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."/>
Skip to main content
Figure 3 | Orphanet Journal of Rare Diseases

Figure 3

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

Figure 3

Post-ERT cardiac valve regurgitation. Percentage of patients of group A, B, C, A + B and A + B + C presenting an improvement, a stabilization or a worsening of regurgitation of the mitral, aortic, tricuspid and pulmonary cardiac valves. Group A: n = 10, group B: n = 6, group C: n = 6 for tricuspid and pulmonary valves, n = 5 for mitral and aortic valves.

Back to article page