Outcome measures in MMN revisited: further improvement needed
Por:
Pruppers, MHJ, Draak, THP, Vanhoutte, EK, Van der Pol, WL, Gorson, KC, Leger, JM, Nobile-Orazio, E, Lewis, RA, van den Berg, LH, Faber, CG, Merkies, ISJ, Illa I., Querol, L., PeriNomS Study Grp
Publicada:
1 sep 2015
Resumen:
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/T12months): 14 muscle pairs MRC (Medical Research Council) scale, the Neuropathy Impairment Scale motor-subset, a self-evaluation scale, grip strength, and MMN-RODS(C) (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 (1/2xSD) 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-SE1.96). 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.
Filiaciones:
Pruppers, MHJ:
Maastricht Univ, Dept Neurol, Med Ctr, Maastricht, Netherlands
Draak, THP:
Maastricht Univ, Dept Neurol, Med Ctr, Maastricht, Netherlands
Vanhoutte, EK:
Maastricht Univ, Dept Clin Genet, Med Ctr, Maastricht, Netherlands
Van der Pol, WL:
Univ Med Ctr, Dept Neurol, Rudolf Magnus Inst Neurosci, Utrecht, Netherlands
Gorson, KC:
Tufts Univ, Sch Med, St Elizabeths Med Ctr, Dept Neurol, Boston, MA 02111 USA
Leger, JM:
Hop La Pitie Salpetriere, Dept Neurol, Paris, France
Nobile-Orazio, E:
Univ Milan, Dept Neurol Sci, Humanitas Clin Inst, Milan, Italy
Lewis, RA:
Cedars Sinai Med Ctr, Dept Neurol, Los Angeles, CA 90048 USA
van den Berg, LH:
Univ Med Ctr, Dept Neurol, Rudolf Magnus Inst Neurosci, Utrecht, Netherlands
Faber, CG:
Maastricht Univ, Dept Neurol, Med Ctr, Maastricht, Netherlands
Merkies, ISJ:
Maastricht Univ, Dept Neurol, Med Ctr, Maastricht, Netherlands
Spaarne Hosp, Dept Neurol, NL-2134 TM Hoofddorp, Netherlands
Illa I.:
Institut d’Investigació Biomèdica Sant Pau (IIB SANT PAU), Sant Quintí 77-79, 08041 Barcelona, Spain
Querol, L.:
Institut d’Investigació Biomèdica Sant Pau (IIB SANT PAU), Sant Quintí 77-79, 08041 Barcelona, Spain
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