0000-0001-8250-0145
Peng LiFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.ORCID 0000-0002-9026-9999
Whitney NarmoreFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.
Claas RöhlFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.
Tena RosserFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.
Brigitte C WidemannFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.
Jaishri O BlakeleyFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.
Scott R PlotkinFrom the Department of Genetics (A.C., W.N.) and School of Nursing (P.L.), University of Alabama at Birmingham; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases (D.C.P.), and Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (D.C.P., P.L.W., B.C.W.), NIH, Bethesda, MD; Children's Tumor Foundation (S.A.), New York, NY; Neurofibromatosis Network (G.E.), Wheaton, IL; Neurofibromatosis Northeast (A.J.L.), Burlington, MA; NF Kinder (C.R.), Vienna, Austria; Children's Hospital Los Angeles (T.R.); Keck School of Medicine (T.R.), University of Southern California, Los Angeles; Neurology (J.O.B.), Johns Hopkins School of Medicine, Baltimore, MD; and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston.ORCID 0000-0002-6109-6419
REiNS International Collaboration
University of Southern California · US
Funding
Therapies for patients with rare tumors and genetic tumor predispositionZIASC010354 · NCI · DIVISION OF CLINICAL SCIENCES - NCI · PI WIDEMANN, BRIGITTE · 2009 to 2025
$19.2MAbstract
objectiveTo assess the perspectives of adults with neurofibromatosis 1 (NF1) regarding cutaneous neurofibroma (cNF) morbidity, treatment options, and acceptable risk-benefit ratio to facilitate the design of patient-centered clinical trials.
methodsAn online survey developed by multidisciplinary experts and patient representatives of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) cNF Working Group was distributed to adults with NF1 (n = 3,734) in the largest international database of individuals with any form of NF. Eligibility criteria included self-reported NF1 diagnosis, age ≥18 years, ≥1 cNF, and ability to read English.
resultsA total of 548 adults with NF1 responded to the survey. Respondents ranked appearance, number, and then location as the most bothersome features of raised cNF. Seventy-five percent of respondents considered a partial decrease of 33%-66% in the number or size of cNF as a meaningful response to experimental treatments. Most respondents (48%-58%) were willing to try available cNF treatments but were not aware of options outside of surgical removal. Regarding experimental agents, respondents favored topical, then oral medications. Most individuals (>65%) reported being "very much" or "extremely willing" to try experimental treatments, especially those with the highest cNF burden. Many respondents were not willing to tolerate side effects like nausea/vomiting (51%) and rash (46%). The greatest barriers to participation in cNF clinical trials were cost of participation and need to take time off work.
conclusionsMost adults with NF1 are willing to consider experimental therapies for treatment of cNF. These data will guide the design of patient-centered clinical trials for adults with cNF.
Indexed as
Clinical Trials as TopicAdolescentAdultConnective Tissue DiseasesHumansNeurilemmomaNeurofibromaNeurofibromatosesNeurofibromatosis 1Skin NeoplasmsSurveys and Questionnaires
Identifiers
What OpenQuestion holds
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Read underepoch 390
Registered trials
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.