Zoing Margie C, Burke David, Pamphlett Roger, Kiernan Matthew C
Multidisciplinary Motor Neurone Disease Service, Institute of Neurological Sciences, Prince of Wales Hospital, Sydney, New South Wales, Australia.
J Clin Neurosci. 2006 Jan;13(1):78-83. doi: 10.1016/j.jocn.2004.04.011.
Riluzole is the only therapy proven in clinical trials to prolong survival in patients with motor neurone disease (MND). Prior to its listing by the Australian Pharmaceutical Benefits Advisory Scheme in June 2003, the aim of the present study was to provide Australian patients with MND early access to riluzole and to expand the safety profile data of this therapy. Patients with MND were referred to the programme by neurologists covering the Sydney metropolitan region. To be eligible to receive riluzole, patients had to be aged between 18 and 75 years and have probable or definite MND based on El Escorial criteria, with a disease duration of less than 5 years and a vital capacity greater than 60% before study entry. Patients were prescribed riluzole 50 mg twice daily. Safety data were collected through documentation of adverse events by clinical history in combination with regular laboratory screening. Full blood count, haematocrit, differential white cell counts and serum liver transaminase levels were obtained monthly for 3 months, and then at each 3-monthly visit for 1 year. In total, 25 patients with MND (17 male, 8 female; age range 40-74 years; mean age 59 years) were commenced on riluzole. Of these, 28% had definite MND and the remaining 72% were diagnosed as probable MND, with the majority (84%) having limb-onset MND. At 12 months, 68% of patients continued on riluzole, 16% had died from their disease and 16% ceased riluzole because of side-effects or other reasons, largely disenchantment owing to a perceived lack of efficacy. Haematological and biochemical assays showed no significant alteration during the initial 12-month period. Long-term survival data for patients in the present series suggest a greater benefit for patients who commenced riluzole early in the course of their illness. In conclusion, riluzole was generally tolerated well by Australian patients with MND. Of the few patients who experienced side-effects attributed to riluzole, all were reversible. Issues related to patient perceptions of efficacy highlight the need for patients and treating physicians to maintain realistic expectations of riluzole therapy.
利鲁唑是唯一经临床试验证明可延长运动神经元病（MND）患者生存期的治疗药物。在2003年6月被澳大利亚药品福利咨询计划列入清单之前，本研究的目的是让澳大利亚的MND患者尽早获得利鲁唑，并扩大该治疗方法的安全性数据。悉尼大都市地区的神经科医生将MND患者转诊至该项目。要符合接受利鲁唑治疗的条件，患者年龄必须在18至75岁之间，根据埃斯科里亚尔标准确诊为可能或确诊的MND，疾病持续时间少于5年，且在研究入组前肺活量大于60%。患者被开处方每日两次服用50毫克利鲁唑。通过临床病史记录不良事件并结合定期实验室筛查来收集安全性数据。在3个月内每月进行全血细胞计数、血细胞比容、白细胞分类计数和血清肝转氨酶水平检测，然后在接下来的1年中每3个月进行一次检测。共有25例MND患者（17例男性，8例女性；年龄范围40 - 74岁；平均年龄59岁）开始服用利鲁唑。其中，28%为确诊的MND，其余72%被诊断为可能的MND，大多数（84%）为肢体起病的MND。在12个月时，68%的患者继续服用利鲁唑，16%因疾病死亡，16%因副作用或其他原因停止服用利鲁唑，主要是由于感觉缺乏疗效而不再抱有幻想。血液学和生化检测在最初的12个月期间未显示出显著变化。本系列患者的长期生存数据表明，在疾病病程早期开始服用利鲁唑的患者获益更大。总之，澳大利亚的MND患者对利鲁唑的耐受性总体良好。在少数经历了归因于利鲁唑的副作用的患者中，所有副作用都是可逆的。与患者对疗效的认知相关的问题凸显了患者和治疗医生对利鲁唑治疗保持现实期望的必要性。
