In the last decade, efforts have been made by the Inflammatory Neuropathy Cause And Treatment (INCAT) group to initiate a scientific approach dealing with the standardisation of outcome measures for studies in patients with polyneuropathy, particularly those with Guillain-Barré syndrome (GBS), chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), and monoclonal gammopathy of undetermined significance related polyneuropathy (MGUSP). These efforts culminated in a recent workshop, guided by the European Neuromuscular Centre (ENMC), addressing the remaining needs for further scientific evaluation to standardise outcome measures in these disorders with extension of these needs to patients with painful neuropathies and with multifocal motor neuropathy (MMN). The ENMC workshop presented a list of recommendations and a general consensus for application of a minimum core set of outcome measures was reached. Based on these recommendations, the current study aims to expand the clinimetric knowledge on outcome measures at various levels of outcome (pathology, impairment, activity & participation limitation, and quality of life) in autoimmune polyneuropathies, particularly in GBS, CIDP, MMN, MGUSP, and autoimmune small fibre neuropathies (AI-SFN). Also, the general applicability of an autonomic symptoms scale plus some selected activity limitation scales will be examined.
ContributorsIngemar Merkies
Catharina Faber
Maastricht University Medical Centre, Maastricht
PeriNomS study group: A.A. Barreira, Brazil; D. Bennett, UK; P.Y.K. Van den Bergh, Belgium; V. Bril, Canada; G. Devigili, Italy; R.D. Hadden, UK; A.F. Hahn, Canada; H-P Hartung, Germany; R.A.C. Hughes, UK; I Illa, Spain; H. Katzberg, Canada; A.J. van der Kooi, the Netherlands; J-M. Léger, France; R.A. Lewis, USA; M.P.T. Lunn, UK; O.J.M. Nascimento, Brazil; E. Nobile-Orazio, Italy; L. Padua, Italy; J. Pouget, France; M.M. Reilly, UK, I. van Schaik, the Netherlands; B. Smith, USA; M. de Visser, the Netherlands; D. Walk, USA, Pieter Antoon van Doorn, the Netherlands, David Reid Cornblath, USA, Anneke Jelly van der Kooi, the Netherlands, Leonard Hendrik van den Berg, the Netherlands, Nicolette Claudia Notermans, the Netherlands, Kenneth Craig Gorson, USA, Giuseppe Lauria, Italy, Alan Tennant, UK
Disease Category: Neurology
Disease Name: Chronic inflammatory demyelinating polyradiculoneuropathy, Guillain-Barre syndrome , Peripheral neuropathy , Multifocal motor neuropathy, Gammopathy related polyneuropathy
Age Range: 18 - 100
Sex: Either
Nature of Intervention: Any
- None
- Recommendations for outcome measures (measurement/how)
For the cross-sectional studies, which is focused on examining the various reliability and validity modalities of various outcome measures, a total of 120 patients with a polyneuropathy and a stable clinical condition were recruited from the Rotterdam, Maastricht, and Utrecht (inflammatory) neuropathy databank. Moreover, 20 patients with AI-SFN were included from the Maastricht University Medical Centre. All patients will be meeting the international criteria for their diagnosis. Within 2-4 weeks, the patient will return for a second visit.
The longitudinal studies will be performed to obtain responsiveness data that may help the researcher to differentiate between comparable valid and reliable measures that are generally being applied, particularly in immune-mediated polyneuropathies. Through comparative responsiveness the most responsive measures will be selected for each polyneuropathy form.
A total of 150 newly diagnosed patients (GBS, CIDP, MGUSP, MMN) will be recruited from the large international centers that take part in the study, to investigate the clinimetrical aspects of the various outcome measures, with emphasis on comparative responsiveness.