Skip to main content

Table 1 Summary of outcome measurements collected in PROPEL

From: Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

Outcome

Baseline

Week 12

Week 26

Week 38

Week 52

Δ baseline and week 52

PROMIS Physical Function short form 20a score

      

 N

121

117

121

121

121

121

 mean, SD

67 (12)

69 (14)

69 (14)

69 (14)

69 (14)

1 (9)

 median, range

67 (37–97)

70 (37–98)

69 (32–99)

70 (36–99)

68 (36–99)

0 (-30–30)

6-minute walk distance in meters

      

 N

122

122

122

122

122

122

 mean, SD

356 (114)

367 (119)

370 (123)

370 (122)

372 (127)

17 (42)

 median, range

363 (79–623)

374 (101–619)

378 (40–678)

376 (77–634)

378 (67–649)

11 (-60–174)

FVC % predicted

      

 N

122

121

120

120

121

121

 mean, SD

70 (20)

70 (21)

69 (20)

69 (20)

69 (20)

-2 (6)

 median, range

70 (31–133)

69 (32–155)

69 (32–148)

68 (31–138)

69 (28–137)

-1.5 (-20–14)

MMT of lower extremities score

      

 N

118

113

112

114

114

114

 mean, SD

28 (6)

29 (6)

29 (6)

29 (6)

29 (6)

1 (3)

 median, range

28 (14–40)

28 (13–40)

29 (13–40)

29 (12–40)

30 (10–40)

1 (-10–18)

GSGC score

      

 N

106

100

100

100

102

102

 mean, SD

15 (5)

14 (5)

14 (5)

14 (5)

14 (5)

0 (2)

 median, range

16 (4–24)

16 (4–23)

16 (4–24)

15 (4–24)

16 (4–24)

0 (-8–5)

R-PAct score

      

 N

102

102

102

102

102

102

 mean, SD

20 (6)

21 (7)

21 (6)

20 (7)

21 (7)

0 (3)

 median, range

20 (10–36)

21 (8–36)

19 (8–36)

19 (9–36)

19 (8–36)

0 (-9–11)

  1. Δ: difference; FVC: forced vital capacity; GSGC: Gait, Stairs, Gowers’ maneuver, Chair; MMT: manual muscle test; N: number of patients; R-PAct: Rasch-built Pompe-specific Activity scale; SD: standard deviation