Table A2.
Parameters to Assess for Adverse Effects of PPS Treatment in MPS II.
| Patient 1 | Patient 2 | Patient 3 | ||||
|---|---|---|---|---|---|---|
| Before | After | Before | After | Before | After | |
| Pulmonary Function Test | Restrictive | Restrictive and obstructive | Normal | |||
| Vital Capacity (VC) | 1.66 | 1.74 | 1.02 | 4.09 | 4.12 | |
| Tidal Volume (TV) | 0.48 | 0.56 | 0.2 | 0.56 | 0.77 | |
| Forced Expiratory Volume/Second (FEV1) | 1.27 | 1.34 | 0.63 | 3.38 | 3.35 | |
| FEV1/VC | 79.37% | 78.33% | 67.74% | 83.40% | 82.10% | |
| Peak Expiratory Flow (PEF) | 2.86 | 2.99 | 1.85 | Not available | Not available | |
| Maximal Flow Rate of Expiration at 50% VC (V.50) | 1.54 | 1.43 | 0.66 | Not available | Not available | |
| Electrocardiogram | Mild left ventricle hypertrophy | No change | PR prolongation, high amplitude of V5 and V6, T-wave flattening | No change | Normal | Normal |
| Echocardiogram | Left Atrial to Aortic Root Ratio: 1.31, Ejection Fraction: 66.6% | Left Atrial to Aortic Root Ratio: 1.3, Ejection Fraction 64.5% | Ejection Fraction: 67% | Ejection Fraction: 63% | Not available | Not available |
| tricuspid regurgitation, pulmonary regurgitation, mitral regurgitation | No change | left ventricular enlargement, tricuspid regurgitation, aortic regurgitation, mitral regurgitation | No change | aortic valvular hypertrophy, aortic regurgitation | No change | |
| Hearing test | Right:55dB Left:53dB |
No change | Right: 73dB Left: 75dB |
No change | Not available | Not available |
| Visual test | Retinitis pigmentosa | No change | Retinitis pigmentosa, Visual field defect |
No change | Retinitis pigmentosa, Manual vision | Retinitis pigmentosa, Manual vision |
| Brain MRI | Bilateral diffuse white matter high signal | No change | Bilateral diffuse white matter high signal |
No change | Bilateral diffuse white matter high signal | No change |
| Perivascular dilation in subcortical white matter | No change | Perivascular dilation in subcortical white matter | No change | Perivascular dilation in subcortical white matter | No change | |
| Enlargement of bilateral ventricles | No change | Diffuse patchy high signal in bilateral thalamus, basal nucleus, midbrain, and pons | No change | |||
| Abdominal CT | No hepatosplenomegaly | No change | No hepatosplenomegaly | No change | No hepatosplenomegaly | No change |