M. Bonno et al., SUCCESSFUL HEMATOPOIETIC RECONSTITUTION BY TRANSPLANTATION OF UMBILICAL-CORD BLOOD-CELLS IN A TRANSFUSION-DEPENDENT CHILD WITH DIAMOND-BLACKFAN ANEMIA, Bone marrow transplantation, 19(1), 1997, pp. 83-85
A 4-year-old boy with Diamond-Blackfan anemia and a history of multipl
e transfusions underwent umbilical cord blood transplantation from his
HLA-identical female sibling born by vaginal delivery at 38 weeks, Th
e patient was prepared,vith busulfan, cyclophosphamide and antilymphoc
yte globulin, Methotrexate and cyclosporin A were given for the prophy
laxis of GVHD, Regimen-related toxicity was not observed and successfu
l engraftment occurred, including the erythroid series. No evidence of
acute or chronic GVHD has been observed for 14 months after transplan
tation. This is the first case of successful umbilical cord blood tran
splantation to a patient with Diamond-Blackfan anemia.