Allogeneic peripheral blood stem cell transplantation (PBSCT) from HLA-identical sibling donors in children with hematological diseases: a single center pilot study

Between February 1995 and July 1999 25 pediatric patients (8 months to 14 years old) underwent peripheral blood stem cell transplantation (PBSCT) from an HLA-identical sibling donor. Diagnoses included ALL (17), non-ALL (6), and non-malignant disease (2). GVHD prophylaxis consisted of cyclosporine plus methotrexate (15), only cyclosporine (8), cyclosporine plus prednisone (1), or nothing (1). All donors (6 months to 41 years old) received G-CSF at 10 μg/kg/day subcutaneously for 4–5 days and on day 5 underwent large volume leukapheresis. Median number of CD34+ and CD3+ cells collected and infused was 6.9 × 106 (range 2.5–32.8) and 4.5 × 108 (0.5–22.1) per kg of recipient body weight respectively. Median time to achieve ANC >0.5 × 109/l and platelets >20 × 109/l was 10 and 12 days, respectively. Acute GVHD grade ⩾II developed in 10 of 24 evaluable patients (42%). Probability of acute GVHD was 62%. Median time to discharge was 25 days (range 14–52). Among 20 evaluable patients, five (25%) developed chronic GVHD at day 100. Probability of chronic GVHD was 29% after 1 year post PBSC. At a median follow-up of 558 (9–2071) days, overall survival for the whole group is 68%. Probabilities of event-free survival, overall survival and relapse for patients with malignant hematological diseases are 53%, 59% and 24% at 5 years, respectively. This study has confirmed the feasibility and safety of mobilization and collection of PBSC products and the applicability of this procedure to the pediatric population, both donors and recipients. Studies including larger numbers of pediatric patients undergoing allogeneic PBSCT are warranted to determine the long-term outcomes of such procedures. Bone Marrow Transplantation (2001) 28, 537–543.