Neurology 2021 08 6;97(7 Suppl 1):S15-S24. Epub 2021 Jul 6.
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.
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. Read More