Allogeneic hematopoietic stem cell transplantation for childhood aplastic anemia: prospective trial in China.
We aim to investigate the efficacy and safety of the treatment with fully matched allogeneic hematopoietic stem cell transplants for children with severe aplastic anemia (SAA) in the first prospective trial in China. Six SAA children received allogeneic hematopoietic stem cell transplantation combined with chemotherapy. Five patients had successful engraftment while the sixth child regained normal peripheral blood counts consistent with spontaneous autologous hematopoiesis. Mean duration of follow-up was 2.75 years, and survival was 83%(5/6). The results indicated that allogeneic hematopoietic stem cell transplantation is a good option for the treatment of children with severe aplastic anemia.
Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou 510120, China. chenchun69@126.com
Chen
C
Chun
Jian-pei
JP
Fang
Hong-man
HM
Xue
Dun-hua
DH
Zhou
Ke
K
Huang
Hong-gui
HG
Xu
Shao-liang
SL
Huang
D000293:Adolescent; D000741:Anemia, Aplastic / Q000188:drug therapy / Q000601:surgery*; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000379:methods / Q000706:statistics & numerical data; D002470:Cell Survival; D002648:Child; D002675:Child, Preschool; D002681:China; D003131:Combined Modality Therapy; D016572:Cyclosporine / Q000627:therapeutic use; D005260:Female; D000069585:Filgrastim; D005500:Follow-Up Studies; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000517:prevention & control; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use; D019650:Hematopoietic Stem Cell Mobilization; D006650:Histocompatibility Testing; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D019520:Living Donors; D008297:Male; D008727:Methotrexate / Q000627:therapeutic use; D019653:Myeloablative Agonists / Q000627:therapeutic use; D036102:Peripheral Blood Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods / Q000706:statistics & numerical data; D011994:Recombinant Proteins; D035781:Siblings; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome
D007166:Immunosuppressive Agents; D019653:Myeloablative Agonists; D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor; D016572:Cyclosporine; D000069585:Filgrastim; D008727:Methotrexate