What is it about?
Assessing the relative responsiveness of clinical scales of the CMT Italian and UK ascorbic acid trial (placebo arm) over two years, by using the standardized response mean (SRM), which is the ratio of the paired scores mean change over time to the standard deviation of the score change (0 is worst responsiveness). the CMT Neuropathy Score version 1 (CMTNSv1, primary outcome measure of the trial) showed low responsiveness (SRM 0.13). Myometric assessments of handgrip and foot dorsiflexion strength were the most responsive (SRM 0.31 and 0.38, respectively). Amongst the other measures, the nine-hole peg test, which assesses upper limb functioning, showed the best responsiveness (SRM 0.28).
Featured Image
Why is it important?
CMT is a very slowly progressive neuropathy which makes it difficult to detect disease progression over time and to assess intervention efficacy. This study confirms the need to improve outcome measures for CMT used for prognostic and interventional studies. The most responsive measures were handgrip and foot dorsiflexion myometry, which are likely to be clinically relevant for patients.
Perspectives
Rigorously planned and conducted clinical trials provide important data. The CMT Italian and UK ascorbic acid trial offered the opportunity to prospectively assess the relative responsiveness of its primary and secondary clinical outcome measures using distribution-based method. A further improvement is the combination of distribution- and anchor-based methods, particularly for anchors obtained from patients.
Dr. Alessandra Solari
Fondazione IRCCS Istituto Neurologico C. Besta
Read the Original
This page is a summary of: Responsiveness of clinical outcome measures in Charcot−Marie−Tooth disease, European Journal of Neurology, July 2015, Wiley,
DOI: 10.1111/ene.12783.
You can read the full text:
Contributors
The following have contributed to this page
