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Allogeneic peripheral blood stem cell transplantation in children with hematologic malignancies from HLA-matched
1Department of Pediatrics, University of Tokushima School of Medicine, Tokushima, Japan. twatanab@clin.med.tokushima-u.ac.jp
Insights
Allogeneic peripheral blood stem cell transplantation (PBSCT) is feasible in children, using granulocyte colony-stimulating factor (G-CSF) for stem cell mobilization. This method offers a viable alternative to bone marrow transplantation (BMT) with good patient outcomes.
Area of Science:
- Pediatric Hematology and Oncology
- Stem Cell Transplantation
- Immunology
Background:
- Allogeneic peripheral blood stem cell transplantation (PBSCT) presents potential advantages over allogeneic bone marrow transplantation (BMT).
- Ethical considerations exist regarding granulocyte colony-stimulating factor (G-CSF) use in pediatric donors.
Purpose of the Study:
- To evaluate the feasibility and outcomes of allogeneic PBSCT in pediatric patients with high-risk hematological malignancies.
- To assess the safety and efficacy of G-CSF mobilization and apheresis in pediatric stem cell donors.
Main Methods:
- Eleven pediatric donors (aged 2-16 years) received G-CSF (10 microg/kg/day for 5 days) followed by apheresis for peripheral blood stem cell (PBSC) collection.
- Eleven pediatric patients (aged 8 months to 14 years) with high-risk hematological malignancies received PBSC following a preparative regimen of busulfan and melphalan.
- Graft-versus-host disease (GVHD) prophylaxis involved cyclosporine and methylprednisolone.
Main Results:
- All donors tolerated G-CSF and apheresis. Patients received adequate numbers of CD34+, CFU-GM, and CD3+ cells.
- Prompt engraftment was observed in all patients, with a median time to absolute neutrophil count (ANC) recovery of 10 days.
- Grade I acute GVHD occurred in 64% of patients; no grade II-IV acute GVHD was observed. Chronic GVHD occurred in 40% of evaluable patients.
- At a median follow-up of 775 days, 73% of patients were alive and disease-free.
Conclusions:
- Allogeneic PBSCT is a feasible and effective treatment modality in the pediatric population.
- PBSC harvest via apheresis is a viable alternative to bone marrow harvest for pediatric donors, even with peripheral access.
- The selection of stem cell source should be guided by a thorough risk/benefit assessment for both the patient and the donor.
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).
Procedure:
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.