title: Phase II trial of pirfenidone in adults with neurofibromatosis type 1.
abstract: We performed an open-label phase II trial of oral pirfenidone in 24 patients with neurofibromatosis type 1 (NF1). Tumors were monitored by three-dimensional MRI. At the end of treatment, four patients had a decrease in tumor volume by 15% or more, three had tumor progression, and 17 remained stable. Pirfenidone warrants further investigation in NF1, which has until now lacked an effective control therapy.
authors.affiliation: Department of Medical Genetics, Mayo Clinic, 200 First Street SW, Rochester, MN 55905, USA. dbabovic@mayo.edu
authors.forename: D
authors.initials: D
authors.lastname: Babovic-Vuksanovic
authors.forename: K
authors.initials: K
authors.lastname: Ballman
authors.forename: V
authors.initials: V
authors.lastname: Michels
authors.forename: P
authors.initials: P
authors.lastname: McGrann
authors.forename: N
authors.initials: N
authors.lastname: Lindor
authors.forename: B
authors.initials: B
authors.lastname: King
authors.forename: J
authors.initials: J
authors.lastname: Camp
authors.forename: V
authors.initials: V
authors.lastname: Micic
authors.forename: N
authors.initials: N
authors.lastname: Babovic
authors.forename: X
authors.initials: X
authors.lastname: Carrero
authors.forename: R
authors.initials: R
authors.lastname: Spinner
authors.forename: B
authors.initials: B
authors.lastname: O'Neill
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000008:administration & dosage* / Q000009:adverse effects; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D009437:Neuralgia / Q000188:drug therapy / Q000209:etiology / Q000503:physiopathology; D009455:Neurofibroma / Q000188:drug therapy / Q000473:pathology / Q000503:physiopathology; D009456:Neurofibromatosis 1 / Q000188:drug therapy* / Q000473:pathology / Q000503:physiopathology; D010525:Peripheral Nerves / Q000187:drug effects / Q000473:pathology / Q000503:physiopathology; D010524:Peripheral Nervous System Neoplasms / Q000188:drug therapy / Q000473:pathology / Q000503:physiopathology; D011728:Pyridones / Q000008:administration & dosage* / Q000009:adverse effects; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D011728:Pyridones; C093844:pirfenidone