Allogeneic peripheral blood stem cell transplantation in children with hematologic malignancies from HLA-matched siblings.
BACKGROUND
Despite the ethical problem of using granulocyte colony-stimulating factor (G-CSF) in normal children, allogeneic peripheral blood stem cell transplantation (PBSCT) might have advantages over allogeneic bone marrow transplantation (BMT).


METHODS
Eleven HLA-matched sibling donors aged 2-16 years received 10 microg/kg/day G-CSF for 5 days and underwent apheresis to harvest peripheral blood stem cells (PBSC). PBSC were then cryopreserved until infusion. The 11 corresponding patients aged 8 months to 14 years with high-risk hematological malignancies received busulfan (16 mg/kg or 600 mg/m(2)) and melphalan (210 mg/m(2)) as a preparative regimen. Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine and methylprednisolone.


RESULTS
All of the donors tolerated G-CSF administration and apheresis procedures. The patients received a median of 5.8 (range 1. 4-11.5) x 10(6)/kg CD34(+) cells, 17.2 (3.8-36.0) x 10(5)/kg colony forming units-granulocyte/macrophage (CFU-GM), and 3.5 (1.4-7.1) x 10(8)/kg CD3(+) cells. All of the patients showed prompt engraftment, with a median time to reach an absolute neutrophil count (ANC) above 0.5 x 10(9)/liter of 10 (9-13) days. Grade I acute GVHD occurred in seven patients (64%), whereas grade II-IV acute GVHD was not seen. Chronic GVHD occurred in four patients (40%) among 10 patients evaluable for chronic GVHD. Three patients showed extensive chronic GVHD. Currently, eight patients (73%) are alive and disease-free for a median follow-up of 775 (103-1,069) days.


CONCLUSIONS
Allogeneic PBSCT is feasible in the pediatric population, and PBSC harvest is an alternative to BM harvest in donors who are not eligible for BM harvest. Furthermore, PBSC were successfully collected in pediatric donors with peripheral access. The choice of a stem cell source should be based on the risk/benefit assessment for both patients and donors.
Watanabe
T
T
Department of Pediatrics, University of Tokushima School of Medicine, Tokushima, Japan. twatanab@clin.med.tokushima-u.ac.jp
Kajiume
T
T
Abe
T
T
Kawano
Y
Y
Iwai
A
A
Iwai
T
T
Takaue
Y
Y
Kuroda
Y
Y
D000293:Adolescent; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D002066:Busulfan / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage; D006680:HLA Antigens / Q000276:immunology*; D019337:Hematologic Neoplasms / Q000276:immunology* / Q000628:therapy*; D019650:Hematopoietic Stem Cell Mobilization / Q000379:methods; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D009679:Nuclear Family; D014184:Transplantation, Homologous; D016896:Treatment Outcome
D018906:Antineoplastic Agents, Alkylating; D006680:HLA Antigens; D007166:Immunosuppressive Agents; D016179:Granulocyte Colony-Stimulating Factor; D002066:Busulfan; D008558:Melphalan