Corcia Philippe, Meininger Vincent
Fédération des maladies du système nerveux, Centre référent SLA, Hôpital Pitié Salpetrière, Paris, France.
Drugs. 2008;68(8):1037-48. doi: 10.2165/00003495-200868080-00003.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease affecting the motor neurons, in both the spinal cord and medulla (lower motor neurons) and cerebral cortex (upper motor neurons). Even though ALS remains fatal, several advances have been made during the last decade in improving the consequences of motor dysfunction, quality of life and survival time of patients. Treatment of ALS cannot be restricted to riluzole, the only molecule that has been proved to modify the evolution of the disease. Symptomatic treatments have an important role in controlling the major consequences of the disease, such as pain, sleep disorders, spasticity, hypersialhorroea, emotional lability, depression and digestive disorders (constipation and reflux). All these symptoms need to be recognized and their possible causes identified in order to provide the most appropriate management of patients with ALS. However, an advance in the daily care of patients is the identification of two important phenomena that occur during the evolution of the disease: swallowing difficulties and the occurrence of diaphragmatic dysfunction. For both, specific medical interventions have been developed to allow correction of the consequences (i.e. weight loss and respiratory insufficiency). Although no controlled trials have been performed, observational studies suggest that gastrostomy and non-invasive ventilation may improve at least quality of life and survival. All of these various approaches, pharmaceutical and non-pharmaceutical therapies, are prescribed according to individual symptoms and require the involvement of a large range of health professionals. This multidisciplinary approach in ALS clinics is considered to be one of the more important factors impacting on survival rate and appears to be the gold standard of medical care of ALS patients. Important findings have been made in understanding the nature of the degenerative process that affects the motor neurons. All these data have allowed new therapeutic molecules to be tested alone or in combination with riluzole. Despite the negative results obtained until now, we hope to demonstrate very soon a greater improvement in therapy.
肌萎缩侧索硬化症(ALS)是一种影响运动神经元的神经退行性疾病,这些运动神经元分布于脊髓和延髓(下运动神经元)以及大脑皮层(上运动神经元)。尽管ALS仍然是致命的,但在过去十年中,在改善运动功能障碍的后果、患者生活质量和生存时间方面已经取得了一些进展。ALS的治疗不能仅限于利鲁唑,这是唯一被证明能改变疾病进展的药物。对症治疗在控制疾病的主要后果方面起着重要作用,如疼痛、睡眠障碍、痉挛、流涎过多、情绪不稳定、抑郁和消化紊乱(便秘和反流)。所有这些症状都需要被识别并找出其可能的原因,以便为ALS患者提供最恰当的管理。然而,在患者日常护理方面的一个进展是识别出疾病进展过程中出现的两个重要现象:吞咽困难和膈肌功能障碍。针对这两种情况,已经开发出了特定的医学干预措施来纠正其后果(即体重减轻和呼吸功能不全)。尽管尚未进行对照试验,但观察性研究表明,胃造口术和无创通气至少可以改善生活质量和延长生存期。所有这些不同的方法,包括药物治疗和非药物治疗,都是根据个体症状来开具处方的,并且需要众多医疗专业人员的参与。ALS诊所的这种多学科方法被认为是影响生存率的更重要因素之一,并且似乎是ALS患者医疗护理的金标准。在理解影响运动神经元的退行性过程的本质方面已经取得了重要发现。所有这些数据使得新的治疗分子得以单独或与利鲁唑联合进行测试。尽管到目前为止得到的都是负面结果,但我们希望很快能证明治疗有更大的改善。