Adults | Children | ||||||
---|---|---|---|---|---|---|---|
PB-06-001 [15] N = 32a | PB-06-003 [18] N = 26 | PB-06-007 [21] N = 19 | PB-06-005 [17] N = 11 | PB-06-006 [20] N = 10 | |||
Parameter | Baseline | 9 Months change from baselineb | 36 Months | 60 Months | Baseline | 12 Months | 36 Months |
Spleen volume, MN | |||||||
30 U/kg | 15 (n = 15) | −26.9% (n = 15) | 8.2 (n = 12) | 6.6 (n = 7) | 22.2 (n = 6) | 14.0 (n = 6) | 9.0 (n = 4) |
60 U/kg | 17 (n = 16) | −38.0% (n = 16) | 5.6 (n = 11) | 3.2 (n = 7) | 29.4 (n = 5) | 12.9 (n = 5) | 6.6 (n = 3) |
Liver volume, MN | |||||||
30 U/kg | 1.7 (n = 15) | −10.5% (n = 14) | 1.3 (n = 12) | 1.2 (n = 7) | 1.8 (n = 6) | 1.5 (n = 6) | 1.3 (n = 4) |
60 U/kg | 1.6 (n = 16) | −11.1% (n = 15) | 1.1 (n = 11) | 1.0 (n = 7) | 2.2 (n = 5) | 1.7 (n = 5) | 1.5 (n = 3) |
Haemoglobin, mg/dL | |||||||
30 U/kg | 12.2 (n = 15) | + 1.6 (n = 14) | 14.3 (n = 11) | 14.1 (n = 7) | 11.3 (n = 6) | 12.7 (n = 6) | 13.2 (n = 5) |
60 U/kg | 11.4 (n = 16) | + 2.2 (n = 15) | 14.0 (n = 11) | 13.9 (n = 8) | 10.6 (n = 5) | 12.2 (n = 5) | 12.7 (n = 4) |
Platelet count, /mm3 | |||||||
30 U/kg | 75,320 (n = 15) | + 11,427 (n = 15) | 94,683 (n = 12) | 104,986 (n = 7) | 162,667 (n = 6) | 208,167 (n = 6) | 220,020 (n = 5) |
60 U/kg | 65,038 (n = 16) | + 41,494 (n = 16) | 147,727 (n = 11) | 180,625 (n = 8) | 99,600 (n = 5) | 172,200 (n = 5) | 243,750 (n = 4) |
Chitotriosidase, percent change from baselinec | |||||||
30 U/kg | ─ | − 47% (n = 14) | − 71.5% (n = 12) | −83.1% (n = 7) | ─ | − 58.5% (n = 6) | −72.7 (n = 5) |
60 U/kg | ─ | −58% (n = 15) | −82.2% (n = 10) | −93.4% (n = 7) | ─ | −66.1% (n = 4) | − 84.4 (n = 3) |
CCL18, percent change from baselineb | |||||||
30 U/kg | ─ | NRd | − 58.1% (n = 12) | −66.7% (n = 7) | ─ | NRd | − 67.7% (n = 5) |
60 U/kg | ─ | NRd | − 71.0% (n = 11) | −83.3% (n = 8) | ─ | NRd | − 73.2% (n = 4) |