Effects of imiglucerase on the growth and metabolism of Gaucher disease type I patients: a systematic review

Nutrition & Metabolism

Table 1 Summary of weight and height in the pre- and post-enzyme replacement therapy (ERT) in children and adolescents with Gaucher disease type I

		Before enzyme replacement therapy						After enzyme replacement therapy
Country	Authors	Sample <18 years (n)	Mean age at diagnosis or presentation (yo)	Mean age at baseline or evaluation (yo)	Low weight (<p5) (%)	Low height (<p5) (%)	Growth retardation (%)	Sample <18 years at the beginning of ERT (n)	Time on ERT (years)	Mean dosage of imiglucerase	Improved percentile or z-score of height (%)
Japan	Ida et al. [10]	19	2.7	7.4	10	74	74	-	-	-	-
Canada	MacKenzie et al. [11]	15	-	-	-	-	47	-	-	-	-
ICGG^a	Kaplan et al. 2006 [12]	496	-	-	-	34	-	-	-	-	-
Tunisia	BenTurkia et al. [13]	11	4.2	8.2	-	-	36	-	-	-	-
Albania	Shehi et al. [8]	8	-	-	-	-	25	-	-	-	-
Israel	Kauli et al. [14]	30	-	5.1	-	-	63^b	11/30	1-7	30 UI/kg/mo^d	-^e
Romania	Grigorescu et al. [15]	6	7.9	14.5	-	33	-	6/6	1.5	45 UI/kg/inf	-^f
Israel	Zevin et al. [16]	30	5.6	9.9	27	33	33	6/30	1	30 UI/kg/mo	50
Italy	Bembi et al. [17]	6	-	10.2	50	50	-	6/6	1.1	43 UI/kg/mo	50
USA	Kaplan et al. [18]	18	4.5	-	-	50	50	18/18	1	48 UI/kg/inf	71
ICGG^a	Kaplan et al. [18]	81	6.6	-	-	48	48	36/81	1.5	-	64
Brazil	Oliveira et al. [19]	13	5.8	-	15^c	36^c	-	13/13	-	-	55
Argentina	Drelichman et al. [20]	5	5.2	6.2	60	60	80	5/5	1.2	-	80
Brazil	Sobreira; Bruniera [21]	41	-	-	-	46^c	-	41/41	2	35 UI/kg/inf	77
ICGG^a	Andersson et al. [22]	702	-	-	-	42	-	702/702	8	39 UI/kg/inf	-^g

Yo= years old; ICGG= International Collaborative Gaucher Group.
a- Anderson et al. 2008 included the patients of the study by Kaplan et al. 2006 and 1996.
b- 63% of patients with growth retardation in childhood (19/30).
c- Less than or equal to score 2 standard deviation.
d- The patients received 2.3 U/kg 3 times weekly, i.e., 30 U/kg/month. In children whose bone disease had worsened, the dose was increased to 30 U/kg every 2 weeks.
e- The authors showed the mean of standard deviation score for the height of the group without the individual patients’ data, but noted that the growth velocity gradually increased.
f -The authors reported that the patients normalised their growth, however did not show the corresponding data.
g -The median height z-score for the study population was −1.4 at baseline and improved to −0.3 after 8 years of ERT, similar to the median for normal population.

ISSN: 1743-7075