Endpoint | Visit (statistics) | Treatment-naïve group (n = 8) | Prior ERT group (n = 93) | Prior ERT and SRT group (n = 5)a | Total patients (N = 106) |
---|---|---|---|---|---|
Hemoglobin, mean (SD), g/dL | Baseline | n = 8 | n = 83 | n = 4 | n = 95 |
Absolute values | 11.6 (1.4) | 13.3 (1.7) | 13.8 (1.5) | 13.2 (1.7) | |
Year 1 (month 12) | n = 3 | n = 54 | n = 1 | n = 58 | |
Absolute values | 13.0 (0.8) | 13.1 (1.5) | 14.4 | 13.2 (1.5) | |
Change from baselineb | 1.2 (1.3) | 0 (0.8) | − 0.7 | 0 (0.9) | |
% change from baselineb | 10.5 (11.3) | 0.1 (6.7) | − 4.6 | 0.5 (7.2) | |
Year 2 (month 24) | n = 0 | n = 44 | n = 0 | n = 44 | |
Absolute values | – | 13.4 (1.3) | – | 13.4 (1.3) | |
Change from baselineb | – | 0.1 (0.9) | – | 0.1 (0.9) | |
% change from baseline | – | 0.7 (7.3) | – | 0.7 (7.3) | |
Platelet count without splenectomy, mean (SD), 103/μL | Baseline | n = 5 | n = 66 | n = 2 | n = 73 |
Absolute values | 128.8 (30.3) | 169.2 (77.0) | 202.5 (21.9) | 167.3 (74.5) | |
Year 1 (month 12) | n = 3 | n = 41 | n = 1 | n = 45 | |
Absolute values | 137.7 (35.4) | 168.0 (52.4) | 187.0 | 166.4 (51.2) | |
Change from baselineb | 17.0 (18.2) | 7.9 (42.5) | − 31.0 | 7.6 (41.2) | |
% change from baselineb | 14.3 (14.4) | 10.7 (32.4) | − 14.2 | 10.4 (31.2) | |
Year 2 (month 24) | n = 0 | n = 36 | n = 0 | n = 36 | |
Absolute values | – | 193.7 (80.0) | – | 193.7 (80.0) | |
Change from baselineb | – | 22.1 (38.1) | – | 22.1 (38.1) | |
% change from baselineb | – | 20.0 (35.1) | – | 20.0 (35.1) | |
Platelet count with splenectomy, mean (SD), 103/μL | Baseline | n = 3 | n = 16 | n = 0 | n = 19 |
Absolute values | 183.7 (28.3) | 250.0 (100.2) | – | 239.7 (95.3) | |
Year 1 (month 12) | n = 0 | n = 9 | n = 0 | n = 9 | |
Absolute values | – | 235.1 (87.8) | – | 235.1 (87.8) | |
Change from baselineb | – | − 7.0 (60.7) | – | − 7.0 (60.7) | |
% change from baselineb | – | 1.7 (25.2) | – | 1.7 (25.2) | |
Year 2 (month 24) | n = 0 | n = 7 | n = 0 | n = 7 | |
Absolute values | – | 281.1 (89.2) | – | 281.1 (89.2) | |
Change from baselineb | – | − 2.1 (68.1) | – | − 2.1 (68.1) | |
% change from baselineb | – | 1.0 (27.8) | – | 1.0 (27.8) |