title: High-dose busulfan and melphalan as conditioning regimen for autologous peripheral blood progenitor cell transplantation in high-risk ewing sarcoma patients: a long-term follow-up single-center study.
abstract: The aim of this retrospective study was to analyze the outcome and identify risk factors associated to progression-free survival (PFS) in 47 children with high-risk Ewing sarcoma who underwent autologous peripheral blood stem cell (PBSC) transplantation in the authors' institution between 1995 and 2009. The conditioning regimen used in all patients consisted of high dose of busulfan and melphalan. Median age was 13 years (range: 4-21 years). Forty-three percent of patients had metastases at diagnosis. The probability of transplant-related mortality (TRM) was 6% +/- 3%. Recurrence/progressive disease was observed in 17 patients. The probability of recurrence/progression was 39% +/- 7%. With a median follow-up of 92 months (range: 6-168 months), the PFS was 56% +/- 4% for the whole group. In multivariate analysis, localized disease at diagnosis and obtaining complete remission (CR) by 3 months after transplantation were variables associated to better outcomes. The probability of PFS was 78% +/- 8% and 27% +/- 10% for patients with localized and metastatic disease at diagnosis, respectively (P = .0001). This retrospective study shows a high long-term survival using high dose of busulfan and melphalan as conditioning regimen in children with high-risk Ewing tumors. Patients with localized disease at diagnosis and those with good response to treatment before or after transplant would benefit most.
authors.affiliation: Department of Pediatrics, Division of Pediatric Hematology-Oncology and Hematopoietic Stem Cell Transplantation and Cell Therapy Unit, Hospital Infantil Universitario Niño Jesus, Madrid, Spain. mdiaz.hnjs@salud.madrid.org
authors.forename: Miguel A
authors.initials: MA
authors.lastname: Diaz
authors.forename: Alvaro
authors.initials: A
authors.lastname: Lassaletta
authors.forename: Antonio
authors.initials: A
authors.lastname: Perez
authors.forename: Julian
authors.initials: J
authors.lastname: Sevilla
authors.forename: Luis
authors.initials: L
authors.lastname: Madero
authors.forename: Marta
authors.initials: M
authors.lastname: Gonzalez-Vicent
mesh_terms: D000293:Adolescent; D000328:Adult; D002066:Busulfan / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D019653:Myeloablative Agonists / Q000008:administration & dosage*; D036102:Peripheral Blood Stem Cell Transplantation; D012074:Remission Induction; D012189:Retrospective Studies; D012307:Risk Factors; D012512:Sarcoma, Ewing / Q000175:diagnosis / Q000401:mortality / Q000628:therapy*; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous
chemical_list: D019653:Myeloablative Agonists; D002066:Busulfan; D008558:Melphalan