Table 1.
Monitoring Recommendations for Mucopolysaccharidosis Type IVA Patients
At Diagnosis | Every 6 Mo | Annually | Every 1–3 Yr |
---|---|---|---|
Medical history | Medical history | Eye examination | Electrocardiogram |
Physical examination | Physical examination | Hearing test | Echocardiogram |
Growth evaluation | Growth evaluation | Pulmonary function | Spine MRI |
Neurological examination | Neurological examination | Electrocardiogram | Sleep study |
Eye examination | Dental evaluation | Echocardiogram | |
Hearing test | Endurance test | ||
Pulmonary function | Quality of life and pain assessment | ||
Sleep study | Cervical spine imaging | ||
Electrocardiogram | |||
Echocardiogram | |||
Dental evaluation | |||
Endurance test | |||
Quality of life and pain assessment | |||
Functional/ADL assessment | |||
Hip/pelvis imaging | |||
Whole Spine imaging | |||
Spine MRI |
Notes: Reproduced with permission from Dove Medical Press, Regier DS, Tanpaiboon P. Role of elosulfase alfa in mucopolysaccharidosis IVA. Appl Clin Genet. 2016;9:67–74.35
Abbreviations: mo, months; yr, years; ADL, activities of daily living.