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) |