Phase II trial of pirfenidone in adults with neurofibromatosis type 1.
case_series · Level IV
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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.
Medical subject headings
- Antineoplastic Agents
- Neurofibromatosis 1
- Pyridones