title: Allogeneic bone marrow transplant in pediatric patients with high-risk hematopoietic malignancies early in the course of their disease.
abstract: OBJECTIVE
  The purpose of this study was to investigate the role of bone marrow transplant (BMT) early in the course of disease for pediatric patients with high-risk leukemia using a preparatory regimen of fractionated total body irradiation (FTBI) and etoposide (VP-16).
  
  
  METHODS
  Those studied were 33 patients aged < or =18 years with either acute leukemia in first complete remission (CR) (n = 29) or chronic myelogenous leukemia (CML) in first chronic phase (n = 4) who received 1,320 cGy FTBI followed by high-dose VP-16 (60 mg/kg) as a preparatory regimen for BMT from matched sibling donors. Patients with acute leukemia included 18 with acute nonlymphocytic leukemia (ANLL), one with biphenotypic acute leukemia (BAL), and 10 with selected "high-risk" acute lymphocytic leukemia (ALL). Patients with ALL were selected for a high risk for recurrence: those who failed standard remission induction chemotherapy, had a t(9;22) or t(4;11) chromosomal translocation, or had certain clinical high-risk features.
  
  
  RESULTS
  At the time of analysis, 28 patients are alive, all of them in continued complete remission for 1.1-7.8 years (median, 5.3 years; mean, 4.9 years). The Kaplan-Meier projected event-free survival (EFS) is 84.5% at 7 years, and the actuarial recurrence hazard is 6.5%. All surviving patients have a performance status of >80%.
  
  
  CONCLUSIONS
  This result of early BMT in a two-institution study of pediatric patients with hematopoietic malignancies suggests that (a) matched sibling allogeneic BMT after conditioning with FTBI and high-dose VP-16 is an excellent treatment for pediatric patients with high-risk leukemia, and (b) children may have a better prognosis than adults treated with allogeneic BMT. Larger multiinstitutional cooperative trials for pediatric patients are needed to confirm this result.
authors.affiliation: Department of Pediatrics, Stanford University Medical Center, California 94305-5119, U.S.A.
authors.forename: M D
authors.initials: MD
authors.lastname: Amylon
authors.forename: J P
authors.initials: JP
authors.lastname: Co
authors.forename: D S
authors.initials: DS
authors.lastname: Snyder
authors.forename: S S
authors.initials: SS
authors.lastname: Donaldson
authors.forename: K G
authors.initials: KG
authors.lastname: Blume
authors.forename: S J
authors.initials: SJ
authors.lastname: Forman
mesh_terms: D000293:Adolescent; D000328:Adult; D000972:Antineoplastic Agents, Phytogenic / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005047:Etoposide / Q000627:therapeutic use*; D005260:Female; D019337:Hematologic Neoplasms / Q000503:physiopathology / Q000628:therapy*; D006801:Humans; D007223:Infant; D008297:Male; D013997:Time Factors; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D000972:Antineoplastic Agents, Phytogenic; D005047:Etoposide