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Table 1 BTHS-SA Weekly item and domain scores

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

Scale/item

Treatment period 1

Pre-dose

Nurse visit 2

End of Tx

N

M (SD)

N

M (SD)

N

M (SD)

2 FS (range = 2–10)

12

5.42 (0.98)

12

4.11 (1.21)

12

4.22 (1.04)

3 FS (range = 3–15)

12

8.0 (1.35)

12

6.09 (1.95)

12

6.26 (1.69)

4 FS (range = 4–20)

12

9.96 (1.65)

12

7.60 (2.44)

12

7.96 (2.14)

5MS (range = 5–25)

12

11.84 (1.76)

12

9.27 (2.61)

12

9.85 (2.64)

Item 1: tiredness at rest (range = 1–5)

12

2.58 (0.84)

12

1.98 (0.85)

12

2.04 (0.83)

Item 2: tiredness during activity (range = 1–5)

12

2.85 (0.64)

12

2.17 (0.81)

12

2.25 (0.58)

Item 3: muscle weakness at rest (range = 1–5)

12

1.96 (0.48)

12

1.52 (0.51)

12

1.70 (0.62)

Item 4: muscle weakness during activity (range = 1–5)

12

2.57 (0.49)

12

1.94 (0.51)

12

1.97 (0.59)

Item 5: muscle pain at rest (range = 1–5)

12

1.67 (0.52)

12

1.47 (0.59)

12

1.69 (0.63)

Scale/item

Treatment period 2

Pre-dose

Nurse visit 2

End of Tx

N

M (SD)

N

M (SD)

N

M (SD)

2 FS (range = 2–10)

12

4.73 (1.09)

11

4.39 (1.24)

11

4.20 (1.29)

3 FS (range = 3–15)

12

7.10 (1.85)

11

6.65 (1.85)

11

6.28 (2.02)

4 FS (range = 4–20)

12

8.94 (2.21)

11

8.26 (2.24)

11

7.78 (2.35)

5MS (range = 5–25)

12

11.20 (2.66)

11

10.13 (2.71)

11

9.54 (3.03)

Item 1: tiredness at rest (range = 1–5)

12

2.38 (0.96)

11

2.26 (0.94)

11

2.08 (0.99)

Item 2: tiredness during activity (range = 1–5)

12

2.57 (0.83)

11

2.47 (0.99)

11

2.23 (0.90)

Item 3: muscle weakness at rest (range = 1–5)

12

1.83 (0.64)

11

1.61 (0.69)

11

1.50 (0.48)

Item 4: muscle weakness during activity (range = 1–5)

12

2.15 (0.52)

11

1.92 (0.58)

11

1.97 (0.70)

Item 5: muscle pain at rest (range = 1 to 5)

12

1.94 (0.74)

11

1.61 (0.67)

11

1.50 (0.58)

  1. 2 FS = 2 item fatigue during activities score; 3 FS = 3 item fatigue score; 4 FS = 4 item fatigue score; 5MS = 5 item myopathy score