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Fig. 1 | Orphanet Journal of Rare Diseases

Fig. 1

From: Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)

Fig. 1

Modeling of percentage change in uGAG levels from pre-ERT over time using data from patients who received ERT with IV idursulfase for 5 years or more. (a) Change in predicted means and 95% CIs by age at ERT start (colored lines) and individual patient values (gray lines) from pre-ERT up to 8 years after IV idursulfase start in the main analysis population (patients with data available for at least two time points in total and at least one post-ERT time point; n = 180). The y-axis was terminated at 100% for clarity; some patients had individual values of up to 297% at certain time points, which are not shown here. (b) Predicted means by age at ERT start at pre-ERT and 8 years post-ERT in the main analysis population and the internal validation population (patients with data available at five or more time points). Normalized uGAG levels above 1500 µg/mg (169.5 mg/mmol) creatinine were excluded from the model. Abbreviations: CI, confidence interval; ERT, enzyme replacement therapy; IV, intravenous; ND, not determined; SE, standard error; uGAG, urinary glycosaminoglycan

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172