|Component||Migalastat (n=34)||ERT (n=18)|
↑Proteinuria (6), ↓GFR (2)
↑Proteinuria (4), ↓GFR (3)
Chest pain, VT/chest pain
Cardiac failure, dyspnoea, arrhythmia
|CNS||0 (0%)||1 (6%)|
|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.