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.
Publication types
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Clinical Trial, Phase II
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Research Support, Non-U.S. Gov't
MeSH terms
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Adolescent
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Adult
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Aged
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Antineoplastic Agents / administration & dosage*
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Antineoplastic Agents / adverse effects
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Dose-Response Relationship, Drug
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Drug Administration Schedule
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Female
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Humans
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Magnetic Resonance Imaging
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Male
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Middle Aged
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Neuralgia / drug therapy
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Neuralgia / etiology
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Neuralgia / physiopathology
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Neurofibroma / drug therapy
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Neurofibroma / pathology
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Neurofibroma / physiopathology
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Neurofibromatosis 1 / drug therapy*
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Neurofibromatosis 1 / pathology
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Neurofibromatosis 1 / physiopathology
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Peripheral Nerves / drug effects
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Peripheral Nerves / pathology
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Peripheral Nerves / physiopathology
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Peripheral Nervous System Neoplasms / drug therapy
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Peripheral Nervous System Neoplasms / pathology
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Peripheral Nervous System Neoplasms / physiopathology
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Pyridones / administration & dosage*
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Pyridones / adverse effects
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Treatment Outcome
Substances
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Antineoplastic Agents
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Pyridones
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pirfenidone