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Fig. 4 | Orphanet Journal of Rare Diseases

Fig. 4

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. 4

Modeling of percent predicted FEV1 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 = 83). (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). Patients who had cognitive impairment at any time were excluded from the model. Patients aged under 5 years at the time of assessment were also excluded owing to the unreliability of these assessments in children aged under 5 years [17]; pre-ERT values are therefore labeled ‘not applicable’ for patients aged under 5 years at ERT start. Values corresponding to absolute FEV1 above 10 L were excluded from the model. Abbreviations: CI, confidence interval; ERT, enzyme replacement therapy; FEV1, forced expiratory volume in 1 s; IV, intravenous; NA, not applicable; ND, not determined; SE, standard error

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