Hahn A F, Bolton C F, Zochodne D, Feasby T E
University of Western Ontario, London, Canada.
Brain. 1996 Aug;119 ( Pt 4):1067-77. doi: 10.1093/brain/119.4.1067.
Thirty patients with definite or probable chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) of chronic progressive (16 patients) or relapsing (14 patients) course were randomly assigned to receive intravenous immunoglobulin (IvIg) 0.4 g per kg body weight or a placebo treatment on 5 consecutive days in a double-blind, cross-over trial. Neurological function was monitored by serial quantitative assessments [neurological disability score (NDS); clinical grade (CG) and grip strength (GS) measurements] and by electrophysiological studies before and after each treatment period. Twenty-five patients completed both treatment periods. A comparison of the observed changes in clinical outcome measures revealed statistically significant differences in favour of IvIg, with (mean +/- SD) improvements in NDS by 24.4 +/- 5.4 points (P < 0.002) in CG by 1 +/- 0.3 points (P < 0.001) in GS by +6.3 +/- 1.7 kg (P < 0.005), whereas scores were unchanged or worse with placebo. A secondary two-groups analysis of the first trial period included all 30 patients; 16 patients had been randomly assigned to IvIg and 14 to placebo treatments. Again significant differences in favour of IvIg were observed in all the clinical end-points: improvement in NDS was 35.6 +/- 25 points (P < 0.0001), in CG it was 1.3 +/- 1.9 points (P < 0.002) and in GS +9.8 +/- 7.7 kg (P < 0.001), whereas all scores worsened with placebo. Of the 30 patients, 19 (63%) improved with IvIg treatments; nine out of 16 patients (56%) with chronic progressive CIDP, and 10 out of 14 patients (71%) with relapsing CIDP (differences were not statistically significant). A placebo response was seen in five patients. Comparison of paired electrophysiological measurements before and 4 weeks after IvIg treatments revealed statistically significant improvements in the summed motor conduction velocities (sigma MCV; P < -0.0001) and in the summed compound muscle action potentials (CMAP) evoked with proximal stimulation (sigma proximal CMAP, P < 0.03) of median, ulnar, peroneal and tibial nerves. Eight of nine IvIg responders with chronic progressive CIDP improved gradually to normal function with a single 5 day course of IvIg; in five of these, small doses of prednisone were prescribed during follow-up. In 10 IvIg responders with relapsing CIDP, improvements lasted a median 6 weeks (range 3-22 weeks) and was reproducible with open label treatments. All 10 patients have been maintained and stabilized with IvIg pulse therapy of 1 g per kg body weight or less, given as a single infusion prior to the expected relapse. A beneficial response to IvIg was found to be most likely in patients with acute relapse or with disease of one year or less. Patients with predominantly sensory signs did not improve.
30例慢性进行性（16例）或复发型（14例）病程的确诊或疑似慢性炎症性脱髓鞘性多发性神经根神经病（CIDP）患者，在一项双盲、交叉试验中被随机分配，连续5天接受静脉注射免疫球蛋白（IvIg），剂量为每千克体重0.4 g，或接受安慰剂治疗。在每个治疗期前后，通过系列定量评估[神经功能障碍评分（NDS）、临床分级（CG）和握力（GS）测量]以及电生理研究来监测神经功能。25例患者完成了两个治疗期。对观察到的临床结局指标变化进行比较，结果显示IvIg组具有统计学显著差异，NDS改善（均值±标准差）24.4±5.4分（P<0.002），CG改善1±0.3分（P<0.001），GS增加6.3±1.7 kg（P<0.005），而安慰剂组评分无变化或恶化。对第一个试验期进行的两组分析纳入了所有30例患者；16例患者被随机分配接受IvIg治疗，14例接受安慰剂治疗。在所有临床终点再次观察到支持IvIg的显著差异：NDS改善35.6±25分（P<0.0001），CG改善1.3±1.9分（P<0.002），GS增加9.8±7.7 kg（P<0.001），而安慰剂组所有评分均恶化。30例患者中，19例（63%）接受IvIg治疗后病情改善；16例慢性进行性CIDP患者中有9例（56%），14例复发型CIDP患者中有10例（71%）（差异无统计学意义）。5例患者出现安慰剂反应。对IvIg治疗前和治疗后4周的配对电生理测量结果进行比较，结果显示正中神经、尺神经、腓总神经和胫神经的运动传导速度总和（σMCV；P<-0.0001）以及近端刺激诱发的复合肌肉动作电位总和（CMAP）（σ近端CMAP，P<0.03）有统计学显著改善。9例慢性进行性CIDP的IvIg反应者中，8例通过单次5天疗程的IvIg逐渐改善至正常功能；其中5例在随访期间服用了小剂量泼尼松。10例复发型CIDP的IvIg反应者中，改善持续的中位数为6周（范围3 - 22周），且开放标签治疗可再现。所有10例患者通过每千克体重1 g或更低剂量的IvIg脉冲治疗得以维持病情稳定，在预期复发前单次输注给药。发现对IvIg有有益反应的最可能是急性复发或病程在一年及以内的患者。以感觉症状为主的患者病情未改善。
