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Table 4 The mean age at baseline and follow-up, and changes in z-scores of LVMI, IVSd, and LVPWd for 12 patients without ERT compared to nine patients with ERT

From: Natural progression of cardiac features and long-term effects of enzyme replacement therapy in Taiwanese patients with mucopolysaccharidosis II

ERT

Age (years)

Duration (years)

LVMI (z score)

Change

IVSd (z score)

Change

LVPWd (z score)

Change

Without ERT (n = 12)

Baseline

7.3

8.1 (2.6–17.0)

0.83

1.52

2.40

1.80

0.58

1.25

Follow-up

15.4

2.35

4.20

1.82

With ERT (n = 9)

Baseline

15.2

7.2 (1.0–12.4)

2.78

0.05

3.01

-0.24

1.68

0.52

Follow-up

22.4

2.83

2.77

2.19

p value

p = 0.013

p = 0.069

p = 0.358

  1. MPS, mucopolysaccharidosis; ERT, enzyme replacement therapy; LVMI, left ventricular mass index; IVSd, interventricular septum thickness in diastole; LVPWd, left ventricular posterior wall thickness in diastole
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172