title: Autologous bone marrow transplantation with 4-hydroperoxycyclophosphamide purged marrows for children with acute non-lymphoblastic leukemia in second remission.
abstract: Although autologous bone marrow transplantation (ABMT) following purging with 4-hydroperoxycyclophosphamide (4HC) has been effective for some adults with acute non-lymphoblastic leukemia (ANLL), there are few data on ABMT for children. We have performed ABMT for 13 patients (median age, 5 years) with ANLL in second remission. Bone marrow was treated ex vivo with 4HC to kill residual leukemic cells. In order to enhance the anti-leukemic effect of 4HC, exogenous red blood cells were eliminated from the marrow/4HC mixture. All patients were conditioned for transplantation with busulfan (16 mg/kg) and cyclophosphamide (200 mg/kg), followed by infusion of the 4HC treated marrow. Eleven of 13 patients had complete hematologic reconstitution; there were no transplant-related deaths. Five patients relapsed at 2, 4, 5, 6 and 6 months post-BMT. Eight patients are disease-free survivors; the median time for survival is 24 months, with a projected disease-free survival of 61%. ABMT is a safe and efficacious treatment modality for children with ANLL in second remission. Our results suggest that the disease-free survival for pediatric patients may be superior to that seen in adults.
authors.affiliation: Division of Research Immunology/Bone Marrow Transplantation, Children's Hospital of Los Angeles, University of Southern California School of Medicine 90027.
authors.forename: C
authors.initials: C
authors.lastname: Lenarsky
authors.forename: K
authors.initials: K
authors.lastname: Weinberg
authors.forename: J
authors.initials: J
authors.lastname: Petersen
authors.forename: J
authors.initials: J
authors.lastname: Nolta
authors.forename: J
authors.initials: J
authors.lastname: Brooks
authors.forename: G
authors.initials: G
authors.lastname: Annett
authors.forename: D
authors.initials: D
authors.lastname: Kohn
authors.forename: R
authors.initials: R
authors.lastname: Parkman
mesh_terms: D000206:Actuarial Analysis; D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation / Q000379:methods; D002066:Busulfan / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000494:pharmacology / Q000627:therapeutic use; D005069:Evaluation Studies as Topic; D006085:Graft Survival; D006801:Humans; D007223:Infant; D015470:Leukemia, Myeloid, Acute / Q000473:pathology / Q000601:surgery*; D014411:Neoplastic Stem Cells / Q000187:drug effects*; D012074:Remission Induction; D014182:Transplantation, Autologous
chemical_list: D003520:Cyclophosphamide; D002066:Busulfan; C011272:perfosfamide