Successful mismatched sibling cord blood transplant in Hb Bart's disease

• Published in: Bone marrow transplantation (Basingstoke) Vol. 28; no. 1; pp. 105 - 107
• Main Authors: ZHOU, X, HA, S. Y, CHAN, G. C. F, LUK, C. W, CHAN, V, HAWKINS, B, LAM, Y. H, LIANG, R. H. S, LAU, Y. L
• Format: Journal Article
• Language: English
• Published: Basingstoke Nature Publishing Group 01.07.2001
• Subjects: alpha-Thalassemia - blood; alpha-Thalassemia - complications; alpha-Thalassemia - therapy; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Biological and medical sciences; Bone marrow, stem cells transplantation. Graft versus host reaction; Female; Fetal Blood; Graft Survival; Hematopoietic Stem Cell Transplantation - adverse effects; Hematopoietic Stem Cell Transplantation - methods; Hemoglobinopathies - blood; Hemoglobinopathies - complications; Hemoglobinopathies - therapy; Hemoglobins, Abnormal; Histocompatibility - immunology; HLA-DR Antigens; Humans; Infant; Medical sciences; Nuclear Family; Purpura, Thrombocytopenic, Idiopathic - drug therapy; Purpura, Thrombocytopenic, Idiopathic - etiology; Stem cell transplantation; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transplantation, Homologous - adverse effects; Transplantation, Homologous - immunology; Transplantation, Homologous - methods; Human; Incompatibility; Hemoglobinopathy; HLA-System; Stem cell; Major histocompatibility system; Class II histocompatibility antigen; Related donor; Hematopoietic cell; Homograft; Infant; Hemopathy; Blood; Genetic disease; Case study; Hemolytic anemia; Treatment; α-Thalassemia; Female; Graft; Umbilical cord
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/sj.bmt.1703104

A 20-month-old girl with Hb Bart's disease, who had survived neonatal complications, underwent HLA-DR antigen mismatched sibling cord blood transplantation successfully. Immune thrombocytopenia, which occurred around 2.5 months after transplant, responded to intravenous gamma-globulin. The fetal hemoglobin level rose to a peak of 52.3% on day +69 post transplant and declined gradually during the following year. Ten percent of hemoglobin Bart's was detected 2 months after transplant and this reflects the alpha-thalassemia trait of the donor.