Phase II Trial of Pirfenidone in Adults with Neurofibromatosis Type 1

Overview

Journal Neurology

Specialty Neurology

Date 2006 Oct 13

PMID 17035676

Citations 25

Authors

D Babovic-Vuksanovic

K Ballman

V Michels

P McGrann

N Lindor

B King

J Camp

V Micic

N Babovic

X Carrero

R Spinner

B ONeill

Affiliations

Soon will be listed here.

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.

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