Table 1.
Baseline characteristics of neuromuscular dystrophy patients.
| Characteristics | Total population (n = 39) |
|---|---|
| Age at evaluation (y), mean (SD) | 31 (11) |
| Male sex, n (%) | 32 (82) |
| Body mass index (kg/m2), mean (SD) | 23 (5) |
| Muscular dystrophy | |
| Duchenne muscular dystrophy, n (%) | 20 (51) |
| Spinal muscular atrophy, n (%) | 5 (13) |
| Becker muscular dystrophy, n (%) | 3 (8) |
| Morbus steinert/Myotonic dystrophy type 1, n (%) | 3 (8) |
| Metabolic myopathy, n (%) | 1 (3) |
| Congenital myopathy, n (%) | 4 (10) |
| Limb girdle muscular dystrophy, n (%) | 3 (8) |
| Respiratory status | |
| FEV1 (L), mean (SD) | 2.0 (1.4) |
| FVC (L), mean (SD) | 2.2 (1.4) |
| No support, n (%) | 21 (54) |
| Invasive ventilation, n (%) | 9 (23) |
| Nocturnal support, n (%) | 9 (23) |
| All day support, n (%) | 9 (23) |
| Ejection fraction (%), mean (SD) | 46 (12) |
| LVEF ≥ 50, n (%) | 20 (56) |
| LVEF 40–49, n (%) | 9 (25) |
| LVEF <40, n (%) | 7 (19) |
| Medication, n (%) | |
| Corticosteroids | 1 (3) |
| ACEi | 18 (46) |
| ARB | 2 (5) |
| MRA | 3 (8) |
| Laboratory measurements | |
| Cystatin C (mg/L), median [IQR] | 0.81 [0.71–0.92] |
| Creatinine (umol/L), median [IQR] | 10 [7–23] |
| Cystatin C-based eGFR (mL/min/1.73 m2), mean (SD) | 110 (23) |
| Creatinine-based eGFR (mL/min/1.73 m2), mean (SD) | 242 (80) |
| NT-proBNP (ng/L), median [IQR] | 56 [28–143] |
| hs-cTnT (ng/L), median [IQR] | 18 [12–42] |
| Galectin-3 (μg/L), median [IQR] | 10.5 [9.3–12.9] |
ACEi, angiotensin-converting enzyme inhibitor; ARB, angiotensin II receptor blocker; eGFR, estimated glomerular filtration rate; FEV1, forced expiratory volume in 1 second; FVC, forced vital capacity; hs-cTnT, high-sensitivity cardiac Troponin-T; LVEF, left ventricular ejection fraction; MRA, mineralocorticoid receptor antagonist; NT-proBNP, N-terminal pro-B-type natriuretic peptide.