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

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

	Adults				Children
	PB-06-001 [15] N = 32^a		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 baseline^b	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 baseline^c
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 baseline^b
30 U/kg	─	NR^d	− 58.1% (n = 12)	−66.7% (n = 7)	─	NR^d	− 67.7% (n = 5)
60 U/kg	─	NR^d	− 71.0% (n = 11)	−83.3% (n = 8)	─	NR^d	− 73.2% (n = 4)

Values represent means
^a32 patients were randomized; 31 received treatment
^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
^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
^dValues are available for individual patients in the publications cited
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

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