Outcome Measures in inflammatory peripheral neuropathies (PeriNoms)

Location: Naarden, The Netherlands

Co-sponsored by the Dutch ZonMw Association

This workshop took place from February 8th -10th, 2013 in Naarden, The Netherlands. There were 20 researchers from 8 different countries (USA, Canada, Spain, Italy, France, Belgium, the Netherlands, UK), including a patient representative of the GBS CIDP Foundation International (Glennys Sanders). The workshop was conducted under the leadership of Ingemar S.J. Merkies and Catharina G. Faber.

There has been inadequate agreement about outcome measures (the instruments used to measure treatment response) for use in clinical trials of treatments in inflammatory neuropathies (Guillain-Barré syndrome, chronic inflammatory demyelinating polyradiculoneuropathy, monoclonal gammopathy-related neuropathy and multifocal motor neuropathy). In 2007 the international Peripheral Neuropathy Outcome Measures Standardisation (PeriNomS) Study was started, with the aim to define a minimum core set of specific outcome measures for each inflammatory neuropathy.  The workshop heard the results of new ordinal scales developed in the PeriNomS Study for measuring strength, sensation, activity and participation.

The workshop participants agreed

• That trials should have outcome measures which address each of strength, sensation, activities, participation and   quality of life.
• That outcome measures should fulfil the most stringent biometric standards which will often involve switching from historical ordinal scales to interval scales including those now developed and validated in the PeriNomS Study.
• A minimum core set of outcome measures for GBS, CIDP and MMN, which differed between each of the diseases.
• A recommended set of outcome measures for MGUSP that requires further exploration.

The workshop identified gaps in existing knowledge and proposed topics for future research.

Participants:

Prof. P. Van den Bergh, Prof. V. Bril, Prof. P. van Doorn, Dr. C.G. Faber, Prof. K. Gorson, Prof. A. Hahn, Prof. R. Hughes, Dr. A. van der Kooi, Dr. G. Lauria, Prof. J-M Leger, Prof. R. Lewis, Dr. M. Lunn, Dr. I.S.J. Merkies, Prof. E. Nobile-Orazio, Dr. N.C. Notermans, Dr. L. Querol, Mrs G. Sanders MBE, Prof. I. van Schaik, Drs. E. Vanhoutte.

A full report has been published in Neuromuscular Disorders.
(For a copy of the report, click here.)