The objectives of this study were to provide an overview of the outcome measures (OMs) applied in clinical trials in multifocal motor neuropathy (MMN) and to determine the responsiveness of a core set of selected OMs as part of the peripheral neuropathy outcome measures standardization (PeriNomS) study. The following OMs were serially applied in 26 patients with newly diagnosed or relapsing MMN, receiving intravenous immunoglobulin (assessments: T0/T3/T12?months): 14 muscle pairs MRC (Medical Research Council) scale, the Neuropathy Impairment Scale motor-subset, a self-evaluation scale, grip strength, and MMN-RODS© (Rasch-built overall disability scale). All data, except the grip strength, were subjected to Rasch analyses before determining responsiveness. For grip strength, responsiveness was examined using a combined anchor- (SF-36 question-2) and distribution-based (½?×?SD) minimum clinically important difference (MCID) techniques, determining the proportion of patients exceeding both the identified cut-offs. For the remaining scales, the magnitude of change for each patient on each scale was determined using the MCID related to the individual SE (responder definition: MCID-SE?=?1.96). Overall, a great assortment of measures has been used in MMN trials with different responsiveness definitions. For the selected OMs, responsiveness was poor and only seen in one fourth to one third of the patients, the grip strength being more responsive. Despite the efforts taken to standardize outcome assessment, further clinimetric responsiveness studies are needed in MMN.
AimThe objectives of this study were to provide an overview of the outcome measures (OMs) applied in clinical trials in multifocal motor neuropathy (MMN) and to determine the responsiveness of a core set of selected OMs as part of the peripheral neuropathy outcome measures standardization (PeriNomS) study.
ContributorsMariëlle H. J. Pruppers1, Thomas H. P. Draak, Els K. Vanhoutte, W-Ludo Van der Pol, Kenneth C. Gorson, Jean-Marc Léger, Eduardo Nobile-Orazio, Richard. A. Lewis, Leonard H. van den Berg, Catharina G. Faber, and Ingemar S. J. Merkies, on behalf of the Peri NomS study group
Disease Category: Neurology
Disease Name: Multifocal motor neuropathy
Age Range: Unknown
Sex: Either
Nature of Intervention:
- COS uptake study
- Systematic review
The study design consisted of two parts: part one provided a literature overview of the OMs used in clinical open-labeled and randomized trials in MMN, and part two focused on determining the comparison responsiveness of a core set of selected OMs by MMN. The systematic search was performed through Medline and reference tracing (period: January 1995to January 2014