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Published online before print October 11, 2006, doi:10.1212/01.wnl.0000243231.12248.67)
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Volume 67, Number 10, November 28, 2006
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NEUROLOGY 2006;67:1860-1862
© 2006 American Academy of Neurology


Brief Communications

Phase II trial of pirfenidone in adults with neurofibromatosis type 1

D. Babovic-Vuksanovic, MD, K. Ballman, PhD, V. Michels, MD, P. McGrann, MD, N. Lindor, MD, B. King, MD, J. Camp, V. Micic, N. Babovic, X. Carrero, R. Spinner, MD and B. O’Neill, MD

From Department of Medical Genetics (D.B.-V., V.M., P.M., N.L.), Radiology (B.K.), Neurology (B.O.), and Neurosurgery (R.S.); Biomedical Imaging Resource (J.C., V.M. N.B.); and Health Sciences Research (X.C.), Mayo College of Medicine, Rochester, MN

Address correspondence and reprint requests to Dr. Dusica Babovic-Vuksanovic, Department of Medical Genetics, Mayo Clinic, 200 First Street SW, Rochester, MN 55905; e-mail: dbabovic{at}mayo.edu

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.


Additional material related to this article can be found on the Neurology Web site. Go to www.neurology.org and scroll down the Table of Contents for the November 28 issue to find the title link for this article.

This article was previously published in electronic format as an Expedited E-Pub on October 11, 2006, at www.neurology.org.

Supported by Clinical Investigator Award, Mayo Foundation (D.B.-V). Neurofibromatosis Inc., Minnesota Chapter, provided financial support for volumetric analysis of MRIs (grant NFIM#1 to D.B.-V.).

Disclosure: Pirfenidone was provided by Solanan, Inc. The authors report no other conflicts of interest.

These data were presented as a poster at the Congress of European Society for Human Genetics, Prague, May 12 to 16, 2005.

Received December 1, 2005. Accepted in final form August 1, 2006.


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