Abstract
Objectives:
To provide an overview of
drug treatment,
transplantation, and
gene therapy for
patients with primary immunodeficiencies. Source of data Non-
systematic review of the
literature in the English
language carried out at
PubMed. Synthesis of data The
treatment of
patients with primary immunodeficiencies aims to control their
disease, especially the
treatment and prevention of
infections through
antibiotic prophylaxis and/or
immunoglobulin replacement
therapy. In several
diseases, it is possible to use specific medications for the affected pathway with control of the condition, especially in autoimmune or autoinflammatory processes associated with inborn
immunity errors. In some
diseases,
treatment can be curative through
hematopoietic stem cell transplantation (HSCT); more recently,
gene therapy has opened new horizons through new
technologies.
Conclusions:
Immunoglobulin replacement
therapy remains the main
therapeutic tool.
Precision medicine with specific
drugs for altered immune pathways is already a reality for several immune defects. Advances in the management of HSCT and
gene therapy have expanded the capacity for curative
treatments in
patients with primary immunodeficiencies.