Baseline characteristics for all participants completing titration and follow-up visits: stratified by urine protein to creatinine ratio goal
| Variable | Total | Met UPCR goal | Above UPCR goal |
|---|---|---|---|
| Participants (n) | 24 (100%) | 18 (75%) | 6 (24%) |
| Age at baseline (years) | 43.1 (39.5–46.7) | 43.6 (39.4–47.8) | 41.7 (31.9–51.1) |
| Males | 15 (63%) | 10 (56%) | 5 (83%) |
| Blacks | 3 (13%) | 2 (11%) | 1 (17%) |
| Duration on agalsidase-beta (years) | 3.1 (0.3–4.4) | 2.9 (0.3–4.8) | 3.6 (1.4–4.1) |
| Agalsidase-beta started at age (years) | 42.8 (36.1–47.5) | 43.3 (38.0–47.9) | 37.2 (33.5–47.2) |
| Qualification values (historical data) | |||
| Urine protein–creatinine ratio (g/g) | 1.5 (1.1–2.3) | 1.4 (1.0–2.3) | 1.9 (1.5–2.3) |
| eGFR (mL/min/1.73 m2) | 71 (56–93) | 71 (55–94) | 73 (56–92) |
| Interval before enrolment (years) | 1.0 (0.1–2.2) | 0.6 (0.1–1.6) | 2.2 (1.8–2.4) |
| Initial baseline values (visit–3) | |||
| Systolic blood pressure (mm Hg) | 112 (103–128) | 110 (100–130) | 119 (112–121) |
| Diastolic blood pressure (mm Hg) | 73 (64–77) | 72 (63–82) | 73 (68–75) |
| Serum potassium (mEq/L) | 4.2 (4.0–4.5) | 4.2 (4.1–4.4) | 4.0 (3.8–4.9) |
| Urine protein–creatinine ratio (g/g) | 0.7 (0.4–1.3) | 0.6 (0.3–1.1) | 1.2 (0.4–2.3) |
| Urine albumin–creatinine ratio (mg/g) | 263 (73–759) | 124 (49–555) | 759 (459–1398) |
| eGFR (mL/min/1.73 m2) | 69 (53–93) | 69 (56–92)a | 62 (46–93) |
| Historical eGFR slope (N) (mL/min/1.73 m2/year;) N=12 | −6.2 (12) (−8.4 to −2.7) | −7.1 (7) (−9.3 to −5.4) | −3.2 (5) (−6.9 to −2.3) |
| Averaged treatment values | |||
| Systolic blood pressure (mm Hg) | 112 (104–124) | 112 (103–127) | 112 (107–117) |
| Diastolic blood pressure (mm Hg) | 71 (63–73) | 71 (62–78) | 70 (64–73) |
| Serum potassium (mEq/L) | 4.4 (3.9–4.7) | 4.4 (4.0–4.7) | 4.3 (3.8–4.9) |
| UPCR (g/g) | 0.5 (0.3–0.9) | 0.4 (0.2–0.5) | 0.9 (0.7–1.5) |
| ACR (mg/g) | 267 (123–637) | 226 (81–365) | 579 (454–733) |
| eGFR (mL/min/1.73 m2) | 65 (44–83) | 67 (48–83) | 46 (40–78) |
Continuous variables presented as median (25th–75th centile). Duration of enzyme replacement therapy (years), interval between starting agalsidase-beta at 1 mg/kg every other week and initial baseline visit
p<0.05 for differences in medians, comparing groups at and above UPCR goal with two-sample Wilcoxon rank-sum test (shown in bold). Qualification values indicate values recorded before enrolment based on local chart review. UPCR goal defined as UPCR for first treatment visit ≤0.5 g/g or averaged treatment UPCR ≤50% of baseline UPCR or averaged treatment UPCR ≤0.5 g/g.ACR, albumin to creatinine ratio; eGFR, estimated glomerular filtration rate (mL/min/1.73 m2); UPCR, urine protein to creatinine ratio.