Dental outcomes for children receiving asfotase alfa for hypophosphatasia

Hypophosphatasia, a genetic disease impeding development of teeth and bones, is associated with premature exfoliation of primary teeth. Hypophosphatasia is caused by mutations in the ALPL gene, which encodes the tissue non-specific form of alkaline phosphatase. Asfotase alfa (Strensiq®) is a human recombinant bone-targeted alkaline phosphatase.

To review development and exfoliation patterns of primary/permanent teeth in a cohort of patients with hypophosphatasia enrolled in an open-label clinical trial of enzyme replacement therapy (ERT) with asfotase alfa.

Data were collected from existing study files of a cohort of patients ≤5 years of age with infantile hypophosphatasia. Children were recruited at the Winnipeg site of a global clinical trial and were treated with ERT. Dental information, including the exfoliation/eruption patterns, were recorded at each visit.

Eleven children (7 females, 4 males) participated. Participants enrolled as infants (5 infants; mean age 3.0 ± 2.3 months) prematurely lost significantly fewer teeth to hypophosphatasia than patients recruited as preschoolers (6 preschoolers; mean age 52.5 ± 11.3 months), who started on asfotase alfa at a later age. Conclusion The oral health of children with early onset infantile hypophosphatasia may be improved with early and continued administration of ERT, compared to institution of therapy later in childhood.