Background:
Despite recent developments of new candidate therapies, the FSHD field remains limited in meaningful and appropriate clinical outcome assessments. Many of the existing measures were developed long ago and not specific to the FSHD population. We recently developed an optimized Timed Up and Go (TUG) test that tests truncal and lower limb impairments. Here, we examined its responsiveness in short-term follow-up.
Objective: To examine the early responsiveness of an FSHD oTUG to disease progression across the disability range.
Approach:
22 FSHD patients categorized by Ricci’s clinical severity scores (CSS 1-1.5: 6; CSS 2-2.5: 4; CSS 3-3.5:6 and CSS 4:6) were recruited. Each group performed the TUG and oTUG on two separate visits (and twice at the first visit for reliability) within 6 to 12 months apart.
Results:
Our cohort mainly consisted of white males between the ages of 19-55 (42+10). Within our cohort, the oTUG (ICC = 0.972; p < 0.000) displayed similar reliability to the standard TUG (ICC = 0.995; p< 000), and a slightly higher correlation to CSS (ρ = 0.635: p = 0.037 vs. ρ = 0.564: p = 0.006). At baseline (time 1) the average times for TUG and oTUG were 12.69 + 7.4 and 19.87 + 9.9, respectively. Six to twelve months later, average times for TUG and oTUG in the same cohort were 11.30 + 6.27 and 18.43 + 10.11, respectfully. The mean (SD) change was 0.60 + 1.5 for the TUG and 0.68 + 3.0 for the oTUG between the two visits. This trend was fairly consistent between subgroups when broken down by CSS for TUG and oTUG.
Conclusion:
Our results indicate that the oTUG for FSHD displays excellent test re-test reliability, with better correlation to clinical impairment than the TUG. There is no indication of significant change in either the TUG or the oTUG over the 6-12 months of follow up period.