Department of Medicine & Data Coordinating Center, Boston University Medical Center, Boston, MA 02118, USA.
Amyloid. 2012 Jun;19 Suppl 1:37-8. doi: 10.3109/13506129.2012.678509. Epub 2012 May 2.
Familial amyloidotic polyneuropathy (FAP) is a protein folding disorder that induces neuropathy and cardiomyopathy, leading to death within 7-15 years after onset of clinical disease. In vitro, small ligands binding the thyroid hormone docking site stabilize tetrameric transthyretin, inhibiting amyloid fibril formation. We undertook a randomized, placebo-controlled clinical trial to determine whether diflunisal, a well-known non-steroidal anti-inflammatory drug (NSAID) alters neurologic disease progression in FAP. We enrolled 130 subjects with wide age and FAP mutation representation. To date, few recognized complications of NSAIDs have occurred in the study cohort. Data collection will be completed by November 2012.
家族性淀粉样多神经病（FAP）是一种蛋白折叠紊乱疾病，可引起神经病和心肌病，在临床疾病发病后 7-15 年内导致死亡。在体外，结合甲状腺激素停泊位的小分子配体稳定四聚转甲状腺素，抑制淀粉样纤维形成。我们进行了一项随机、安慰剂对照临床试验，以确定熟知的非甾体抗炎药（NSAID）双氯芬酸是否改变 FAP 的神经病变进展。我们纳入了 130 名具有广泛年龄和 FAP 突变代表性的受试者。迄今为止，研究队列中仅出现了少数 NSAID 的公认并发症。数据收集将于 2012 年 11 月完成。
