A PLASMA-EXCHANGE VERSUS IMMUNE GLOBULIN INFUSION TRIAL IN CHRONIC INFLAMMATORY DEMYELINATING POLYRADICULONEUROPATHY
A PLASMA-EXCHANGE VERSUS IMMUNE GLOBULIN INFUSION TRIAL IN CHRONIC INFLAMMATORY DEMYELINATING POLYRADICULONEUROPATHY
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DOI：
10.1002/ana.410360607
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发表时间：
1994-12-01
影响因子：
11.2
通讯作者：
OBRIEN, PC
中科院分区：
文献类型：
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作者：
DYCK, PJ;LITCHY, WJ;OBRIEN, PC
慢性炎症性脱髓鞘性多发性神经根神经病是一种麻痹综合征，可导致严重残疾甚至死亡。在对照临床试验中，血浆置换可以预防或改善神经功能缺损，但免疫球蛋白输注的功效尚未得到证实。同样未知的是免疫球蛋白输注是否与血浆置换一样有效或更有效，以及最佳剂量和频率。在这项观察者盲法研究中，使用一些不受偏倚影响的客观终点（例如，复合肌肉动作电位总和），20 名患有进行性或静态性多发性神经病的患者被随机分配接受两种治疗中的任何一种治疗，持续 6 周，随后是洗脱期，然后被分配接受另一种治疗。使用血浆置换（每周两次，持续 3 周，然后每周一次，持续 3 周）和免疫球蛋白输注（0.4 gm/kg，每周一次，持续 3 周，然后在接下来的 3 周，每周一次 0.2 gm/kg）。治疗方案前后评估的终点是神经功能障碍评分；肌无力的神经功能障碍评分；尺神经、正中神经和腓神经的复合肌肉动作电位总和；尺神经和腓肠神经的感觉神经动作电位总和；以及使用案例 IV 的大脚趾振动检测阈值。观察者对所使用的治疗方法不知情。在 20 名患者中，13 名患者接受了两种治疗，而 4 名患者病情并未恶化到足以接受第二次治疗的程度——1 名患者在第一次治疗期间离开研究，2 名患者在第一次治疗后离开研究，前往其他地方接受计划外的治疗。在任一治疗期间接受血浆置换的 17 名患者中，神经功能障碍评分（38.3 +/- 34.6 分，平均值 +/- 标准差，p < 0.001）、评分的无力子集（33.4 +/- 29.5 分，p < 0.001）和复合肌肉动作电位总和（3.7 +/- 3.5 mV，p < 0.001）均出现统计学显着改善。这种改善是巨大且明确的，相当于从双侧骨盆和下肢肌肉50%无力变为无无力。在任一方案中接受免疫球蛋白输注的 15 名患者中，可比较的变化如下：神经系统评分，36.1 +/- 32.0 分（p = 0.006）；神经系统评分的弱点子集，31.4 +/- 31.5 分 (p < 0.002)；和总肌肉动作电位，3.3 +/- 2.8 mV (p < 0.001)。在比较两种治疗之间的神经病理终点变化时，没有发现任何终点有统计学上的显着差异。在一项对 8 名患者进行的公开试验中，为了确定维持良好功能所需的免疫球蛋白输注频率和剂量，我们发现可以通过每隔 6 周评估神经功能障碍评分和肌肉动作电位总和来调整频率。达到高功能水平所需的剂量和频率在患者之间差异很大。两种治疗均可有效改善慢性炎症性脱髓鞘性多发性神经根神经病的神经功能缺损。效果很大，但对于大多数患者来说是短暂的，需要持续间歇性治疗以获得最大或持续的改善。对于费用大致相同的治疗方案，血浆置换和免疫球蛋白输注之间的疗效没有显示出明显的差异。由于免疫球蛋白无需昂贵的设备即可输注，并且可以在家中进行，因此它可能是更好的治疗方法。维持改善所需的输注球蛋白的频率和剂量变化很大（从每 10 天一次 0.025 克/公斤到每 2-4 天一次 0.4 克/公斤[很少更多]）。
Chronic inflammatory demyelinating polyradiculoneuropathy is a paralytic syndrome, causing considerable disability and even death. In controlled clinical trials, plasma exchange prevented or ameliorated neurological deficits, but the efficacy of immune globulin infusion remains unproved. Also unknown is whether immune globulin infusion is as effective, or more effective, than plasma exchange and what dosages and frequencies are best. In this observer-blinded study, using some objective end points not subject to bias (e.g., summated compound muscle action potential), 20 patients with progressive or static polyneuropathy were randomly assigned to receive either of the two treatments for 6 weeks, followed by a washout period, and then were assigned to receive the other treatment. Plasma exchange (twice a week for 3 weeks then once a week for 3 weeks) and immune globulin infusion (0.4 gm/kg once a week for 3 weeks, then 0.2 gm/kg once a week for the next 3 weeks) were used. End points assessed before and after treatment schedules were neurological disability score; muscle weakness of the neurological disability score; summated compound muscle action potentials of ulnar, median, and peroneal nerves; summated sensory nerve action potentials of ulnar and sural nerves; and vibratory detection threshold of the great toe using CASE IV. Observers were masked as to treatment used. Of 20 patients, 13 received both treatments whereas 4 did not worsen sufficiently to receive the second treatment-1 patient left the study during and 2 after the first treatment to receive unscheduled treatment elsewhere. Of the 17 patients who received plasma exchange in either treatment period, statistically significant improvement occurred in the neurological disability score (38.3 +/- 34.6 points, mean +/- standard deviation, p < 0.001), weakness subset of the score (33.4 +/- 29.5 points, p < 0.001), and summated compound muscle action potentials (3.7 +/- 3.5 mV, p < 0.001). This improvement is large and unequivocal, being equivalent to a change from a 50% bilateral weakness of pelvic and lower limb muscles to no weakness. Of the 15 patients who received immune globulin infusion in either schedule, the comparable changes were as follows: the neurological score, 36.1 +/- 32.0 points (p = 0.006); the weakness subset of the neurological score, 31.4 +/- 31.5 points (p < 0.002); and summated muscle action potentials, 3.3 +/- 2.8 mV (p < 0.001). In a comparison of the change in neuropathic end points between the two treatments, statistically significant differences were not found for any end point. In an open trial of 8 patients, to determine the frequency and dosage of immune globulin infusion needed to maintain good function, we found that frequency could be titrated by assessing the neurological disability score and the summated muscle action potentials at 6-week intervals. The dosage and frequency required to achieve a high functional level were quite variable among patients. Both treatments are efficacious in ameliorating neurological deficit in chronic inflammatory demyelinating polyradiculoneuropathy. The effect is large, but for most patients is short-lived, requiring continued intermittent treatment for maximal or sustained improvement. For schedules of treatment that cost approximately the same, a clear difference in efficacy was not shown between plasma exchange and immune globulin infusion.Since immune globulin can be infused without expensive devices and can be given in the home, it may be the preferable treatment. The frequency and dosage of infused globulin needed to maintain improvement was quite variable (from as little as 0.025 gm/kg once every 10 days to 0.4 gm/kg [seldom more) every 2-4 days).