TY - JOUR
T1 - Phase II trial of pirfenidone in adults with neurofibromatosis type 1
AU - Babovic-Vuksanovic, D.
AU - Ballman, K.
AU - Michels, V.
AU - McGrann, P.
AU - Lindor, N.
AU - King, B.
AU - Camp, J.
AU - Micic, V.
AU - Babovic, N.
AU - Carrero, X.
AU - Spinner, R.
AU - O'Neill, B.
N1 - Funding Information:
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.).
PY - 2006/11
Y1 - 2006/11
N2 - 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.
AB - 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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U2 - 10.1212/01.wnl.0000243231.12248.67
DO - 10.1212/01.wnl.0000243231.12248.67
M3 - Article
C2 - 17035676
AN - SCOPUS:33845413549
SN - 0028-3878
VL - 67
SP - 1860
EP - 1862
JO - Neurology
JF - Neurology
IS - 10
ER -