Babovic-Vuksanovic D, Ballman K, Michels V, McGrann P, Lindor N, King B, Camp J, Micic V, Babovic N, Carrero X, Spinner R, O'Neill B
Department of Medical Genetics, Mayo Clinic, 200 First Street SW, Rochester, MN 55905, USA.
Neurology. 2006 Nov 28;67(10):1860-2. doi: 10.1212/01.wnl.0000243231.12248.67. Epub 2006 Oct 11.
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.
我们对24例1型神经纤维瘤病（NF1）患者进行了口服吡非尼酮的开放标签II期试验。通过三维MRI监测肿瘤。治疗结束时，4例患者肿瘤体积缩小15%或更多，3例患者肿瘤进展，17例患者病情稳定。吡非尼酮值得在NF1中进一步研究，迄今为止NF1缺乏有效的对照治疗。
