Demographics and patient characteristics
| Characteristic | Low renal involvement (LRI)* pretreatment | High renal involvement (HRI)* pretreatment | Total | p Value (LRI vs HRI) |
|---|---|---|---|---|
| Patients enrolled (men/women) | 31, 1 | 19, 1 | 52 (50, 2) | |
| Age at treatment initiation (years) | <0.001 | |||
| Mean (SD) | 25.3 (7.7) | 37.7 (8.7) | 30.1 (10.0) | |
| Median (range) | 22.5 (16.5–44.7) | 36.6 (26.3–62.2) | 29.4 (16.5–62.2) | |
| Age at last Registry follow-up (years) | <0.001 | |||
| Mean (SD) | 37.2 (7.6) | 48.7 (8.2) | 41.6 (9.6) | |
| Median (range) | 34.8 (26.8–53.5) | 47.3 (38.5–73.8) | 40.0 (26.8–73.8) | |
| Follow-up time (years) | 0.659 | |||
| Mean (SD) | 8.7 (2.5) | 8.4 (2.5) | 8.6 (2.5) | |
| Median (range) | 10.0 (1.4–10.5) | 10.0 (4.5–10.5) | 10.0 (1.4–10.5) | |
| UPCR (g/g) at baseline | <0.001 | |||
| Mean (SD) | 0.2 (0.1) | 1.3 (0.9) | 0.6 (0.8) | |
| Median (range) | 0.1 (0–0.5) | 1.1 (0.4–3.6) | 0.3 (0–3.6) | |
| eGFR (mL/min/1.73 m2) at baseline | <0.001 | |||
| Mean (SD) | 126.0 (18.71) | 101.6 (22.78) | 116.6 (23.44) | |
| Median (range) | 127.7 (73.1–170.0) | 108.9 (49.1–138.5) | 118.8 (49.1–170.0) | |
| LPWT at baseline (mm) | 0.005 | |||
| Mean (SD) | 9.8 (1.66) | 11.9 (2.67) | 10.6 (2.31) | |
| Median (range) | 10.0 (7.0–13.0) | 11.0 (9.0–18.9) | 10.4 (7.0–18.9) | |
| IVST (mm) at baseline | 0.007 | |||
| Mean (SD) | 10.0 (1.73) | 11.9 (2.63) | 10.7 (2.31) | |
| Median (range) | 10.0 (7.0–16.0) | 11.0 (9.0–20.0) | 10.3 (7.0–20.0) | |
| Plasma GL-3 (μg/mL) at baseline | 0.696 | |||
| Mean (SD) | 9.5 (3.02) | 9.9 (2.66) | 9.7 (2.86) | |
| Median (range) | 9.5 (3.6–16.7) | 9.6 (4.7–14.5) | 9.5 (3.6–16.7) | |
*LRI=UPCR ≤0.5 g/g and <50% sclerotic glomeruli; HRI=UPCR >0.5 g/g or ≥50% sclerotic glomeruli.
N=52 for all parameters, except for LPWT and IVST (both N=50), and UPCR (N=47).
eGFR, estimated glomerular filtration rate; GL-3, globotriaosylceramide; IVST, interventricular septum thickness; LPWT, left ventricular posterior wall thickness; UPCR, urine protein to creatinine ratio.