Umbilical cord blood transplantation in Chinese children with beta-thalassemia.
To evaluate factors affecting outcome of sibling umbilical cord blood transplantation in Chinese children with thalassemia. The authors conducted a retrospective review of all patients undergoing such transplants in a single institution. Nine children with thalassemia major were diagnosed at a median age of 12 months. They received irregular blood transfusions and suboptimal iron chelation therapy before transplant. Sibling cord blood transplant was performed at a median of 5.5 years (range 3.5-10 years). Six donors were HLA-identical; three were one- to three-antigen mismatched. The mean number of nucleated cells infused was 6.6 x 10(7)/kg (range 3.4-12.7); the mean number of CD34+ cells infused was 3.8 x 10(5)kg (range 0.6-11.7). Seven patients had engraftment of donor cells. The median number of days to achieve a neutrophil count of > 0.5 x 10(9)/L was 19 days (range 10-25); the median number of days to achieve a platelet count of > 20 x 10(9)/L was 33 days (range 19-63). Of the six patients who received HLA-identical transplants, one developed grade 2 and two developed grade 1 acute graft-versus-host disease. Two of the three patients receiving mismatched cord blood did not achieve engraftment, and the other one engrafted but developed grade 4 acute graft-versus-host disease. Two patients subsequently developed secondary graft rejection and had autologous marrow regeneration before day 60 posttransplantation. With a median follow-up of 49 months (range 38-64), eight patients survived but only four were transfusion-independent. Umbilical cord blood transplant appears to have a higher chance of nonengraftment and secondary rejection. A more intensive immunosuppressive conditioning regimen may be required.
Fang
Jianpei
J
Department of Pediatrics, The Second Affiliated Hospital of Zhongshan University, No. 107, Yan Jiang Road West, Guang-zhou, People's Republic of China. infor@gdtc.net
Huang
Shaoliang
S
Chen
Chun
C
Zhou
Dunhua
D
Li
Chi-Kong
CK
Li
Yang
Y
Huang
Ke
K
D000293:Adolescent; D001803:Blood Transfusion; D002648:Child; D002681:China; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods*; D003520:Cyclophosphamide / Q000627:therapeutic use; D006084:Graft Rejection / Q000453:epidemiology; D006086:Graft vs Host Disease / Q000276:immunology / Q000517:prevention & control; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D012189:Retrospective Studies; D014019:Tissue Donors / Q000706:statistics & numerical data; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000628:therapy*
D007166:Immunosuppressive Agents; D003520:Cyclophosphamide