Table 3 Characteristics of individual response groups with regard to skeletal muscle strength and pulmonary function
Non-responders | Moderate Responders | Good responders | P-value | |
|---|---|---|---|---|
Response groups for MRC sumscore – No. of patients (%) | 6 (9) | 32 (46) | 31 (45) | |
Female – No. of patients (%) | 1 (17) | 11 (34) | 21 (68) | 0.002 |
Age at start of ERT in years – median (range) | 51.6 (38.0 – 66.2) | 50.0 (26.2 – 67.0) | 52.7 (29.2 – 76.3) | 0.47 |
Disease duration at start of ERT in years – median (range) | 12.1 (3.6 – 22.7) | 11.1 (0.9 – 31.2) | 9.0 (0.2 – 29.1) | 0.55 |
MRC sumscore at start of ERT in percentage – median (range) | 80.8 (60.8 – 86.9) | 80.0 (48.3 – 91.5) | 74.6 (53.3 – 92.3) | 0.12 |
Wheelchair use at start of ERT – No. of patients (%) | 2 (33) | 9 (28) | 16 (52) | 0.11 |
FVC in upright position at start of ERT in percentage – median (range) | 61.5 (34.4 – 79.3) | 69.9 (11.3 – 105.0) | 69.3 (16.4 – 106.9) | 0.48 |
Ventilation use at start of ERT – No. of patients (%) | 1 (17) | 12 (38) | 12 (39) | 0.49 |
Response groups for FVC in upright position – No. of patients (%) | 9 (15) | 26 (42) | 27 (44) | |
Female – No. of patients (%) | 5 (56%) | 14 (54%) | 13 (48%) | 0.64 |
Age at start of ERT in years – median (range) | 52.6 (31.9 – 69.2) | 51.2 (35.7 – 71.7) | 43.3 (26.2 – 76.3) | 0.16 |
Disease duration at start of ERT in years – median (range) | 8.3 (0.6 – 31.2) | 14.1 (0.9 – 27.0) | 4.7 (0.2 – 29.1) | 0.06 |
MRC sumscore at start of ERT in percentage – median (range) | 74.6 (63.1 – 91.5) | 77.7 (48.3 – 90.0) | 80.8 (67.5 – 92.3) | 0.26 |
Wheelchair use at start of ERT – No. of patients (%) | 4 (44%) | 10 (38%) | 6 (22%) | 0.15 |
FVC in upright position at start of ERT in percentage – median (range) | 66.6 (51.4 – 100.5) | 68.3 (11.3 – 106.9) | 69.9 (16.4 – 105.0) | 0.95 |
Ventilation use at start of ERT – No. of patients (%) | 2 (22) | 8 (31) | 8 (30) | 0.76 |
Response groups for FVC in supine position – No. of patients (%) | 21 (39) | 17 (31) | 16 (30) | |
Female – No. of patients (%) | 10 (48) | 10 (59) | 10 (63) | 0.36 |
Age at start of ERT in years – median (range) | 51.7 (31.9 – 67.0) | 47.9 (26.2 – 62.9) | 40.4 (29.2 – 74.0) | 0.007 |
Disease duration at start of ERT in years – median (range) | 10.7 (0.6 – 31.2) | 4.7 (0.5-21.0) | 5.0 (0.9 – 29.1) | 0.12 |
MRC sumscore at start of ERT in percentage – median (range) | 76.9 (60.8 – 91.5) | 80.8 (69.2 – 90.8) | 81.9 (69.2 – 92.3) | 0.02 |
Wheelchair use at start of ERT – No. of patients (%) | 8 (38) | 2 (12) | 2 (13) | 0.05 |
FVC in upright position at start of ERT in percentage – median (range) | 66.6 (45.4 – 106.9) | 65.7 (41.5 – 105.8) | 81.0 (62.6 – 105.0) | 0.02 |
Ventilation use at start of ERT – No. of patients (%) | 4 (19) | 6 (35) | 0 (0) | 0.01 |