{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,8]],"date-time":"2026-10-08T20:06:52Z","timestamp":1791490012643,"version":"4.3.4"},"reference-count":39,"publisher":"Oxford University Press (OUP)","issue":"1","license":[{"start":{"date-parts":[[2026,2,16]],"date-time":"2026-02-16T00:00:00Z","timestamp":1771200000000},"content-version":"vor","delay-in-days":46,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"AstraZeneca and Merck Sharp & Dohme LLC"},{"name":"Merck & Co., Inc., Rahway, NJ, USA"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,1,12]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Adults with neurofibromatosis type 1 and plexiform neurofibroma (NF1-PN) experience multiple clinical symptoms and diverse manifestations, which place a burden on patients and impact quality of life. This qualitative study aimed to improve understanding of disease burden, healthcare experience, and unmet needs of adults with NF1-PN in the United States, from patient and caregiver perspectives.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Telephone interviews were conducted with adults who had a diagnosis of NF1-PN, or with caregivers, focusing on patient background, diagnosis, interactions with healthcare professionals, disease and symptom management, transition of care, and unmet needs.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The study included 11 adult patients with NF1-PN and 2 caregivers; 85% of patients were diagnosed in childhood. Patients lived with multiple conditions associated with NF1, including pain disorders, psychiatric disorders, and chronic migraines. NF1-PN impacted daily living, work, school, relationships, and mental and emotional health. Most patients (82%) transitioned from pediatric to adult care, although there was variability in the transition experience. Some dropped out of care (23%) due to various factors, including time constraints, physician location, financial insecurity, lack of insurance, and perception of no available treatments\/cure. Medical management primarily comprised medications to relieve symptoms associated with NF1 manifestations. Respondents identified a need to be more informed about their care. Improved treatment options for NF1-PN are desired, particularly medications that stop or slow PN growth.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>The study demonstrated that NF1-PN has a profound impact on adult patients\u2019 lives. Several unmet needs exist for the adult population, including medications to treat the PN and its associated symptoms.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1093\/noajnl\/vdag033","type":"journal-article","created":{"date-parts":[[2026,2,10]],"date-time":"2026-02-10T12:31:38Z","timestamp":1770726698000},"source":"Crossref","is-referenced-by-count":1,"title":["Adult patient and caregiver perspectives on the impact of NF1-PN: Insights from a US qualitative survey"],"prefix":"10.1093","volume":"8","author":[{"given":"Phioanh L","family":"Nghiemphu","sequence":"first","affiliation":[{"name":"Department of Neurology, David Geffen School of Medicine, University of California , Los Angeles (P.L.N.)"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Conrad L","family":"Cordova","sequence":"additional","affiliation":[{"name":"Patient author 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