重新使用双氟尼拉治疗家族性淀粉样蛋白多发性神经病
2013年12月25日
An international consortium of investigators led by 淀粉样变中心 Clinical Director Dr. John Berk have completed a randomized, placebo-controlled, phase III clinical trial of the non-steroidal anti-inflammatory drug diflunisal for patients with ATTR polyneuropathy, published today in the 美国医学协会杂志. 130名参与澳门威尼斯人注册网站研究的患者接受了diflunisal（250毫克，每天两次）或安慰剂治疗，疗程长达两年。 双氟尼柳达到了主要终点，显示神经病变损害评分+ 7的进展显著减少，其次要终点是生活质量，通过SF-36问卷测量。 Details can be found on the JAMA 网站. This ground-breaking research derived from biophysical studies of Dr. Jeffery Kelly and colleagues of the Scripps Institute in La Jolla, California, and used techniques for measuring neuropathy developed by Dr. Peter Dyck of the Mayo Clinic. The diflunisal clinical trial was supported by the National Institute of Neurological Diseases and Stroke at the NIH (R01 NS051306), the Food and Drug Administration Office of Orphan Products Development (FD-R 002532), the Young Family Amyloid 澳门威尼斯人注册网站研究 Fund, the BU Clinical and Translational Science Institute, and by private donors; 默克夏普和多美公司捐赠了双氟尼松。
Further information and Dr. Berk’s comments can be found in the 波士顿大学 School of Medicine and 波士顿医疗中心 press release: 迪氟尼司新闻稿
This story was featured in Deborah Kotz’s “Daily Dose” blog on Boston.com: 每日剂量，2013年12月26日