Literature DB >> 17035676

Phase II trial of pirfenidone in adults with neurofibromatosis type 1.

D Babovic-Vuksanovic1, K Ballman, V Michels, P McGrann, N Lindor, B King, J Camp, V Micic, N Babovic, X Carrero, R Spinner, B O'Neill.   

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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Year:  2006        PMID： 17035676     DOI： 10.1212/01.wnl.0000243231.12248.67

Source DB:  PubMed          Journal:  Neurology        ISSN： 0028-3878            Impact factor:   9.910


  20 in total

1.  Preclinical Evidence for the Use of Sunitinib Malate in the Treatment of Plexiform Neurofibromas.

Authors:  Michael J Ferguson; Steven D Rhodes; Li Jiang; Xiaohong Li; Jin Yuan; Xianlin Yang; Shaobo Zhang; Saeed T Vakili; Paul Territo; Gary Hutchins; Feng-Chun Yang; David A Ingram; D Wade Clapp; Shi Chen
Journal:  Pediatr Blood Cancer       Date:  2015-09-16       Impact factor: 3.167

2.  Phase II trial of pirfenidone in children and young adults with neurofibromatosis type 1 and progressive plexiform neurofibromas.

Authors:  Brigitte C Widemann; Dusica Babovic-Vuksanovic; Eva Dombi; Pamela L Wolters; Stewart Goldman; Staci Martin; Anne Goodwin; Wendy Goodspeed; Mark W Kieran; Bruce Cohen; Susan M Blaney; Allison King; Jeffrey Solomon; Nicholas Patronas; Frank M Balis; Elizabeth Fox; Seth M Steinberg; Roger J Packer
Journal:  Pediatr Blood Cancer       Date:  2014-04-22       Impact factor: 3.167

Review 3.  Orbital/Periorbital Plexiform Neurofibromas in Children with Neurofibromatosis Type 1: Multidisciplinary Recommendations for Care.

Authors:  Robert A Avery; James A Katowitz; Michael J Fisher; Gena Heidary; Eva Dombi; Roger J Packer; Brigitte C Widemann
Journal:  Ophthalmology       Date:  2016-11-03       Impact factor: 12.079

4.  Therapeutics for childhood neurofibromatosis type 1 and type 2.

Authors:  Simone L Ardern-Holmes; Kathryn N North
Journal:  Curr Treat Options Neurol       Date:  2011-12       Impact factor: 3.598

Review 5.  Emerging therapeutic targets for neurofibromatosis type 1.

Authors:  James A Walker; Meena Upadhyaya
Journal:  Expert Opin Ther Targets       Date:  2018-05-07       Impact factor: 6.902

6.  Diffuse plexiform neurofibroma of the back: report of a case.

Authors:  Ezella N Washington; Timothy P Placket; Ronald A Gagliano; Jeffery Kavolius; Donald A Person
Journal:  Hawaii Med J       Date:  2010-08

Review 7.  Optimizing biologically targeted clinical trials for neurofibromatosis.

Authors:  David H Gutmann; Jaishri O Blakeley; Bruce R Korf; Roger J Packer
Journal:  Expert Opin Investig Drugs       Date:  2013-02-21       Impact factor: 6.206

Review 8.  Pirfenidone: an anti-fibrotic therapy for progressive kidney disease.

Authors:  Monique E Cho; Jeffrey B Kopp
Journal:  Expert Opin Investig Drugs       Date:  2010-02       Impact factor: 6.206

Review 9.  Neurofibromatosis type 1.

Authors:  Kevin P Boyd; Bruce R Korf; Amy Theos
Journal:  J Am Acad Dermatol       Date:  2009-07       Impact factor: 11.527

Review 10.  Suggested response criteria for phase II antitumor drug studies for neurofibromatosis type 2 related vestibular schwannoma.

Authors:  Scott R Plotkin; Chris Halpin; Jaishri O Blakeley; William H Slattery; D Bradley Welling; Susan M Chang; Jay S Loeffler; Gordon J Harris; A Gregory Sorensen; Michael J McKenna; Fred G Barker
Journal:  J Neurooncol       Date:  2009-05-09       Impact factor: 4.130
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