Primary immunodeficiencies (PIDs) are a diverse category of hereditary diseases that impair the immune system’s development and/or function. This review focuses on recent advancements in hematopoietic stem cell transplantation (HSCT) for PIDs, as it is the only possible curative option for many of these illnesses. We present the most current HSCT European results and discuss some potential therapeutic options for specific PIDs. Gene therapy progress is also highlighted since it appears to be an intriguing alternative for PID treatment.
Progress in the treatment of primary immune deficiency with HSCT necessitates a deeper understanding of the pathophysiology and uniqueness of each of these illnesses, allowing us to choose the optimal donor, conditioning regimen, allograft modification, and immunosuppressive medication. For some PIDs, alternative treatments, such as gene therapy, are emerging as an intriguing possibility.