Table 3.
Monitoring of patients with Hunter syndrome aged ≥5 years receiving ERT. Adapted from (Wraith et al., 2008b; Muenzer et al., 2009; Guelbert et al., 2011).
| Organ system/involvement | Assessment | Recommendationa |
|---|---|---|
| Medical history | Clinical evaluation, including developmental milestones | Every 6 mo |
| Physical examination | Clinical evaluation, including height, weight, head circumference, BP, | Every 6 mo |
| neurological examination | ||
| Infections/surgeries | Clinical evaluation | Every 6 mo |
| Neurological | Cognitive assessment | Every 12 mo |
| Cardiovascular | Echocardiogram, ECG | Every 12 mo |
| Pulmonary | Spirometry | Every 12 mo |
| Musculoskeletal | JROM | Every 12 mo |
| 6MWT | Every 6 mo | |
| General | ERT status: start date, dosage, any missed infusions | Every 6 mo |
| uGAG level | Every 6 mo | |
| Antibody testing | Prior to ERT start, then every 6 mo |
a
Conduct upon enrollment, and monitor thereafter, as indicated.
6MWT, 6-minute walk test; BP, blood pressure; ECG, electrocardiogram; ERT, enzyme replacement therapy; uGAG, urinary glycosaminoglycan; JROM, joint range of motion.