Transplant outcomes in bone marrow failure syndromes and hemoglobinopathies.
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only potential cure for most bone marrow (BM) failure syndromes and hemoglobinopathies. Over the past decade, umbilical cord blood (UCB) has been used more frequently as a stem cell source in patients who lack a suitable BM donor. Although graft failure remains a significant problem, UCB transplantation (UCBT) using the optimal conditioning regimen can be a salvage treatment for patients without a suitable BM donor and warrants evaluation in further prospective studies.
MacMillan
Margaret L
ML
Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, University of Minnesota Medical School, Minneapolis, MN 55455, USA. macmi002@umn.edu
Walters
Mark C
MC
Gluckman
Eliane
E
D001771:Blood Banks; D001782:Blood Donors / Q000600:supply & distribution; D001855:Bone Marrow Diseases / Q000601:surgery*; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006453:Hemoglobinopathies / Q000601:surgery*; D006801:Humans; D018579:Patient Selection; D014184:Transplantation, Homologous; D016896:Treatment Outcome