5 and ≤ 12 years. Although other clinical signs improved in some of the patients monitored, statistical analysis of their variation did not reveal any significant changes following enzyme administration. The evaluation of ERT efficacy in relation to the severity of the disease evidenced slightly higher improvements as for hepatomegaly, splenomegaly, otological disorders and adenotonsillar hypertrophy in severe vs attenuated patients. Conclusions Although the present protocol of idursulfase administration may result efficacious in delaying the MPS II somatic disease progression at some extent, in this study we observed that several signs and symptoms did not improve during the therapy. Therefore, a strict monitoring of the efficacy obtained in the patients under ERT is becoming mandatory for clinical, ethical and economic reasons."/>
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Table 2 Statistical analysis of GAG percentage variation from pre-ERT values

From: Clinical efficacy of Enzyme Replacement Therapy in paediatric Hunter patients, an independent study of 3.5 years

Age group Time point from start of ERT n Median (minimum-maximum) p-value
A 1 yr ± 4 m 12 37.6 (7.8-76.0) 0.0005*
2 yrs ± 4 m 9 29.4 (3.9-92.1) 0.0039*
3 yrs ± 4 m 7 24.5 (13.7-81.8) 0.0156*
Last 13 25.4 (5.7-79.2) 0.0002*
B 1 yr ± 4 m 6 33.9 (17.1-88.1) 0.0313*
2 yrs ± 4 m 4 19.5 (17.4-36.0) 0.1250
3 yrs ± 4 m 4 19.2 (18.0-23.1) 0.1250
Last 7 18.2 (14.6-55.8) 0.0156*
C 1 yr ± 4 m 5 26.4 (8.9-38.8) 0.0625
2 yrs ± 4 m 4 16.5 (9.3-29.6) 0.1250
3 yrs ± 4 m 5 26.4 (5.7-35.1) 0.0625
Last 5 13.3 (5.1-28.2) 0.0625
A + B 1 yr ± 4 m 18 35.6 (7.8-88.1) < .0001*
2 yrs ± 4 m 13 20.2 (3.9-92.1) 0.0002*
3 yrs ± 4 m 11 21.0 (13.7-81.8) 0.0010*
Last 20 24.0 (5.7-79.2) < .0001*
A + B + C 1 yr ± 4 m 23 31.8 (7.8-88.1) < .0001*
2 yrs ± 4 m 17 20.0 (3.9-92.1) < .0001*
3 yrs ± 4 m 16 22.0 (5.7-81.8) < .0001*
Last 25 18.2 (5.1-79.2) < .0001*
  1. Urinary GAG values are expressed at each time-point, from start of ERT as percentage with respect to the pre-ERT baseline level, taken as 100%. The number of available samples considered in the calculation (n), the median value and minimum and maximum values, together with the p-value of the statistical test applied (Wilcoxon signed rank test), are reported. Statistically significant values (p < 0.05) are marked with an asterisk.