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

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

	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 baseline^a	Baseline	9 Months	33 Months, percent change from baseline^a
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 baseline^b	–	− 21.3% (n = 23)	−51.5% (n = 10)	–	− 29.7% (n = 5)	−97.1% (n = 2)
CCL18, percent change from baseline^b	–	Decrease (n = 23)	−36.5% (n = 10)	–	Decrease (n = 5)	−10.8% (n = 2)

Values represent means
^aAbsolute values at 33 months not reported; percent change from baseline reported
^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
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)

ISSN: 1750-1172