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 3 Contingency table and statistical analysis of hepatomegaly data

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

Group Pre-post McNemar test p-value Positive Outcomes
Y-Y Y-N N-Y N-N Proportion CI (95%)
A 3 3 4 1 1.0 0.36 (0.109, 0.692)
B 1 6 0 0 0.03* 0.86 (0.421, 0.996)
C 3 2 0 2 0.50 0.57 (0.184, 0.901)
A + B 4 9 4 1 0.27 0.56 (0.308, 0.785)
A + B + C 7 11 4 3 0.12 0.56 (0.349, 0.756)
  1. The category 'Positive Outcomes" includes both YES-NO (Y-N: improvement) and NO-NO (N-N: stabilization) cases. Confidence intervals, calculated by the Clopper and Pearson method and the p-value of McNemar test are reported. Statistically significant values (p < 0.05) are marked with an asterisk. Group A: n = 11, group B: n = 7, group C: n = 7.