Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study

Table 3 Correlations for BTHS-SA scores and reference measures averaged across timepoints

To facilitate interpretation, each of the 2 FS (range = 2–10), 3 FS (range = 3–15), 4 FS (range = 4–20), and 5MS (range = 5–25) are scored such that higher scores reflect poorer health status; PROMIS Fatigue SF (range = 0–52) is scored such that lower scores reflect poorer health status (i.e., more fatigue); 6MWT, a measure of distance walked, reflects scores in meters/feet (e.g., the distance a healthy individual can walk on a hard, flat surface in six minutes is approximately 400–700 m (or approximately 1300–2300 feet) [30] with lower scores indicating worse level of physical activity; EQ- 5D VAS (range = 0–100) is scored such that lower scores reflect poorer health status; the 5XSST measures the time it takes an individual to stand up and sit down five times in a row with higher times indicative of poorer health status; the SWAY quantifies postural sway from 0 to 100 with lower scores indicative of greater instability/poorer health status; Activity Duration and Activity Intensity, as assessed by accelerometry data collected from the AVIVO™ Mobile Patient Management System, reflects patient activity/mobility with lower scores indicative of poorer health; each of the CGI-S, PGI-S, CaGI-S, 6MWT PRO fatigue, and 6MWT PRO dyspnea measure concept severity with higher scores indicative of poorer health status;
*Pearson correlations
**Spearman correlations

ISSN: 1750-1172