New Advance in Stem-Cell Transplants Offers New Cure for Immunodeficiency

High-risk patients with primary immunodeficiency were cured with minimal toxicity.

Although bone-marrow transplantation is a known cure for primary immunodeficiency, some patients cannot tolerate traditional immunosuppression regimens. Investigators in the U.K. examined whether a novel antibody-based, minimal-intensity conditioning regimen (administered before transplant) improved engraftment in 16 high-risk children. The conditioning regimen included two rat anti-CD45 monoclonal antibodies.

The high-risk patients were younger than 1 year at transplant or had preexisting organ injury and could not tolerate traditional high-intensity immunosuppression (including mechanical ventilation for pulmonary infections). Grafts were obtained from peripheral blood and bone marrow. Bone-marrow grafts were associated with better outcome…

Reviewing Author

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)