Phase II trial of pirfenidone in adults with neurofibromatosis type 1.
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.
Babovic-Vuksanovic
D
D
Department of Medical Genetics, Mayo Clinic, 200 First Street SW, Rochester, MN 55905, USA. dbabovic@mayo.edu
Ballman
K
K
Michels
V
V
McGrann
P
P
Lindor
N
N
King
B
B
Camp
J
J
Micic
V
V
Babovic
N
N
Carrero
X
X
Spinner
R
R
O'Neill
B
B
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
D000970:Antineoplastic Agents; D011728:Pyridones; C093844:pirfenidone