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

Gwaltney, Chad; Shields, Alan; Love, Emily; Ollis, Sarah; Stokes, Jonathan; Mazar, Iyar; Arenson, Ethan; Aiudi, Anthony; Wirth, R. J.; Houts, Carrie

doi:10.1186/s13023-025-03693-5

Orphanet Journal of Rare Diseases

Table 4 Correlations of BTHS-SA change scores with reference measure change scores

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

Measure	2 FS	3 FS	4 FS	5MS
Measure	r	r	r	r
PROMIS fatigue SF*	0.74	0.66	0.59	0.59
6MWT distance*	− 0.53	− 0.55	− 0.57	− 0.57
EQ- 5D VAS*	− 0.50	− 0.46	− 0.50	− 0.48
5XSST*	0.27	0.26	0.29	0.28
Leg strength*	− 0.57	− 0.44	− 0.43	− 0.42
SWAY*	− 0.55	− 0.59	− 0.54	− 0.47
Activity duration*	− 0.42	− 0.34	− 0.19	− 0.18
Activity intensity*	− 0.42	− 0.34	− 0.18	− 0.17
CGI-S (overall item)**	0.36	0.32	0.36	0.28
CGI-C (overall item)**	0.56	0.54	0.49	0.46
PGI-S (overall item)**	0.73	0.77	0.67	0.61
PGI-C (overall item)**	0.81	0.79	0.82	0.80
CaGI-S (overall item)**	0.65	0.64	0.58	0.58
CaGI-C (overall item)**	0.59	0.64	0.66	0.66
6MWT PRO fatigue (post)**	0.42	0.39	0.50	0.48
6MWT PRO dyspnea (post)**	0.48	0.44	0.45	0.40

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; leg strength, as measured by HHD, reflects pounds or kilograms of force exerted by an individual upon muscle contraction with lower scores indicative of less strength; 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; and each of the CGI-C, PGI-C, and CaGI-C assess perceived change from study baseline with lower scores indicative no change in health status
*Pearson correlation
**Spearman correlation

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ISSN: 1750-1172

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