Table 1.
Patients’ characteristics and baseline values of clinical outcome measures at start of ERT
| Characteristic | Total study population (N = 69) | Patients with pre-ERT and ERT follow-up (N = 49) | P-value* |
|---|---|---|---|
| Age at start of ERT in years – median (range) |
52.1 (26.2 – 76.3) |
50.1 (26.2 – 74.0) |
0.83 |
| Age at onset of symptoms in years – median (range) |
30.8 (1.4 – 62.0) |
32.0 (1.4 – 62.0) |
0.63 |
| Age at diagnosis in years – median (range) |
39.6 (1.4 – 63.8) |
40.9 (1.4 – 63.0) |
0.97 |
| Duration of disease at start of ERT in years – median (range) |
9.3 (0.2 – 31.2) |
8.3 (0.5 – 31.2) |
0.74 |
| MRC sumscore at start of ERT in percentage – median (range) |
77.7 (48.3 – 92.3) |
79.2 (60.8 – 92.3) |
0.52 |
| HHD sumscore at start of ERT in percentage – median (range) |
69.3 (25.9 – 94.1) |
69.3 (25.9 – 94.1) |
0.97 |
| QMFT score at start of ERT in percentage – median (range) |
55.6 (10.0 – 89.1) |
59.5 (21.7 – 89.1) |
0.42 |
| Time of mechanical ventilation at start of ERT per day in hours – median (range) |
11.5 (0.0 –24.0) |
10.5 (0.0 –24.0) |
0.81 |
| FVC in upright position at start of ERT in percentage – median (range) |
68.3 (11.3 – 106.9) |
71.9 (26.5 – 106.9) |
0.43 |
| FVC in supine position at start of ERT in percentage – median (range) |
46.8 (23.0 – 99.0) |
52.4 (23.0 – 99.0) |
0.71 |
| Gender – No. of patients (%) |
|
|
0.43 |
| - Male |
36 (52) |
21 (43) |
|
| - Female |
33 (48) |
28 (57) |
|
| Wheelchair at start of ERT – No. of patients (%) |
|
|
|
| - No wheelchair use |
42 (61) |
33 (67) |
0.22 |
| - Partial wheelchair use |
8 (12) |
8 (16) |
|
| - Permanent wheelchair use |
19 (28) |
8 (16) |
|
| Mechanical ventilation at start of ERT – No. of patients (%) |
|
|
|
| - No mechanical ventilation |
44 (64) |
36 (74) |
0.72 |
| - Non-invasive mechanical ventilation |
19 (28) |
10 (20) |
|
| - Invasive mechanical ventilation | 6 (9) | 3 (6) |
ERT = Enzyme Replacement Therapy; MRC sumscore = Medical Research Council sumscore; HHD sumscore = Hand-Held Dynamometry sumscore; QMFT score = Quick Motor Function Test score; FVC = Forced Vital Capacity; N/No. = Number of patients.
* Differences between the total group (N = 69) and the group with pre-ERT and ERT follow-up (N = 49) were calculated using chi-square tests for the categorical variables, and using Mann–Whitney tests for continuous variables.