Systematic literature reviews to identify epidemiological, clinical, economic and health-related quality of life evidence in activated PI3Kδ syndrome (APDS).

ABSTRACT:

Background

Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) is an ultra-rare inborn error of immunity, characterised by immunodeficiency and immune dysregulation. Having only been recognised in 2013, evidence on APDS is limited. We carried out four systematic literature reviews (SLRs) to identify and narratively synthesise evidence on the following for APDS: epidemiology (epidemiology SLR), clinical efficacy/safety of treatments (clinical SLR), cost-effectiveness and costs/healthcare (HCRU) associated with (economic SLR) and health-related quality of life (HRQoL) and utility data (HRQoL SLR) from a global perspective.

Methods

The Cochrane Collaboration and the University of York's Centre for Reviews and Dissemination (CRD) guidelines were followed. MEDLINE,

SUBMITTER: Vlachopoulou K 

PROVIDER: S-EPMC12275335 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature