title: [Hematopoietic stem cell transplantation with busulfanthiotepa-cyclophosphamide conditioning for pediatric patients with high-risk acute lymphoblastic leukemia].
abstract: Twelve children with high-risk acute lymphoblastic leukemia underwent stem cell transplantation (SCT) with a conditioning regimen consisting of busulfan, cyclophosphamide and thiotepa. Eight of them underwent SCT while in complete remission (CR) and the other 4 while not in CR. Three children underwent HLA-matched related bone marrow transplantation (BMT), 7 HLA-matched unrelated BMT, 1 HLA one-locus-mismatched unrelated cord blood cell transplantation, and 1 autologous peripheral blood stem cell transplantation. Grade II-IV acute GVHD was observed in 3 of the 11 allo-SCT cases, while chronic GVHD was seen in 3 of 9 evaluable cases. None of the 12 cases showed thrombotic microangiopathy, and veno-occlusive disease (VOD) was observed in 3. Nine of the patients are alive and disease-free 6-45 months after diagnosis. The event-free survival rate at 3 years was 72.2% for the 12 patients, including 8 of the 9 who received SCT during CR, and 2 of the 4 who did so while not in CR. The other 3 patients died: 2 of disease progression and 1 of VOD with pneumonia. All of those who died had undergone unrelated BMT.
authors.affiliation: Department of Pediatrics, Graduate School of Medicine, Kyoto University.
authors.forename: K
authors.initials: K
authors.lastname: Umeda
authors.forename: Y W
authors.initials: YW
authors.lastname: Lin
authors.forename: K
authors.initials: K
authors.lastname: Watanabe
authors.forename: S
authors.initials: S
authors.lastname: Adachi
authors.forename: I
authors.initials: I
authors.lastname: Usami
authors.forename: Y
authors.initials: Y
authors.lastname: Akiyama
authors.forename: M
authors.initials: M
authors.lastname: Kubota
authors.forename: T
authors.initials: T
authors.lastname: Nakahata
mesh_terms: D000293:Adolescent; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage*; D002066:Busulfan / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage*; D018572:Disease-Free Survival; D004359:Drug Therapy, Combination; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000628:therapy*; D012306:Risk; D013852:Thiotepa / Q000008:administration & dosage*; D019172:Transplantation Conditioning; D016896:Treatment Outcome
chemical_list: D018906:Antineoplastic Agents, Alkylating; D003520:Cyclophosphamide; D013852:Thiotepa; D002066:Busulfan