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

肌萎缩性侧索硬化症（ALS）的特点是下部（脊髓和球）和上部（皮质脊髓）运动神经元同时变性，导致进行性肌肉萎缩和瘫痪。ALS通常在诊断后2至3年内死亡，主要死于呼吸衰竭。体重减轻是ALS的常见现象，也是一个独立的预后因素。讨论了包括内在高代谢和食物摄入不足在内的几种潜在的因果机制。很少有临床研究关注营养。回顾性数据分析表明，空腹胆固醇和甘油三酯的高血清水平对生存率有显著的积极影响。甘油三酯水平高的患者预期寿命延长14个月。因此，我们将在一项多中心、1:1随机、分层（发病：脊柱和球型；BMI状态：低BMI和高BMI）、双盲、安慰剂对照研究中调查高热量脂肪饮食（39.3%脂质）对ALS患者生存的影响。324名患者将被德国的12个中心（具有专门运动神经元门诊单位的神经病学诊所）招募。这项研究的结果将提供营养影响的循证评估，如脂质，对ALS患者的生存。此外，研究结果可以改善病人的护理。

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.