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.
aDivision of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, University of Minnesota Medical School, Minneapolis, MN
bChildren's Hospital and Research Center, Oakland, CA
cHospital Saint Louis and University Paris VII, Paris, France
Address correspondence to Margaret L. MacMillan, MD, Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, University of Minnesota Medical School, Mayo Mail Code 484, D-557 Mayo, Minneapolis, MN 55455
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