To consider the most appropriate outcome measures for trials of treatment in peripheral neuropathy. The group sought to identify available outcome measures for different types of peripheral neuropathy at each level of outcome, ‘pathology’, especially of skin biopsies, ‘symptoms including pain and fatigue’, other ‘impairments’ including neurological deficit and electrophysiological measures, ‘disability’, ‘handicap’, and ‘quality of life’
ContributorsMerkies, I. S. J. Lauria, G.
Disease Category: Neurology
Disease Name: Peripheral neuropathy
Age Range: 18 - 100
Sex: Either
Nature of Intervention: Not specified
- Clinical experts
- Patient/ support group representatives
- Pharmaceutical industry representatives
- COS for clinical trials or clinical research
- Literature review
- Semi structured discussion
A Medline search was conducted for the last two decades focusing on outcome measures applied in clinical trials that included patients with a peripheral neuropathy.
Experts in diabetic neuropathy provided additional information regarding potential outcome measures. The applied outcome measures in these studies were categorised according to the international guidelines.The reported scientific soundness (simplicity, validity, reliability, and responsiveness) of identified outcome measures was also considered. Finally, an attempt was made to reach consensus on the most appropriate measures for use in clinical trials in conditions of interest to the European neuromuscular centre (ENMC) such as inflammatory neuropathies, painful neuropathies, and hereditary neuropathy. Diabetic neuropathy was used for comparison since it has been the subject of more work on outcome measures.
In advance of the workshop, a list of outcome measures applied in treatment trials was prepared including their scientific soundness, WHO and quality of life classification. Most participants took part in pre-workshop subgroups and prepared pre-workshop papers focused on controversial areas as a common background for discussion at the workshop.