Table 1.
Demographics and baseline characteristics (OLE-safety population)
| Cipa + mig group (N = 81) |
Switch group (N = 37) |
Total (N = 118) |
|
|---|---|---|---|
| Age at informed consent date, years | |||
| Mean (SD) | 48.9 (13.5) | 46.0 (13.5) | 48.0 (13.5) |
| Median (range) | 49.0 (20–75) | 47.0 (23–67) | 49.0 (20–75) |
| Age at diagnosis, years | |||
| Mean (SD) | 40.3 (13.8) | 37.2 (15.4) | 39.3 (14.4) |
| Median (range) | 40.0 (1–66) | 40.0 (7–63) | 40.0 (1–66) |
| Gender, n (%) | |||
| Male | 33 (40.7) | 19 (51.4) | 52 (44.1) |
| Female | 48 (59.3) | 18 (48.6) | 66 (55.9) |
| Race, n (%)a | |||
| Asian | 3 (3.7) | 1 (2.7) | 4 (3.4) |
| Japanese | 2 (2.5) | 4 (10.8) | 6 (5.1) |
| Black/African American | 0 (0.0) | 1 (2.7) | 1 (0.8) |
| White | 71 (87.7) | 30 (81.1) | 101 (85.6) |
| Other | 5 (6.2) | 1 (2.7) | 6 (5.1) |
| Region, n (%) | |||
| North/South America | 24 (29.6) | 14 (37.8) | 38 (32.2) |
| Europe | 42 (51.9) | 12 (32.4) | 54 (45.8) |
| Asia Pacific | 15 (18.5) | 11 (29.7) | 26 (22.0) |
| Height, cm | |||
| Mean (SD) | 171.2 (9.7) | 171.2 (11.3)b | 171.2 (10.2)c |
| Weight, kg | |||
| Mean (SD) | 73.3 (15.3) | 78.9 (26.8) | 75.1 (19.7) |
| ERT status at entry into PROPEL, n (%) | |||
| Naïve | 20 (24.7) | 8 (21.6) | 28 (23.7) |
| Experienced | 61 (75.3) | 29 (78.4) | 90 (76.3) |
| ERT duration prior to PROPEL,d years | |||
| Mean (SD) | 7.5 (3.4) | 7.0 (3.7) | 7.3 (3.5) |
| Median (range) | 7.6 (2–14) | 7.1 (2–13) | 7.4 (2–14) |
Cipa + mig cipaglucosidase alfa + miglustat; ERT enzyme replacement therapy; OLE open-label extension; SD standard deviation
aPatients could choose more than one category
bn = 36
cn = 117
dERT-experienced patients only