Phase II trial of pirfenidone in adults with neurofibromatosis type 1

D. Babovic-Vuksanovic, Karla V Ballman, V. Michels, P. McGrann, N. Lindor, B. King, J. Camp, V. Micic, N. Babovic, X. Carrero, R. Spinner, B. O'Neill

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51 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)1860-1862
Number of pages3
JournalNeurology
Volume67
Issue number10
DOIs
StatePublished - Nov 1 2006

ASJC Scopus subject areas

  • Clinical Neurology

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    Babovic-Vuksanovic, D., Ballman, K. V., Michels, V., McGrann, P., Lindor, N., King, B., Camp, J., Micic, V., Babovic, N., Carrero, X., Spinner, R., & O'Neill, B. (2006). Phase II trial of pirfenidone in adults with neurofibromatosis type 1. Neurology, 67(10), 1860-1862. https://doi.org/10.1212/01.wnl.0000243231.12248.67