Dalakas M C, Stein D P, Otero C, Sekul E, Cupler E J, McCrosky S
Neuromuscular Diseases Section, National Institutes of Health, Bethesda, Md.
Arch Neurol. 1994 Sep;51(9):861-4. doi: 10.1001/archneur.1994.00540210031010.
To determine if high-dose intravenous immunoglobulin therapy is effective in improving muscle strength or in arresting the pace of disease progression in patients with rapidly progressive amyotrophic lateral sclerosis.
An open-label pilot study of intravenous infusions of high-dose immunoglobulin administered once a month for 3 months in nine patients with classic amyotrophic lateral sclerosis. Selected patients had a rapidly progressive course with signs of worsening noticeably evident every 6 weeks prior to therapy. A patient with multifocal motor neuropathy with conduction block that presented as a lower motor neuron syndrome was concurrently treated to document the efficacy of the same preparation of immunoglobulin in a potentially treatable disease that simulates lower motor neuron syndrome. The efficacy of high-dose intravenous immunoglobulin infusions was assessed by objective measurement of maximum voluntary isometric contraction in all muscle groups of two limbs before and after therapy.
The Clinical Center of the National Institutes of Health, Bethesda, Md.
All patients with amyotrophic lateral sclerosis worsened during the study. By the end of the third month, their mean total muscle scores (megascores) had declined by 71.2 points, from a mean of 369.7 (range, 200 to 605) to 298.5 (range, 130 to 552) points. The pace of progression did not change during the 4-month observation period. In contrast, the patient with multifocal motor neuropathy responded to intravenous immunoglobulin therapy and increased his megascores by 146 points after 3 months. The GM1 antibody titers were normal in all the patients.
High-dose intravenous immunoglobulin, a prohibitively expensive drug, has no apparent therapeutic role in improving the symptoms or arresting the pace of progression in patients with amyotrophic lateral sclerosis. In contrast, multifocal motor neuropathy is an immunopathologically different disease that responds to intravenous immunoglobulin therapy.
确定大剂量静脉注射免疫球蛋白疗法是否能有效改善快速进展性肌萎缩侧索硬化症患者的肌肉力量或阻止疾病进展速度。
一项开放标签的试点研究，对9例经典型肌萎缩侧索硬化症患者每月静脉输注一次大剂量免疫球蛋白，共3个月。入选患者病程进展迅速，在治疗前每6周症状明显恶化。一名表现为下运动神经元综合征的多灶性运动神经病伴传导阻滞患者同时接受治疗，以证明相同制剂的免疫球蛋白在一种模拟下运动神经元综合征的潜在可治疗疾病中的疗效。通过客观测量治疗前后双下肢所有肌肉群的最大随意等长收缩来评估大剂量静脉注射免疫球蛋白输注的疗效。
马里兰州贝塞斯达国立卫生研究院临床中心。
所有肌萎缩侧索硬化症患者在研究期间病情均恶化。到第三个月末，他们的平均总肌肉评分（总分）下降了71.2分，从平均369.7分（范围200至605分）降至298.5分（范围130至552分）。在4个月的观察期内，疾病进展速度没有变化。相比之下，多灶性运动神经病患者对静脉注射免疫球蛋白治疗有反应，3个月后其总分增加了146分。所有患者的GM1抗体滴度均正常。
大剂量静脉注射免疫球蛋白是一种价格昂贵的药物，对改善肌萎缩侧索硬化症患者的症状或阻止疾病进展速度没有明显的治疗作用。相比之下，多灶性运动神经病是一种免疫病理学上不同的疾病，对静脉注射免疫球蛋白治疗有反应。
