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:
The majority of people with APDS have antibody deficiencies and receive immunoglobulin replacement therapy (IRT) given subcutaneously or intravenously.
Rituximab is one of many immunosuppressive treatments used for autoimmune complications.
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 risk – benefit 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.
Leniolisib (CDZ173) – an investigational drug not yet approved by world health authorities, 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