title: Intensified conditioning regimen with busulfan followed by allogeneic BMT in children with myelodysplastic syndromes.
abstract: Four consecutive children with myelodysplastic syndromes (MDS) underwent matched allogeneic bone marrow transplantation (BMT). Ages ranged from 3.2 to 6.3 years. Diagnosis was assessed according to FAB classification: refractory anemia-RA (n = 1), RA with excess of blasts (RAEB) (n = 1), and juvenile chronic myelogenous leukemia (JCML) (n = 2). Initial treatment included transfusions for all of them, splenectomy (n = 2) and chemotherapy (n = 1). Patients were all prepared with busulfan 21 mg/kg (480 mg/m2), cytosine arabinoside 24,000 mg/m2, melphalan 140 mg/m2. Graft-versus-host disease (GVHD) prophylaxis associated cyclosporine-methotrexate. Engraftment was prompt and complete in all children. Toxicity included severe mucositis (n = 3), moderate veno-occlusive disease (n = 2), acute GVHD (n = 3), chronic GVHD (n = 1). Sequelae have not yet been seen. All patients are alive and disease-free with a follow-up ranging from 7 to 35 months, with a Karnofsky score of 90-100%. Combined busulphan conditioning can offer an alternative to total body irradiation-based regimens in order to avoid late side-effects in children.
authors.affiliation: Bone Marrow Transplant Unit, CHU Purpan, Toulouse, France.
authors.forename: H
authors.initials: H
authors.lastname: Rubie
authors.forename: M
authors.initials: M
authors.lastname: Attal
authors.forename: C
authors.initials: C
authors.lastname: Demur
authors.forename: P
authors.initials: P
authors.lastname: Brousset
authors.forename: E
authors.initials: E
authors.lastname: Duchayne
authors.forename: F
authors.initials: F
authors.lastname: Rigal-Huguet
authors.forename: N
authors.initials: N
authors.lastname: Dastugue
authors.forename: A
authors.initials: A
authors.lastname: Robert
mesh_terms: D000753:Anemia, Refractory / Q000628:therapy; D016026:Bone Marrow Transplantation; D002066:Busulfan / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D016572:Cyclosporine / Q000627:therapeutic use; D003561:Cytarabine / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D008297:Male; D008558:Melphalan / Q000627:therapeutic use; D008727:Methotrexate / Q000627:therapeutic use; D009190:Myelodysplastic Syndromes / Q000628:therapy*; D014184:Transplantation, Homologous; D014916:Whole-Body Irradiation
chemical_list: D003561:Cytarabine; D016572:Cyclosporine; D002066:Busulfan; D008558:Melphalan; D008727:Methotrexate