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JAMA:非甾体类抗炎剂可明显减缓神经变性疾病的恶化

  1. 二氟尼柳
  2. 安慰剂
  3. 抗炎剂
  4. 神经变性疾病
  5. 非甾体

来源:生物谷 2014-01-04 23:15

在家族性淀粉样多神经病(familial amyloid polyneuropathy)患者中使用一种非甾体类抗炎剂二氟尼柳,相比安慰剂来说可以明显降低个体神经损伤的恶化比率。

2014年1月4日 讯 /生物谷BIOON/ --近日,一项刊登在国际杂志JAMA上的研究报道指出,在家族性淀粉样多神经病(familial amyloid polyneuropathy)患者中使用一种非甾体类抗炎剂二氟尼柳,相比安慰剂来说可以明显降低个体神经损伤的恶化比率。

家族性淀粉样多神经病是一种遗传性的神经变性疾病,其主要表现为恶化的神经缺失状况以及个体残疾,如果患者没有得到及时治疗非常容易致死;这项研究由波士顿大学医学院的研究者John L. Berk来领导,研究者对来自意大利、日本、英国等地随机抽取的130名患者进行长达两年的试验,其中64人使用二氟尼柳进行治疗,而66人使用安慰剂,研究者目的是想确定二氟尼柳在患者多神经病恶化过程中的作用。

结果显示,相比安慰剂组,二氟尼柳组患者的病情恶化程度减轻了,而且在第一年和第二年的时候就可以明显检测到这种减轻效应,安慰剂组减轻比例为9.4%,而二氟尼柳组则为29.7%。

研究者表示,这项研究是首次对这种广谱疾病进行随机试验,其对于研究神经变性疾病的恶化有非常大的研究意义,尽管后期还需要进行更多更为深入的试验,但是当前的研究揭示了二氟尼柳对家族性淀粉样多神经病的有效作用。(生物谷Bioon.com)

Repurposing Diflunisal for Familial Amyloid Polyneuropathy A Randomized Clinical Trial

John L. Berk, MD1; Ole B. Suhr, MD, PhD2; Laura Obici, MD3; Yoshiki Sekijima, MD, PhD4; Steven R. Zeldenrust, MD, PhD5; Taro Yamashita, MD, PhD6; Michael A. Heneghan, MD7; Peter D. Gorevic, MD10; William J. Litchy, MD5; Janice F. Wiesman, MD1; Erik Nordh, MD, PhD2; Manuel Corato, MD, PhD8; Alessandro Lozza, MD9; Andrea Cortese, MD9; Jessica Robinson-Papp, MD10; Theodore Colton, ScD11; Denis V. Rybin, MS12; Alice B. Bisbee, MPH12; Yukio Ando, MD, PhD6; Shu-ichi Ikeda, MD, PhD4; David C. Seldin, MD, PhD1; Giampaolo Merlini, MD3; Martha Skinner, MD1; Jeffery W. Kelly, PhD13; Peter J. Dyck, MD5; for the Diflunisal Trial Consortium

Importance Familial amyloid polyneuropathy, a lethal genetic disease caused by aggregation of variant transthyretin, induces progressive peripheral nerve deficits and disability. Diflunisal, a nonsteroidal anti-inflammatory agent, stabilizes transthyretin tetramers and prevents amyloid fibril formation in vitro. Objective To determine the effect of diflunisal on polyneuropathy progression in patients with familial amyloid polyneuropathy. Design, Setting, and Participants International randomized, double-blind, placebo-controlled study conducted among 130 patients with familial amyloid polyneuropathy exhibiting clinically detectable peripheral or autonomic neuropathy at amyloid centers in Sweden (Umeå), Italy (Pavia), Japan (Matsumoto and Kumamoto), England (London), and the United States (Boston, Massachusetts; New York, New York; and Rochester, Minnesota) from 2006 through 2012. Intervention Participants were randomly assigned to receive diflunisal, 250 mg (n=64), or placebo (n=66) twice daily for 2 years. Main Outcomes and Measures The primary end point, the difference in polyneuropathy progression between treatments, was measured by the Neuropathy Impairment Score plus 7 nerve tests (NIS+7) which ranges from 0 (no neurological deficits) to 270 points (no detectable peripheral nerve function). Secondary outcomes included a quality-of-life questionnaire (36-Item Short-Form Health Survey [SF-36]) and modified body mass index. Because of attrition, we used likelihood-based modeling and multiple imputation analysis of baseline to 2-year data. Results By multiple imputation, the NIS+7 score increased by 25.0 (95% CI, 18.4-31.6) points in the placebo group and by 8.7 (95% CI, 3.3-14.1) points in the diflunisal group, a difference of 16.3 points (95% CI, 8.1-24.5 points; P < .001). Mean SF-36 physical scores decreased by 4.9 (95% CI, −7.6 to −2.2) points in the placebo group and increased by 1.5 (95% CI, −0.8 to 3.7) points in the diflunisal group (P < .001). Mean SF-36 mental scores declined by 1.1 (95% CI, −4.3 to 2.0) points in the placebo group while increasing by 3.7 (95% CI, 1.0-6.4) points in the diflunisal group (P = .02). By responder analysis, 29.7% of the diflunisal group and 9.4% of the placebo group exhibited neurological stability at 2 years (<2-point increase in NIS+7 score; P = .007). Conclusions and Relevance Among patients with familial amyloid polyneuropathy, the use of diflunisal compared with placebo for 2 years reduced the rate of progression of neurological impairment and preserved quality of life. Although longer-term follow-up studies are needed, these findings suggest benefit of this treatment for familial amyloid polyneuropathy. Trial Registration clinicaltrials.gov Identifier: NCT00294671

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