title: Tandem high-dose chemotherapy with thiotepa and busulfan-melphalan and autologous stem cell transplantation in very high-risk neuroblastoma patients.
abstract: High-risk neuroblastoma is characterised by poor long-term survival, especially for very high-risk (VHR) patients (poor response of metastases after induction therapy). We report the results of an intensified high-dose chemotherapy (HDC) strategy to improve the prognosis of VHR patients. This strategy was based on tandem HDC with thiotepa and busulfan-melphalan (Bu-Mel) followed by autologous stem cell transplantation (ASCT). All data were prospectively recorded in the Gustave Roussy Paediatric ASCT database. From April 2004 to August 2011, 26 patients were eligible for tandem HDC. The median age at diagnosis was 4.4 years (1-15.9). All patients had metastatic disease. MYCN was amplified in 5/26 tumours. Despite the cumulative toxicity of alkylating agents, the toxicity of the intensified HDC strategy was manageable. Thiotepa-related toxicity was mainly digestive, whereas sinusoidal obstruction syndrome was the main toxicity observed after Bu-Mel. The 3-year event-free survival of this cohort was 37.3% (21.3-56.7). This strategy will be compared with combined (131)I-mIBG/Bu-Mel in the upcoming SIOPEN VHR Neuroblastoma Protocol.
authors.affiliation: Department of Paediatric and Adolescent Oncology, Gustave Roussy Cancer Campus, Villejuif, France.
authors.forename: C
authors.initials: C
authors.lastname: Pasqualini
authors.affiliation: Department of Paediatric and Adolescent Oncology, Gustave Roussy Cancer Campus, Villejuif, France.
authors.forename: C
authors.initials: C
authors.lastname: Dufour
authors.affiliation: Biostatistics and Epidemiology Unit, Gustave Roussy Cancer Campus, Villejuif, France.
authors.forename: G
authors.initials: G
authors.lastname: Goma
authors.affiliation: Department of Paediatric and Adolescent Oncology, Gustave Roussy Cancer Campus, Villejuif, France.
authors.forename: M-A
authors.initials: MA
authors.lastname: Raquin
authors.affiliation: Cell Therapy Unit, Gustave Roussy Cancer Campus, Villejuif, France.
authors.forename: V
authors.initials: V
authors.lastname: Lapierre
authors.affiliation: Department of Paediatric and Adolescent Oncology, Gustave Roussy Cancer Campus, Villejuif, France.
authors.forename: D
authors.initials: D
authors.lastname: Valteau-Couanet
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage* / Q000009:adverse effects; D064592:Autografts; D002066:Busulfan / Q000008:administration & dosage / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000009:adverse effects; D009447:Neuroblastoma / Q000401:mortality / Q000628:therapy; D012189:Retrospective Studies; D033581:Stem Cell Transplantation; D015996:Survival Rate; D013852:Thiotepa / Q000008:administration & dosage / Q000009:adverse effects
chemical_list: D013852:Thiotepa; D002066:Busulfan; D008558:Melphalan