Alkaline phosphatase (ALP) enzyme replacement therapy, asfotase alfa, is indicated in children with HPP who display evidence of rickets-like changes, which suggest an underlying defect in bone mineralization. These changes may predispose children to fractures and developmental delays. Radiographic changes of rickets include metaphyseal widening and fraying, bowing of the long bones, and generalized hypomineralization. Asfotase alfa has been shown to significantly improve gross motor, fine motor, and cognitive development in patients with childhood HPP. In addition, improvement in physical function and pain levels was observed, allowing patients the ability to perform daily activities.
Increased PLP is present in most patients diagnosed with HPP. However, HPP displays variability in the initial clinical presentation. For patients with mild or no symptoms of childhood HPP, conservative management with periodic follow-up is recommended. At the onset of functional limitation, the initiation of enzyme replacement therapy may be indicated.
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Cite this: Eric T. Rush. Fast Five Quiz: Juvenile Hypophosphatasia Management - Medscape - Jul 22, 2022.