Sirolimus and Immunoglobulin Replacement Therapy in Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS): A Systematic Literature Review

Introduction

Activated phosphoinositide 3-kinase delta syndrome (APDS) is an ultra-rare disease associated with immunodeficiency and immune dysregulation. Management may include sirolimus and immunoglobulin replacement therapy (IRT); neither is specifically approved for APDS in the USA. This systematic literature review evaluated the effectiveness and safety of sirolimus and IRT in patients with APDS.