Cytokine-mobilized allogeneic peripheral blood stem cell transplants in children result in rapid engraftment and a high incidence of chronic GVHD

Between October 1995 and October 1998, 24 children aged 9 months to 17 year s (median 11 years) underwent cytokine-mobilized allogeneic peripheral bloo d stem cell (PBSC) transplantation for treatment of hematological disorders . All of the transplants were the first allogeneic transplant for the recip ient. Twenty patients were transplanted for hematological malignancies (ALL = 8, AML = 6, CML = 4, MDS = 2) and four patients were transplanted for no n-malignant disease (thalassemia major = 2, Wiskott-Aldrich syndrome = 1, K ostmann's syndrome = 1). Nineteen donors were HLA-identical siblings, four were HLA-matched or single antigen mismatched parents, and one was a syngen eic transplant. Donors aged 8 to 38 years (median 15 years, 14 donors <18 y ears) received G-CSF 10 mu g/kg/day subcutaneously beginning 4 days before PBSC collection and were submitted to one to three leukapheresis collection s. The median CD34(+) cell yield was 7.8 x 10(6) cells/kg recipient body we ight. All patients achieved an ANC >0.5 x 10(9)/l after a median of 13 days (range 10-21), Twenty-three patients eventually achieved platelet transfus ion independence. One patient died on day 63 without ever achieving platele t transfusion independence. Four patients received platelet transfusions to maintain a platelet count well above 20 x 10(9)/l due to bleeding complica tions. Of the 19 evaluable patients, the median time to a non-transfused pl atelet count of 20 x 10(9)/l was 12 days (range 0-44), Ten of 23 at-risk pa tients developed acute GVHD grades II to IV, with grades III to IV in four patients. Twelve of 19 patients followed for at least 100 days have develop ed chronic GVHD (extensive = 2, limited = 10) with an actuarial risk of chr onic GVHD of 75% at 1 year. The Kaplan-Meier estimate of event-free surviva l is 65% at 2 years. Four patients died (GVHD = 3, VOD = 1), three patients relapsed, and one patient with thalassemia major had a late graft failure with autologous recovery. Based upon our experience, allogeneic PBSCT is sa fe for both pediatric donors and recipients and engraftment of neutrophils and platelets is rapid.