Table 2 Patient characteristics for the ERT group at study entry or start of ERT and for untreated patients at study entry*
ERT group (n = 204) | Non-ERT group (n = 79) | P Value† | ||
|---|---|---|---|---|
Characteristics | At study entry | At start of ERT | At study entry | |
Female, n (%) | 104 (51) | 104 (51) | 45 (57) | 0.37 |
Median age at study entry/start of ERT, years (range) | 47 (19–73) | 51 (24–76) | 51 (20–81) | 0.48 |
Median age at diagnosis, years (range) | 38 (1–72) | 38 (1–72) | 42 (2–67) | 0.45 |
Median disease duration at study entry/start of ERT, years (range) | 7 (0–31) | 11 (0.2-33) | 12 (0–32) | 0.75 |
Country of residence, n (%) | 0.05 | |||
Netherlands | 86 (42) | 86 (42) | 23 (29) | |
United Kingdom | 18 (9) | 18 (9) | 5 (6) | |
United States | 44 (22) | 44 (22) | 27 (34) | |
Germany | 37 (18) | 37 (18) | 11 (14) | |
Other‡ | 19 (9) | 19 (9) | 13 (17) | |
Disease severity at study entry/start of ERT, n (%) | 0.45 | |||
No wheelchair use or respiratory support§ | 99 (49) | 70 (34) | 35 (44) | |
Wheelchair use | 26 (13) | 37(18) | 11 (14) | |
Use of respiratory support | 31(15) | 29 (14) | 11 (14) | |
Both wheelchair use and respiratory support | 48 (24) | 68 (33) | 22 (28) | |
Median follow-up time from study entry/start of ERT, years (range) | 7 (1–9) | 4 (0.2-8) | 4 (0.04-9) | 0.05 |
Died during follow-up, n (%) | 18 (9) | 18 (9) | 28 (35) | <0.001 |