Efficacy, safety and tolerability of high lipid supplementation in amyotrophic lateral sclerosis

高脂补充剂治疗肌萎缩侧索硬化症的有效性、安全性和耐受性

• 批准号: 201529569
• 负责人: Professor Dr. Albert Christian Ludolph
• 金额: --
• 依托单位: Universitätsklinik für Neurologie
• 依托单位国家: 德国
• 项目类别: Clinical Trials
• 财政年份: 2012
• 资助国家: 德国
• 起止时间: 2011-12-31 至 2016-12-31
• 项目状态: 已结题
• 来源: https://gepris.dfg.de/gepris/projekt/201529569?language=en
• 关键词: Efficacy; safety; tolerability; lipid; supplementation

Amyotrophic lateral sclerosis (ALS) is characterized by the simultaneous degeneration of lower (spinal and bulbar) and upper (corticospinal) motor neurons, leading to progressive muscle atrophy and paralysis. ALS usually leads to death within 2 to 3 years after diagnosis, mostly from respiratory failure. Weight loss is a common phenomenon and an independent prognostic factor in ALS. Several potential causal mechanisms including intrinsic hypermetabolism and deficient food intake have been discussed. Very few clinical efforts focused on nutrition. It was shown in a retrospective data analysis that high serum levels of both fasting cholesterol and triglycerides had a significant positive effect on survival. Life expectancy of patients with high triglyceride levels was prolonged by 14 months. Therefore we will investigate in a multicentre, 1:1-randomised, stratified (onset of disease: spinal and bulbar form; BMI status: low and high BMI), double-blind, placebo-controlled study the effect of a high caloric fatty diet (39.3% lipids) for drinking additionally to daily food intake on survival of ALS patients. 324 patients will be recruited by 12 centres (neurological clinics with specialised motoneuron outpatient units) in Germany. The results of this study will provide an evidence-based evaluation of a nutritional impact, e.g. lipids, on survival of ALS patients. Furthermore the results could improve patient care.

肌萎缩性侧索硬化症（ALS）的特征在于下部（脊髓和延髓）和上部（皮质脊髓）运动神经元的同时变性，导致进行性肌肉萎缩和瘫痪。ALS通常在诊断后2至3年内导致死亡，主要是呼吸衰竭。体重减轻是ALS的一种常见现象，也是独立的预后因素。一些潜在的致病机制，包括内在的高代谢和食物摄入不足进行了讨论。很少有临床工作集中在营养方面。一项回顾性数据分析显示，高血清空腹胆固醇和甘油三酯水平对生存率有显著的积极影响。高甘油三酯水平患者的预期寿命延长了14个月。因此，我们将在一项多中心、1：1随机化、分层（疾病发作：脊柱和延髓形式; BMI状态：低和高BMI）、双盲、安慰剂对照研究中，研究除每日食物摄入外，高热量脂肪饮食（39.3%脂质）饮酒对ALS患者生存率的影响。将在德国的12个中心（具有专门运动神经元门诊部的神经科诊所）招募324例患者。这项研究的结果将提供营养影响的循证评估，例如脂质对ALS患者的生存率。此外，这些结果可以改善患者护理。