Table 3.
Abdominal ultrasonographic assessments of liver and spleen size at baseline and after 5.9–11.2 years of enzyme replacement therapy (ERT) for 9 patients with mucopolysaccharidosis VI.
| Patient | Age at start of ERT (years) | Treatment duration (years) | Baseline | After ERT | Liver and spleen size |
|---|---|---|---|---|---|
| 1 | 11.7 | 11.2 | Hepatospenomegaly | Mild hepatomegaly | Improvement |
| 2 | 7.6 | 9.2 | Hepatospenomegaly | No hepatospenomegaly | Improvement |
| 3 | 8.3 | 9.1 | Hepatospenomegaly | Borderline hepatosplenomegaly | Improvement |
| 4 | 8.5 | 9.2 | Hepatospenomegaly | No hepatospenomegaly | Improvement |
| 5 | 21.1 | 5.9 | Hepatomegaly, normal spleen | Hepatomegaly, normal spleen | Stationary |
| 6 | 17.7 | 7.3 | Hepatospenomegaly | No hepatospenomegaly | Improvement |
| 7 | 1.4 | 7.0 | No hepatomegaly, borderline enlarged spleen | No hepatosplenomegaly | Improvement |
| 8 | 3.6 | 9.4 | No hepatosplenomegaly | No hepatosplenomegaly | Stationary |
| 9 | 4.4 | 8.0 | No hepatosplenomegaly | No hepatosplenomegaly | Stationary |