Cord blood stem cell transplantation for Diamond-Blackfan anemia.

Bone marrow transplantation has been successfully employed in the treatment of Diamond-Blackfan anemia (DBA). Transplantation with bone marrow stem cells involves, however, considerable treatment-related toxicity, partially in the form of GVHD. Through the use of cord blood stem cells it may be possible to reduce the risk for both acute and chronic GVHD, although possibly in the face of an increased risk for non-engraftment. We report the successful treatment of a boy with DBA by cord blood stem transplantation from an HLA-identical sibling.