دورية أكاديمية
Perspective of Adults With Neurofibromatosis 1 and Cutaneous Neurofibromas: Implications for Clinical Trials.
العنوان: | Perspective of Adults With Neurofibromatosis 1 and Cutaneous Neurofibromas: Implications for Clinical Trials. |
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المؤلفون: | Cannon A; From 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., Pichard DC; From 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., Wolters PL; From 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., Adsit S; From 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., Erickson G; From 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., Lessing AJ; From 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., Li P; From 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., Narmore W; From 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., Röhl C; From 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., Rosser T; From 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., Widemann BC; From 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., Blakeley JO; From 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., Plotkin SR; From 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. |
مؤلفون مشاركون: | REiNS International Collaboration |
المصدر: | Neurology [Neurology] 2021 Aug 17; Vol. 97 (7 Suppl 1), pp. S15-S24. Date of Electronic Publication: 2021 Jul 06. |
نوع المنشور: | Journal Article; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't |
اللغة: | English |
بيانات الدورية: | Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0401060 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1526-632X (Electronic) Linking ISSN: 00283878 NLM ISO Abbreviation: Neurology Subsets: MEDLINE |
أسماء مطبوعة: | Publication: Hagerstown, MD : Lippincott Williams & Wilkins Original Publication: Minneapolis. |
مواضيع طبية MeSH: | Clinical Trials as Topic*, Neurofibroma/*therapy , Neurofibromatoses/*therapy , Neurofibromatosis 1/*metabolism, Adolescent ; Adult ; Connective Tissue Diseases/therapy ; Humans ; Neurilemmoma/therapy ; Skin Neoplasms/therapy ; Surveys and Questionnaires |
مستخلص: | Objective: To 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. Methods: An 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. Results: A 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. Conclusions: Most 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. (© 2021 American Academy of Neurology.) |
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SCR Disease Name: | Schwannomatosis |
تواريخ الأحداث: | Date Created: 20210707 Date Completed: 20210901 Latest Revision: 20220818 |
رمز التحديث: | 20240628 |
مُعرف محوري في PubMed: | PMC8594006 |
DOI: | 10.1212/WNL.0000000000012425 |
PMID: | 34230202 |
قاعدة البيانات: | MEDLINE |
تدمد: | 1526-632X |
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DOI: | 10.1212/WNL.0000000000012425 |