Vanhoutte Els K, Faber Catharina G, van Nes Sonja I, Cats Elisabeth A, Van der Pol W-Ludo, Gorson Kenneth C, van Doorn Pieter A, Cornblath David R, van den Berg Leonard H, Merkies Ingemar S J
Department of Neurology, University Medical Centre Maastricht, Maastricht, The Netherlands.
Department of Neurology, Erasmus Medical Centre Rotterdam, Rotterdam, The Netherlands.
J Peripher Nerv Syst. 2015 Sep;20(3):296-305. doi: 10.1111/jns.12141.
Clinical trials in multifocal motor neuropathy (MMN) have often used ordinal-based measures that may not accurately capture changes. We aimed to construct a disability interval outcome measure specifically for MMN using the Rasch model and to examine its clinimetric properties. A total of 146 preliminary activity and participation items were assessed twice (reliability studies) in 96 clinically stable MMN patients. These patients also assessed the ordinal-based overall disability sum score (construct, sample-dependent validity). The final Rasch-built overall disability scale for MMN (MMN-RODS(©) ) was serially applied in 26 patients with newly diagnosed or relapsing MMN, treated with intravenous immunoglobulin (IVIg) (1-year follow-up; responsiveness study). The magnitude of change for each patient was calculated using the minimum clinically important difference technique related to the individually obtained standard errors. A total of 121 items not fulfilling Rasch requirements were removed. The final 25-item MMN-RODS(©) fulfilled all Rasch model's expectations and showed acceptable reliability and validity including good discriminatory capacity. Most serially examined patients improved, but its magnitude was low, reflecting poor responsiveness. The constructed MMN-RODS(©) is a disease-specific, interval measure to detect activity limitations in patients with MMN and overcomes the shortcomings of ordinal scales. However, future clinimetric studies are needed to improve the MMN-RODS(©) 's responsiveness by longer observations and/or more rigorous treatment regimens.
多灶性运动神经病(MMN)的临床试验通常使用基于序数的测量方法,这些方法可能无法准确反映变化情况。我们旨在使用拉施模型构建一种专门针对MMN的残疾区间结局测量方法,并检验其测量学特性。对96例临床稳定的MMN患者的146项初步活动和参与项目进行了两次评估(可靠性研究)。这些患者还评估了基于序数的总体残疾总分(结构效度、样本依赖效度)。针对新诊断或复发的MMN患者,对最终通过拉施模型构建的MMN总体残疾量表(MMN-RODS(©))进行了连续应用,这些患者接受静脉注射免疫球蛋白(IVIg)治疗(1年随访;反应度研究)。使用与个体获得的标准误差相关的最小临床重要差异技术计算每位患者的变化幅度。总共删除了121项不符合拉施要求的项目。最终的25项MMN-RODS(©)满足了拉施模型的所有期望,并显示出可接受的信度和效度,包括良好的区分能力。大多数接受连续检查的患者有所改善,但其改善幅度较小,反映出反应度较差。构建的MMN-RODS(©)是一种针对疾病的区间测量方法,用于检测MMN患者的活动受限情况,克服了序数量表的缺点。然而,未来需要进行测量学研究,通过更长时间的观察和/或更严格的治疗方案来提高MMN-RODS(©)的反应度。