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Table 4 Efficacy results for taliglucerase alfa in treatment-switched patients

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

  Adults Children
  PB-06-002 [16]
N = 26
PB-06-003 [19]
N = 10
PB-06-002 [16]
N = 5
PB-06-006 [20]
N = 5
Parameter Baseline 9 Months 33 Months, percent change from baselinea Baseline 9 Months 33 Months, percent change from baselinea
Spleen volume, MN 5.5
(n = 20)
5.1
(n = 20)
−19.8%
(n = 7)
4.1
(n = 5)
3.3
(n = 5)
−5.3%
(n = 2)
Liver volume, MN 1.0
(n = 23)
0.9
(n = 23)
No change
(n = 8)
1.3
(n = 5)
1.2
(n = 5)
− 8.8%
(n = 2)
Haemoglobin, mg/dL 13.5
(n = 25)
Stable
(n = 25)
−1.0%
(n = 10)
13.5
(n = 5)
Stable
(n = 5)
+ 3.3%
(n = 2)
Platelet count, /mm3 160,447
(n = 25)
Stable
(n = 25)
Nominal
(n = 10)
164,587
(n = 5)
Stable
(n = 5)
+ 2.3%
(n = 2)
Chitotriosidase, percent change from baselineb − 21.3%
(n = 23)
−51.5%
(n = 10)
− 29.7%
(n = 5)
−97.1%
(n = 2)
CCL18, percent change from baselineb Decrease
(n = 23)
−36.5%
(n = 10)
Decrease
(n = 5)
−10.8%
(n = 2)
  1. Values represent means
  2. aAbsolute values at 33 months not reported; percent change from baseline reported
  3. bTo determine change from baseline for biomarkers, baseline values from entry into the original study (PB-06-002) were re-run at each time point due to high assay variability
  4. 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)