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Fig. 6 | Orphanet Journal of Rare Diseases

Fig. 6

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. 6

Modeling of palpable liver size over time following IV idursulfase treatment using data from patients who received ERT 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 = 413). (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). The size of a non-palpable liver was imputed as zero in the model. Abbreviations: CI, confidence interval; ERT, enzyme replacement therapy; IV, intravenous; SE, standard error

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172