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 |
* Continuous variables are expressed as median (range). Categorical variables are expressed as n (%). ERT denotes enzyme replacement therapy.
† P value for differences between ever-treated patients at the start of ERT and never-treated patients (at study entry) as assessed with a Mann–Whitney test or the χ2 test.
‡ Including patients from Australia and Canada.
§ Respiratory support includes partial and full-time invasive and non-invasive respiratory support.