Table 4.
Composite clinical outcome: number of patients (mITT population)
| Component | Migalastat (n=34) | ERT (n=18) |
|---|---|---|
| Renal | 8 (24%) ↑Proteinuria (6), ↓GFR (2) |
6 (33%) ↑Proteinuria (4), ↓GFR (3) |
| Cardiac | 2 (6%) Chest pain, VT/chest pain |
3 (17%) Cardiac failure, dyspnoea, arrhythmia |
| CNS | 0 (0%) | 1 (6%) TIA |
| Death | 0 (0%) | 0 (0%) |
| Any | 10 (29%) | 8 (44%) |
Analyses undertaken in the mITT patients (randomised patients with amenable mutations receiving at least one dose of study drug and having baseline and postbaseline mGFRiohexol and eGFRCKD-EPI measures). Proteinuria event defined as >33% increase in 24-hour urine protein and level >300 mg; GFR event defined as >15 mL/min decline in eGFRCKD-EPI and level <90 mL/min/1.73 m2/year. Two patients in the ERT group experienced one cardiac and one renal event each.
CNS, central nervous system; eGFRCKD-EPI, estimated GFR using the Chronic Kidney Disease Epidemiology Collaboration formula; ERT, enzyme replacement therapy; GFR, glomerular filtration rate; mGFRiohexol, measured GFR using iohexol clearance; mITT, modified intention to treat; TIA, transient ischaemic attack; VT, ventricular tachycardia.