Thursday, August 3, 2023

The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic literature review

Poleur, M., Markati, T. & Servais, L. The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic literature review. Orphanet J Rare Dis 18, 224 (2023). doi:10.1186/s13023-023-02813-3 

 We found two studies of patients with FRDA that used inertial technology. Remote monitoring of several voice parameters, upper limb function through 14 parameters that can be grouped into parameters related to movement velocity, spectral frequency, and parameters related to deviation of the ideal trajectory, and 15 spatiotemporal gait parameters was feasible over 1 week. The sensitivity of an upper limb composite score, the AIM-S, obtained through sensors contained in a spoon designed to detect deterioration in upper limb function, was greater than other measures.