title: High-dose busulfan and melphalan as conditioning regimen for autologous peripheral blood progenitor cell transplantation in high-risk neuroblastoma patients.
abstract: The aim of this retrospective study was to analyze the outcome and identify risk factors associated with progression-free survival (PFS) in 36 children with high-risk neuroblastoma who underwent autologous peripheral blood progenitor cell (PBPC) transplantation between 1994 and 2010. The conditioning regimen used in all cases consisted of high-dose of busulfan and melphalan. Median age at transplantation was 3 years (range: 0.7-14 years). The median times to neutrophil and platelet engraftment were 11 days (range: 9.16 days) and 13 days (range: 9.33), respectively. Twenty-one patients developed nonhematologic toxicity: 15 patients had mucositis, 4 patients developed an engraftment syndrome, and there were 2 cases of liver toxicity. No toxic deaths were observed. There were 15 patients who relapsed. The median time to relapse was 6 months after the transplant (range: 3-13 months). With a median follow-up of 55 months (range: 4-180 months), the PFS was 57% ± 8.5% for the whole group. In multivariate analysis, age below 3 years (P < .005), complete remission (CR) pretransplantation (P < .07) and 1p germline status (P < .01) were variables associated with better outcomes. Patients who were or achieved early CR following transplantation (3 months posttransplantation) had a probability of PFS of 91% ± 6% as compared to patients who did not (PFS 9% ± 8%) (P < .0001). This retrospective study shows that high dose of busulfan and melphalan as conditioning regimen in children with high-risk neuroblastoma is associated with very low morbidity and no mortality in the authors' hands. Younger patients with no 1p deletions and in first CR at transplantation had the better outcome.
authors.affiliation: Division of Pediatric Hematology-Oncology and Hematopoietic Stem Cell Transplantation, Hospital Infantil Universitario Nino Jesus, Madrid, Spain.
authors.forename: Blanca
authors.initials: B
authors.lastname: Molina
authors.forename: Laura
authors.initials: L
authors.lastname: Alonso
authors.forename: Marta
authors.initials: M
authors.lastname: Gonzalez-Vicent
authors.forename: Maitane
authors.initials: M
authors.lastname: Andion
authors.forename: Carmen
authors.initials: C
authors.lastname: Hernandez
authors.forename: Alvaro
authors.initials: A
authors.lastname: Lassaletta
authors.forename: Maria
authors.initials: M
authors.lastname: Cormenzana
authors.forename: Blanca
authors.initials: B
authors.lastname: Lopez-Ibor
authors.forename: Marta
authors.initials: M
authors.lastname: Villa
authors.forename: Javier
authors.initials: J
authors.lastname: Molina
authors.forename: Miguel A
authors.initials: MA
authors.lastname: Diaz
mesh_terms: D000293:Adolescent; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage*; D019046:Bone Marrow Neoplasms / Q000276:immunology / Q000556:secondary / Q000628:therapy; D001859:Bone Neoplasms / Q000276:immunology / Q000556:secondary / Q000628:therapy; D002066:Busulfan / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D005500:Follow-Up Studies; D019650:Hematopoietic Stem Cell Mobilization; D006801:Humans; D007223:Infant; D008113:Liver Neoplasms / Q000276:immunology / Q000556:secondary / Q000628:therapy; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D009367:Neoplasm Staging; D009447:Neuroblastoma / Q000276:immunology / Q000473:pathology / Q000628:therapy*; D036102:Peripheral Blood Stem Cell Transplantation; D012074:Remission Induction; D012307:Risk Factors; D015996:Survival Rate; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D018906:Antineoplastic Agents, Alkylating; D002066:Busulfan; D008558:Melphalan