Homozygous α-Thalassemia Treated with Intrauterine Transfusions and Unrelated Donor Hematopoietic Cell Transplantation
Recently, intrauterine transfusions and hematopoietic cell transplantation (HCT) have changed homozygous alpha-thalassemia from a frequently fatal disease to a potentially survivable condition. We present a patient with Hemoglobin Bart's disease who was cured after failing to engraft with 1 unrelated HCT, but engrafting after a second unrelated donor HCT.
Abbreviations: ATG, Anti-thymocyte globulin, BMT, Bone marrow transplant, GVHD, Graft versus host disease, Hb, Hemoglobin, HCT, Hematopoietic cell transplantation, HLA, Human leukocyte antigen, TBI, Total body irradiation, TLI, Total lymphoid irradiation
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The authors declare no conflicts of interest.
PII: S0022-3476(08)01040-8
doi:10.1016/j.jpeds.2008.11.031
© 2009 Mosby, Inc. All rights reserved.
