Organ | Visit (statistics) | Treatment-naïve group (n = 8) | Prior ERT group (n = 93) | Prior ERT and SRT group (n = 5)a | Total patients (N = 106) |
---|---|---|---|---|---|
Liver, median (range) | Baseline | n = 6 | n = 68 | n = 4 | n = 78 |
Absolute values | 1.4 (0.3–2.1) | 2.4 (0.1–10.9) | 0.7 (0.6–0.8) | 2.2 (0.1–10.9) | |
Year 1 (month 12) | n = 2b | n = 40 | n = 0 | n = 42 | |
Absolute values | 0.9 (0.6, 1.2) | 2.4 (0.5–6.2) | – | 2.3 (0.5–6.2) | |
Change from baselinec | 0.1 (− 0.1, 0.4) | 0.0 (− 5.3–1.0) | – | 0.0 (− 5.3–1.0) | |
% change from baselinec | 63.4 (− 5.3, 132.1) | 1.5 (− 50.9–877.5) | – | 1.5 (− 50.9–877.5) | |
Year 2 (month 24) | n = 0 | n = 29 | n = 0 | n = 29 | |
Absolute values | – | 2.7 (0.6–4.7) | 2.7 (0.6–4.7) | ||
Change from baselinec | – | − 0.1 (− 6.6–2.3) | – | − 0.1 (− 6.6–2.3) | |
% change from baselinec | – | − 3.0 (− 60.0–159.3) | – | − 3.0 (− 60.0–159.3) | |
Spleen, median (range) | Baseline | n = 4 | n = 53 | n = 2 | n = 59 |
Absolute values | 9.5 (6.8–18.1) | 4.9 (0.6–121.5) | 1.8 (1.6, 2.0) | 5.3 (0.6–121.5) | |
Year 1 (month 12) | n = 2b | n = 33 | n = 0 | n = 35 | |
Absolute values | 4.5 (1.9, 7.0) | 4.9 (1.7–120.0) | – | 4.9 (1.7–120.0) | |
Change from baselinec | − 9.7 (− 11.1, − 8.3) | 0.0 (− 82.5–42.3) | – | − 0.2 (− 82.5–42.3) | |
% change from baselinec | − 71.1 (− 81.0, − 61.3) | − 0.9 (− 67.9–863.0) | – | − 6.5 (− 81.0–863.0) | |
Year 2 (month 24) | n = 0 | n = 24 | n = 0 | n = 24 | |
Absolute values | – | 4.2 (0.8–23.4) | 4.2 (0.8–23.4) | ||
Change from baselinec | – | − 1.0 (− 98.1–4.2) | – | − 1.0 (− 98.1–4.2) | |
% change from baselinec | – | − 25.0 (− 80.7–114.8) | – | − 25.0 (− 80.7–114.8) |