Advances in therapy
Sirolimus and Immunoglobulin Replacement Therapy in Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS): A Systematic Literature Review
By Hartog NL, Pisarczyk K, Samad M and 2 others.
Abstract
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.
Methods
MEDLINE, Embase, and Cochrane Library databases were searched through July 2025, supplemented by grey literature and bibliographic searches. Eligible records reported treatment outcomes in patients with APDS receiving sirolimus and/or IRT. Quality assessment was performed; findings were narratively synthesized.