Echocardiography-derived changes in patients with amenable mutations
| Parameter | Baseline mean | Change from baseline to month 18 (95% CI) |
|---|---|---|
| Migalastat: LVMi (g/m2) | ||
| All (n=33) (% abnormal) | 95.3 (39) | −6.6 (−11.0 to −2.2)* |
| LVH† at baseline (9 females and 4 males) | 116.7 | −8.4 (−15.7 to 2.6) |
| ERT: LVMi (g/m2) | ||
| All (n=16) (% abnormal) | 92.9 (31) | −2.0 (−11.0 to 7−0) |
| LVH† at baseline (n=5) (1 female and 4 males) | 123.3 (100%) | 4.5 (−20.9 to 30.0) |
| Migalastat: LVPWT (cm) | ||
| All (n=33) | 1.17 | −0.035 (−0.077 to 0.007) |
| ERT: LVPWT (cm) | ||
| All (n=16) | 1.08 | 0.029 (−0.037 to 0.094) |
| Migalastat: IVSWT (cm) | ||
| All (n=33) | 1.16 | 0.058 (−0.200 to 0.140) |
| ERT: IVSWT (cm) | ||
| All (n=16) | 1.18 | 0.037 (−0.051 to 0.124) |
LVMi (g/m2): normal: female, 43–95, male, 49–115; LVPMT (cm): normal: female, 0.6–<1.0, male, 0.6–<1.1; IVSWT (cm): normal: female, 0.6–0.9, male, 0.6–1.0.
*Statistically significant (95% CI does not overlap zero).
†LVH; defined as LVMi (g/m2) >95 (females) or >115 (males).
ERT, enzyme replacement therapy; IVSWT, intraventricular septal wall thickness; LVH, left ventricular hypertrophy; LVMi, left ventricular mass index; LVPMT, left ventricular posterior wall thickness; LVPWT, left ventricular posterior wall thickness diastolic.