Skip to main content

Advertisement

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

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

   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 - - - -
ICGGa 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 - - 63b 11/30 1-7 30 UI/kg/mod -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
ICGGa Kaplan et al. [18] 81 6.6 - - 48 48 36/81 1.5 - 64
Brazil Oliveira et al. [19] 13 5.8 - 15c 36c - 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 - - - 46c - 41/41 2 35 UI/kg/inf 77
ICGGa Andersson et al. [22] 702 - - - 42 - 702/702 8 39 UI/kg/inf -g
  1. Yo= years old; ICGG= International Collaborative Gaucher Group.
  2. a- Anderson et al. 2008 included the patients of the study by Kaplan et al. 2006 and 1996.
  3. b- 63% of patients with growth retardation in childhood (19/30).
  4. c- Less than or equal to score 2 standard deviation.
  5. 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.
  6. 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.
  7. f -The authors reported that the patients normalised their growth, however did not show the corresponding data.
  8. 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.