Table 4:
Demographics and clinical characteristics for all participants at their first study visit stratified by survival
| Overall | Survivors | Deceased | *SMD | |
|---|---|---|---|---|
| n | N=440 | N=395 | N=45 | |
| Participant .age at visit (mean (SD)) | 10.67 (5.74) | 10.02 (5.39) | 16.40 (5.56) | 1.164 |
| Angiotensin converting enzyme | ||||
| inhibitor or angiotensin-receptor blocker = Yes/Prophylactic (%) | 50 (11.4) | 38 (9.6) | 12 (26.7) | 0.454 |
| Diuretics use = No (%) | 440 (100.0) | 395 (100.0) | 45 (100.0) | <0.001 |
| Cardiac inotropic agents use = To treat signs and/or symptoms (%) | 8 (1.8) | 5 (1.3) | 3 (6.7) | 0.279 |
| Anti-arrhythmic use = To treat signs and/or symptoms (%) | 1 (0.2) | 0 (0.0) | 1 (2.2) | 0.213 |
| Beta-blockers use = Yes/Prophylactic (%) | 14 (3.2) | 6 (1.5) | 8 (17.8) | 0.573 |
| LVEF/SF (%) | 0.503 | |||
| LVEF % >= 55, SF % >=26 | 397 (90.2) | 363 (91.9) | 34 (75.6) | |
| LVEF % 45–54, SF % 20–25 | 30 (6.8) | 24 (6.1) | 6 (13.3) | |
| LVEF % 35–44, SF % 15–19 | 10 (2.3) | 8 (2.0) | 2 (4.4) | |
| LVEF % <= 35 %, SF % <=14 | 3 (0.7) | 0 (0.0) | 3 (6.7) | |
| Currently on steroid = Yes (%) | 260 (59.1) | 240 (60.8) | 20 (44.4) | 0.331 |
| Currently taking steroid drug = Prednisone (%) | 98 (22.3) | 92 (23.3) | 6 (13.3) | 0.26 |
| Functional tests - lower extremity (Vignos score) (%) | 1.579 | |||
| 1 | 248 (56.4) | 246 (62.3) | 2 (4.4) | |
| 2–7 | 33 (7.5) | 28 (7.1) | 5 (11.1) | |
| 8 | 158 (35.9) | 121 (30.6) | 37 (82.2) | |
| 9 | 1 (0.2) | 0 (0.0) | 1 (2.2) | |
| Functional tests - upper extremity (Brooke score) (%) | 1.151 | |||
| 1 | 265 (60.2) | 257 (65.1) | 8 (17.8) | |
| 2 | 51 (11.6) | 45 (11.4) | 6 (13.3) | |
| 3–4 | 43 (9.8) | 34 (8.6) | 9 (20.0) | |
| 5–6 | 81 (18.4) | 59 (14.9) | 22 (48.9) | |
| Ambulatory status = Nonambulatory (%) | 148 (33.6) | 113 (28.6) | 35 (77.8) | 1.132 |
| Formula/caloric supplements = As needed (%) | 15 (3.4) | 13 (3.3) | 2 (4.4) | 0.06 |
| gastrostomy tube = Daily (%) | 10 (2.3) | 6 (1.5) | 4 (8.9) | 0.336 |
| Calculated FVC % Predicted value | (%) | 1.065 | ||
| Not specified | 268 (60.9) | 258 (65.3) | 10 (22.2) | |
| 61–75% | 62 (14.1) | 56 (14.2) | 6 (13.3) | |
| 51–60% | 26 (5.9) | 20 (5.1) | 6 (13.3) | |
| 31–50% | 36 (8.2) | 27 (6.8) | 9 (20.0) | |
| <=30% | 48 (10.9) | 34 (8.6) | 14 (31.1) | |
| Ventilatory assistance used = Yes (%) | 36 (8.2) | 25 (6.3) | 11 (24.4) | <0.001 |
| Required full-time ventilatory assistance = Yes (%) | 7 (1.6) | 5 (1.3) | 2 (4.4) | 0.192 |
| Bi-PAP / Mask use (%) | 0.265 | |||
| Not specified | 420 (95.5) | 379 (95.9) | 41 (91.1) | |
| Occasionally or with illness | 2 (0.5) | 2 (0.5) | 0 (0.0) | |
| Nocturnal | 17 (3.9) | 13 (3.3) | 4 (8.9) | |
| Full-time | 7 (1.6) | 1 (0.3) | 0 (0.0) | |
| BiPAP / Nasal pillows use = No (%) | 0.088 | |||
| Not specified | 432 (98.2) | 388 (98.2) | 44 (97.8) | |
| Occasionally or with illness | 1 (0.2) | 1 (0.3) | 0 (0.0) | |
| Nocturnal | 7 (1.6) | 6 (1.5) | 1 (2.2) | |
| CPAP use = Occasionally or with illness (%) | 3 (0.7) | 1 (0.3) | 2 (4.4) | 0.279 |
| Mouthpiece IPPV use = Fulltime(%) | 2 (0.5) | 1 (0.3) | 1 (2.2) | 0.179 |
| Negative pressure use = Fulltime (%) | 1 (0.2) | 1 (0.3) | 0 (0.0) | 0.071 |
| Invasive (with tracheotomy) use = No (%) | 0.236 | |||
| Not Specified | 437 (99.3) | 393 (99.5) | 44 (97.8) | |
| Nocturnal | 1 (0.2) | 0 (0.0) | 1 (2.2) | |
| Full-time | 2 (0.5) | 2 (0.5) | 0 (0.0) | |
| Supplemental oxygen use = No (%) | 0.176 | |||
| Not Specified | 434 (98.6) | 389 (98.5) | 45 (100.0) | |
| Nocturnal | 3 (0.7) | 3 (0.8) | 0 (0.0) | |
| Full-time | 3 (0.7) | 3 (0.8) | 0 (0.0) | |
| Age Group (%) | 1.621 | |||
| 0–4 | 51 (11.6) | 51 (12.9) | 0 (0.0) | |
| 5–8 | 170 (38.6) | 170 (43.0) | 0 (0.0) | |
| 9–12 | 89 (20.2) | 72 (18.2) | 17 (37.8) | |
| 13–16 | 57 (13.0) | 48 (12.2) | 9 (20.0) | |
| 17+ | 73 (16.6) | 54 (13.7) | 19 (42.2) |
Abbreviations: BIPAP bilevel positive airway pressure, CPAP continuous positive airway pressure, EF ejection fraction, FVC forced vital capacity, IPPV intermittent positive pressure ventilation, SF shortening fraction
*
SMD standardized mean difference: <0.2 = small, 0.2– 0.5 medium, and >0.8 large difference between means