Dalakas M C, Sonies B, Dambrosia J, Sekul E, Cupler E, Sivakumar K
Neuromuscular Diseases Section, Medical Neurology Branch, NINDS, National Institutes of Health, Bethesda, MD 20892-1382, USA.
Neurology. 1997 Mar;48(3):712-6. doi: 10.1212/wnl.48.3.712.
We randomized 19 patients with inclusion-body myositis (IBM) to a double-blind, placebo-controlled, crossover study using monthly infusions of 2 g/kg intravenous immunoglobulin (IVIg) or placebo for 3 months. Patients crossed over to the alternate treatment after a washout period. We evaluated responses at baseline and at the end of each treatment period using expanded (0-10) MRC scales, the Maximum Voluntary Isometric Contraction (MVIC) method, symptom and disability scores, and quantitative swallowing studies. We calculated the differences in scores between IVIg and placebo from baseline to end of treatment. Of the 19 patients, 9 (mean age, 61.2 years; mean disease duration, 5.6 years) were randomized to IVIg and 10 (mean age, 66.1 years; mean disease duration, 7.4 years) to placebo. During IVIg the patients gained a mean of 4.2 (-16 to +39.8) MRC points, and during placebo lost 2.7 (-10 to +8) points (p < 0.1). These gains were not significant. Similar results were obtained with the MRC and MVIC scores when the patients crossed to the alternate treatment. Six patients had a functionally important improvement by more than 10 MRC points that declined when crossed over to placebo. Limb-by-limb analysis demonstrated that during IVIg the muscle strength in 39% of the lower extremity limbs significantly increased compared with placebo (p < 0.05), while a simultaneous decrease in 28% of other limbs was detected. The clinical importance of these minor gains is unclear. The duration of swallowing functions measured in seconds with ultrasound improved statistically in the IVIg-randomized patients (p < 0.05) compared with placebo. Although the study did not establish efficacy of IVIg, possibly because of the small sample size, the drug induced functionally important improvement in 6 (28%) of the 19 patients. Whether the modest gains noted in certain muscle groups justify the high cost of trying IVIg in IBM patients at a given stage of the disease remains unclear.
我们将19例包涵体肌炎（IBM）患者随机分组，进行一项双盲、安慰剂对照的交叉研究，每月静脉输注2 g/kg静脉注射免疫球蛋白（IVIg）或安慰剂，为期3个月。在洗脱期后，患者交叉接受替代治疗。我们使用扩展的（0 - 10）MRC量表、最大自主等长收缩（MVIC）方法、症状和残疾评分以及定量吞咽研究，在基线和每个治疗期结束时评估反应。我们计算了从基线到治疗结束时IVIg和安慰剂之间的评分差异。19例患者中，9例（平均年龄61.2岁；平均病程5.6年）被随机分配至IVIg组，10例（平均年龄66.1岁；平均病程7.4年）被分配至安慰剂组。接受IVIg治疗期间，患者MRC评分平均增加4.2（-16至+39.8）分，而接受安慰剂治疗期间平均下降2.7（-10至+8）分（p < 0.1）。这些增加并不显著。当患者交叉接受替代治疗时，MRC和MVIC评分也得到了类似结果。6例患者功能上有重要改善，MRC评分提高超过10分，但交叉接受安慰剂治疗时改善程度下降。逐肢体分析表明，接受IVIg治疗期间，39%的下肢肌肉力量与安慰剂相比显著增加（p < 0.05），同时发现28%的其他肢体肌肉力量下降。这些微小改善的临床意义尚不清楚。与安慰剂相比，IVIg随机分组患者中，通过超声以秒为单位测量的吞咽功能持续时间有统计学意义的改善（p < 0.05）。尽管该研究未证实IVIg的疗效，可能是由于样本量小，但该药物使19例患者中的6例（28%）出现了功能上的重要改善。在疾病的特定阶段，在IBM患者中尝试使用IVIg所带来的适度改善是否足以证明其高昂成本，仍不明确。
