Estimated annual direct medical costs of manifestations among patients with activated phosphoinositide 3-kinase delta syndrome.

ABSTRACT: Activated phosphoinositide 3-kinase delta syndrome (APDS) is a rare genetic disease associated with heterogeneous manifestations, including recurrent infections, lymphoproliferation, and autoimmunity. This analysis sought to estimate the mean annual direct medical costs of manifestations associated with APDS. A burden-of-illness cost calculator was developed based on survey responses from the US clinical experts, published evidence, and the US cost sources (2023). Results from the survey provided estimates of mean annual prevalence of various manifestations associated with APDS and the mean number of times patients experienced recurrent manifestations in a year. In the base case analysis, the annual mean cost of manifestations associated with APDS per patient in the US was estimated to be

SUBMITTER: Hartog NL 

PROVIDER: S-EPMC12255542 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature