Dorst Johannes, Ludolph Albert C, Huebers Annemarie
Universitätsklinik Ulm, RKU, Oberer Eselsberg 45, D-89081 Ulm, Germany.
Universitätsklinik Ulm, Ulm, Germany.
Ther Adv Neurol Disord. 2017 Oct 9;11:1756285617734734. doi: 10.1177/1756285617734734. eCollection 2018.
In this review, we summarize the most important recent developments in the treatment of amyotrophic lateral sclerosis (ALS). In terms of disease-modifying treatment options, several drugs such as dexpramipexole, pioglitazone, lithium, and many others have been tested in large multicenter trials, albeit with disappointing results. Therefore, riluzole remains the only directly disease-modifying drug. In addition, we discuss antisense oligonucleotides (ASOs) as a new and potentially causal treatment option. Progress in symptomatic treatments has been more important. Nutrition and ventilation are now an important focus of ALS therapy. Several studies have firmly established that noninvasive ventilation improves patients' quality of life and prolongs survival. On the other hand, there is still no consensus regarding best nutritional management, but big multicenter trials addressing this issue are currently ongoing. Evidence regarding secondary symptoms like spasticity, muscle cramps or sialorrhea remains generally scarce, but some new insights will also be discussed. Growing evidence suggests that multidisciplinary care in specialized clinics improves survival.
在本综述中，我们总结了肌萎缩侧索硬化症（ALS）治疗方面最近最重要的进展。在疾病修饰治疗选择方面，诸如右丙氧芬、吡格列酮、锂等多种药物已在大型多中心试验中进行了测试，尽管结果令人失望。因此，利鲁唑仍然是唯一直接具有疾病修饰作用的药物。此外，我们讨论了反义寡核苷酸（ASO）作为一种新的且可能具有病因治疗作用的选择。对症治疗方面的进展更为重要。营养和通气现在是ALS治疗的一个重要重点。多项研究已明确证实无创通气可改善患者生活质量并延长生存期。另一方面，关于最佳营养管理仍未达成共识，但目前正在进行解决这一问题的大型多中心试验。关于痉挛、肌肉痉挛或流涎等继发症状的证据总体上仍然很少，但也将讨论一些新的见解。越来越多的证据表明，在专科诊所进行多学科护理可提高生存率。
