Japanese Journal of Clinical Oncology 31:30-34 (2001)
© 2001 Foundation for Promotion of Cancer Research
Possible Clinical Benefits of the Use of Peripheral Blood Stem Cells Over Bone Marrow in the Allogeneic Transplantation Setting for the Treatment of Childhood Leukemia

1Pediatric Oncology Division and 2Hematopoietic Stem Cell Transplantation Division, National Cancer Center Hospital, Tokyo, Japan
Background: The benefits of allogeneic peripheral blood stem/progenitor cell transplantation (PBSCT) over bone marrow transplantation (BMT), if any, have not been seriously evaluated in a pediatric population. We report here our experience with this procedure and demonstrate rapid engraftment to reduce procedure-related complications and enhanced allogeneic immune reaction to reduce leukemic relapse.
Methods: The feasibility of PBSCT was reviewed retrospectively. Four patients (2 AML and 2 ALL, aged 818 years) underwent allogeneic PBSCT for relapsed leukemia after primary allogeneic BMT (n = 2), for active hepatosplenic fungal abscess (n = 1) or for refractory relapse with conventional chemotherapy (n = 1). Four healthy donors (aged 1049 years) received granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day by subcutaneous injection for 5 days. An individualized cytoreductive regimen was used before transplantation.
Results: No significant toxicities were observed in normal donors on G-CSF treatment or at collection of PBSC. After PBSCT, no significant acute toxicities were observed and the median duration to an absolute granulocyte count of 0.5 x 109/l and a platelet count of 20 x 109/l was 16 and 21 days, respectively. Although none of our patients developed acute graft-versus-host disease (GVHD), two developed chronic GVHD involving the liver and skin. Among those who developed chronic GVHD, one died of recurrent disease and another died of pneumonia 235 days after PBSCT. The two remaining patients have been alive without evidence of disease with follow-ups of 193 and 123 days, respectively.
Conclusions: Allogeneic PBSCT can be a safe procedure in a pediatric population with fewer acute complications, although the potential risk of G-CSF treatment in normal donors should be seriously weighed against the existing risks of marrow aspiration under general anesthesia. The risk of chronic GVHD may need to be balanced against a possible graft-versus-leukemia benefit in patients at higher risk of leukemic relapse.
+ For reprints and all correspondence: Atsushi Makimoto, Pediatric Oncology Division, National Cancer Center Hospital, 11 Tsukiji 5-Chome, Chuo-ku, Tokyo 104-0045, Japan. E-mail: amakimot@ncc.go.jp
Abbreviations: PBSCT, peripheral blood stem cell transplantation; BMT, bone marrow transplantation; GVHD, graft-versus-host disease; GVL, graft-versus-leukemia; G-CSF, granulocyte colony-stimulating factor; NCCH, National Cancer Center Hospital of Japan; AML, acute myelogenous leukemia; ALL, acute lymphoblastic leukemia; ACD, acid citrate dextrose; BU, busulfan; CY, cyclophosphamide; TBI, total body irradiation; CA, cytarabine; PAM, melphalan; VP-16, etoposide; CsA, cyclosporine A; MTX, methotrexate; AGC, absolute granulocyte counts; FISH, fluorescence in situ hybridization; STR, short tandem repeats; MIT, mitoxantrone; THP, pirarubicin; US, ultrasonography; CR, complete remission; S, skin; Li, liver; Lu, lung