Fig. 2

Modeling of LVMI over time following IV idursulfase treatment for patients irrespective of LVH status of baseline using data from patients who received ERT for 5 years or more. (a) Change in predicted means and 95% CIs (colored lines) and individual patient values (gray lines) by age at ERT start 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 = 250). The y-axis was terminated at 250 g/m² for clarity; some patients had individual values of up to 314 g/m² 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). LVMI values > 400 g/m² were excluded from the model. For reference, 50–102 g/m² (indexed to BSA) has previously been defined as a normal LVMI range in male patients [19]. Abbreviations: BSA, body surface area; CI, confidence interval; ERT, enzyme replacement therapy; IV, intravenous; LVH, left ventricular hypertrophy; LVMI, left ventricular mass index; SE, standard error