Change in fracture rate and healthcare resource utilization among patients with hypophosphatasia following initiation of asfotase alfa: a retrospective US claims database analysis.

ABSTRACT: Hypophosphatasia (HPP) is a rare, inherited, systemic disease characterized by skeletal and systemic manifestations. Asfotase alfa (AA) is the only US Food and Drug Administration-approved treatment for perinatal/infantile- and juvenile-onset HPP. Real-world data are scarce on fracture rates and healthcare resource utilization (HCRU) in patients treated with AA. This retrospective, observational study evaluated fracture-related outcomes and HCRU in patients with HPP treated with AA from January 2016 to December 2022 using the US Medicare Fee-for-Service and Inovalon Medical Outcomes Research for Effectiveness and Economics closed claims databases. Patients (≥2 yr of age) treated with AA (≥1 pharmacy claim for AA between January 2017 and December 2021 [identification period]) and ≥12 mo of

SUBMITTER: Lyons G 

PROVIDER: S-EPMC12771369 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature