Iancu Ferfoglia Ruxandra, Guimarães-Costa Raquel, Viala Karine, Musset Lucile, Neil Jean, Marin Benoit, Léger Jean-Marc
Department of Neurology, National Referral Center for Rare Neuromuscular Diseases, University Hospital Pitié-Salpêtrière and University Paris VI, Paris, France.
Department of Neurology, Geneva University Hospitals, Geneva, Switzerland.
J Peripher Nerv Syst. 2016 Mar;21(1):10-4. doi: 10.1111/jns.12156.
The Rituximab vs. Placebo in Polyneuropathy Associated With Anti-MAG IgM Monoclonal Gammopathy (RIMAG) study showed no improvement using the inflammatory neuropathy cause and treatment sensory score (ISS) as primary outcome in patients with IgM anti-myelin-associated glycoprotein neuropathy (IgM anti-MAG neuropathy) treated with rituximab, when compared with placebo. However, some secondary outcomes seemed to improve in the per protocol analysis. Patients from one participating center in the RIMAG study underwent a new evaluation after a median follow-up of 6 (interquartile range (IQR) 4.9; 6.5) years, using the same outcome measures used in the original study. Data were recorded in seven rituximab patients (group 1) and in eight placebo patients (group 2). In group 2, six of eight patients received immunotherapy during follow-up, while only two of seven did in group 1. No significant change was observed in either the ISS or the secondary outcomes in both groups, with the exception of worsening in the 10-m walk time in group 2 (p = 0.016). The RIMAG follow-up study failed to find any significant change in most outcome measures in patients from the RIMAG study, some of them having received new immunotherapies. This study stresses the lack of useful clinical scales sensitive enough to capture small, even meaningful, improvement in IgM anti-MAG neuropathy.
利妥昔单抗对比安慰剂治疗抗MAG IgM单克隆丙种球蛋白病相关性多发性神经病（RIMAG）研究显示，与安慰剂相比，接受利妥昔单抗治疗的IgM抗髓鞘相关糖蛋白神经病（IgM抗MAG神经病）患者，以炎症性神经病病因及治疗感觉评分（ISS）作为主要结局指标，未出现改善。然而，在意向性分析中一些次要结局似乎有所改善。RIMAG研究中一个参与中心的患者在中位随访6（四分位间距（IQR）4.9；6.5）年后，使用与原研究相同的结局指标进行了重新评估。记录了7名利妥昔单抗治疗患者（第1组）和8名安慰剂治疗患者（第2组）的数据。在第2组中，8名患者中有6名在随访期间接受了免疫治疗，而第1组7名患者中只有2名接受了免疫治疗。两组的ISS或次要结局均未观察到显著变化，但第2组的10米步行时间恶化除外（p = 0.016）。RIMAG随访研究未能在RIMAG研究患者的大多数结局指标中发现任何显著变化，其中一些患者接受了新的免疫治疗。这项研究强调缺乏足够敏感的有用临床量表来捕捉IgM抗MAG神经病中微小甚至有意义的改善。
