Table 4.
Comparison of patients’ characteristics between patients with and without neuromuscular symptoms
| Patient characteristics | Patients with neuromuscular symptoms, n = 24 | Patients without neuromuscular symptoms, n = 76 | P value |
|---|---|---|---|
| Age at diagnosis, mo, median (IQR) | 16 (12–23) | 18 (12–24) | 0.5 |
| Age at cysteamine initiation, mo, median (IQR) | 18 (12–30) | 18 (13–30) | 0.74 |
| Male sex, n (%) | 13 (54) | 24 (48) | 0.62 |
| History of dialysis, n (%) | 8 (33) | 21 (42) | 0.47 |
| History of kidney transplantation, n (%) | 16 (67) | 32 (64) | 0.82 |
| Age at inclusion, yr, median (IQR) | 25.0 (12.0–33.0) | 26.0 (15.0–32.0) | 0.67 |
| Pediatric patients (vs. adults), n (%) | 10 (42) | 16 (32) | 0.41 |
| Type of treatment at inclusion, n (%) | 0.78 | ||
| Immediate release cysteaminea | 8 (33) | 17 (34) | |
| Delayed release cysteamineb | 16 (67) | 32 (64) | |
| eGFR at inclusion, ml/min per 1.73 m2, median (IQR) | 48 (32–62) | 60 (37–79) | 0.28 |
| CKD stage, n (%) | |||
| 1 | 1 (6) | 3 (8) | 0.12 |
| 2 | 3 (19) | 17 (44) | |
| 3A | 6 (38) | 5 (13) | |
| 3B | 2 (13) | 10 (26) | |
| 4 | 4 (25) | 4 (10) |
CKD, chronic kidney disease; eGFR, estimated glomerular filtration rate; IQR, interquartile range.
a
Immediate release cysteamine (Cystagon; Mylan Pharmaceuticals, Canonsburg, PA).
b
Delayed release cysteamine (Procysbi; Horizon Therapeutics, Dublin, Ireland).