Table 1

Patient characteristics at start of ERT

Agalsidase alfa (0.2 mg/kg)Agalsidase beta (1.0 mg/kg)P value
Patients248139
Men, classical*69 (28%)71 (51%)<0.001
Men, non-classical47 (19%)7 (5%)0.22
Women, classical95 (38%)43 (30%)0.14
Women, non-classical37 (15%)18 (13%)0.86
ERT start <18 years of age15 (6%)3 (2%)0.13
Follow-up time (years)5.2 (0.8–14.4)3.8 (0.8–12.1)<0.001
Events before initiation of ERT
 Dialysis/renal transplant8 (3%)12 (9%)0.007
 PM/ICD21 (8%)9 (7%)0.87
 Stroke22 (9%)17 (12%)0.09
 Any of the above†46 (19%)31 (22%)0.08
LysoGb3 (nmol/L)10 (0.7–146)80 (2.0–178)<0.001
eGFR (mL/min/1.73 m2)89 (10–159)86 (10–140)0.009
CKD category A344/195 (23%)42/113 (37%)0.008
LVMI (g/m2.7)49 (15–117)52 (20–148)0.14
Use of ACEi/ARBs89/248 (36%)52/139 (37%)0.83
Hypertension109/236 (39%)62/137 (45%)0.23
BMI (kg/m2)26 (±4.9)25 (±5.6)0.30
HDL cholesterol (mmol/L)1.5 (±0.4)1.5 (±0.4)0.92
LDL cholesterol (mmol/L)2.7 (±0.9)2.7 (±0.8)0.76
Total cholesterol (mmol/L)4.8 (±1.1)4.7 (±1.0)0.51
Triglycerides (mmol/L)1.2 (0.2–5.9)1.2 (0.3–3.6)0.18
  • Continuous variables are presented as mean (±SD) or median (range). Hypertension is defined as a diagnosis of increased blood pressure by the treating physician.CKD category A3 is defined as AER > 300 g/day or equivalent. Missing values (percentage): lysoGb3 (54%), eGFR (5%), LVMI (18%), BMI (5%), HDL cholesterol (28%), LDL cholesterol (18%), total cholesterol (17%), triglycerides (17%). For baseline characteristics per sex and phenotype see online supplementary table S1, for detailed genotype-phenotype information see online supplementary table S2.

  • *Classical, strictly defined including the presence of at least one characteristic symptom (angiokeratoma, acroparesthesia or cornea verticillata).

  • †Reflects the number of patients with one or more events (either dialysis or renal transplant, PM/ICD and/or stroke).

  • ERT, enzyme replacement therapy; PM, pacemaker; ICD, implantable cardiac device; lysoGb3, globotriaosylsphingosine; eGFR, estimated glomerular filtration rate; CKDA, chronic kidney disease albuminuria categories; LVMI, left ventricular mass index measured by echocardiography; ACEi, ACE inhibitors; ARB, angiotensin receptor blocker; BMI, body mass index; HDL, high-density lipoprotein cholesterol; LDL, low-density lipoprotein cholesterol.