Management and treatment of APDS vary from patient to patient, even within
affected families, and depends on the symptoms present and disease severity.¹
Symptom Management ²
Antibiotics and Antivirals:
Antibiotics and antivirals are often given to treat or prevent recurrent infections.
Immunoglobulin Replacement Therapy:
Majority of APDS patients have antibody deficiencies and reeive immunoglobulin replacement therapy (IRT) given subcutaneously or intravenously.
Rituximab is one of many immuno-suppresive treatments used for autoimmune complications.
Current Treatments Under Investigation ²
Stem Cell Transplantation:
For the most severe patients with life threatening complications hematopoietic stem cell transplantation (HSCT) has been used to correct the underlying dysfunction though with a high benefit risk consideration.
Selective PI3K-delta inhibitors are currently being studied in clinical trials and have the potential in the future to offer a targeted treatment option for APDS patients.
Leniosilib (CDZ173) an investigational drug not yet approved by the US Food and Drug Administration (FDA), is an oral selective PI3K-delta inhibitor currently under investigation for the treatment of APDS.
1. Condliffe AM, Chandra A. Respiratory Manifestations of the Activated Phosphoinositide 3-Kinase Delta Syndrome. Front Immunol. 2018;9:338. Published 2018 Mar 5. doi:10.3389/fimmu.2018.00338
2. Coulter TI, Cant AJ. The Treatment of Activated PI3Kδ Syndrome. Front Immunol. 2018;9:2043. Published 2018 Sep 7. doi:10.3389/fimmu.2018.02043