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Table 3 Efficacy results for taliglucerase alfa in treatment-naïve patients

From: Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease

  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)
  1. Values represent means
  2. a32 patients were randomized; 31 received treatment
  3. bAbsolute values at 9 months were not reported; percent change from baseline was reported for organ volumes and absolute change from baseline was reported for haematological parameters
  4. cTo determine change from baseline for biomarkers, baseline values from entry into the original study (PB-06-001 for adults; PB-06-005 for children) were re-run at each time point due to high assay variability
  5. dValues are available for individual patients in the publications cited
  6. CCL18, chemokine (C-C) motif ligand 18; MN, multiples of normal where normal spleen volume is 2 mL/kg × body weight (kg) and normal liver volume is 25 mL/kg × body weight (kg); NR, not reported