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雷帕霉素对多发性硬化症的疗效显著。

Promising effect of rapamycin on multiple sclerosis.

机构信息

Acquired Immunodeficiency Research Center, Isfahan University of Medical Sciences, Isfahan, Iran; Gastrointestinal and Hepatobiliary Diseases Research Center, Poursina Hakim Research Institute for Health Care Development, Isfahan, Iran.

Cellular, Molecular and Genetics Research Center, Isfahan, Iran; Departments of Genetics and Molecular Biology, School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran.

出版信息

Mult Scler Relat Disord. 2018 Nov;26:40-45. doi: 10.1016/j.msard.2018.08.009. Epub 2018 Aug 10.

Abstract

The routine therapies for relapsing-remitting multiple sclerosis (RRMS) are common disease-modifying medications, yet are not effective in all patients. The aim of the present clinical trial was to evaluate the therapeutic effects of rapamycin on the clinical and radiological aspects, regulatory T cells proliferation and FOXP3 and GARP gene expression in the patients with RRMS. In this study, eight patients with RRMS were chosen and included in the trial. Patients received rapamycin (Rapacan, Biocon, India) for six months. Magnetic resonance imaging (MRI) of the patients' brain was taken before and after the therapy. Patients' expanded disability status scale (EDSS), and FoxP3 and GARP gene expression, and Treg cell proliferation were also been evaluated. All the patients had some degrees of significant reduction in mean plaque area size (P = 0.012, Z = -2.520), and minimum and maximum size of the plaques (P = 0.012, Z = -2.521). EDSS of 50% of patients was decreased after the treatment, yet it was not significant (P = 0.059, Z = -1.89). The expression rate of FOXP3 (P = 0.003) and GARP genes in Tregs increased after the therapy. We found a promising response to rapamycin among our cases with minor side effects and it may be considered as a therapeutic option of this disease.

摘要

本临床试验旨在评估雷帕霉素对复发缓解型多发性硬化症(RRMS)患者临床和影像学方面、调节性 T 细胞增殖以及 FOXP3 和 GARP 基因表达的治疗效果。在这项研究中,选择了 8 名 RRMS 患者参与试验。患者接受雷帕霉素(Rapacan,Biocon,印度)治疗六个月。在治疗前后对患者的大脑进行磁共振成像(MRI)检查。还评估了患者的扩展残疾状态量表(EDSS)以及 FOXP3 和 GARP 基因表达和 Treg 细胞增殖。所有患者的平均斑块面积大小(P=0.012,Z=-2.520)以及斑块的最小和最大尺寸(P=0.012,Z=-2.521)均有一定程度的显著减少。治疗后,有 50%的患者 EDSS 下降,但无统计学意义(P=0.059,Z=-1.89)。治疗后 Tregs 中 FOXP3(P=0.003)和 GARP 基因的表达率增加。我们发现雷帕霉素在我们的病例中具有良好的反应,副作用较小,可作为该病的治疗选择之一。

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