Advances in hematopoietic stem cell transplantation for primary immunodeficiency

Abstract

The transplantation of hematopoietic stem cells in patients with primary immunodeficiencies has improved significantly over the last 40 years. In favorable circumstances when there is minimal or no infection present, no end-organ damage and the availability of a well HLA-matched donor, survival and cure reaches 90%. Barriers to further success include late identification of disease, with accumulation of infection- and inflammation-related organ damage, stem cell manipulation when there is no HLA-matched donor, toxicity of conditioning regimens and prediction and treatment of graft-versus-host disease. This review will outline recent developments in conditioning regimens, stem cell source manipulation and early detection and treatment of graft-versus-host disease, with a particular emphasis on patients with primary immunodeficiency.

Keywords::

• extracorporeal photopheresis
• graft-versus-host disease
• mesenchymal stromal cells
• newborn screening for severe combined immunodeficiency
• primary immunodeficiency
• reduced intensity conditioning
• severe combined immunodeficiency
• targeted busulfan
• treosulfan

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending or royalties.

No writing assistance was utilized in the production of this manuscript.