Fast Five Quiz: Juvenile Hypophosphatasia Management

Eric T. Rush, MD, FAAP, FACMG


July 22, 2022

For children with HPP, the efficacy of treatment using asfotase alfa is assessed according to improvement in clinical symptoms, including motor function, bones symptoms, growth, and mental or motor development. The 6-minute walk test may be useful to assess endurance.

Patients with childhood HPP typically lack life-threatening symptoms. Pulmonary insufficiency is typically a symptom of infantile HPP.

A measurement of ALP activity is essential for the diagnosis of HPP and may be helpful in monitoring compliance with asfotase alfa. Currently, no biomarkers reliably assess the efficacy of enzyme replacement, according to Tournis and colleagues.

Learn more about HPP.


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