Tandem high-dose chemotherapy with thiotepa and busulfan-melphalan and autologous stem cell transplantation in very high-risk neuroblastoma patients
| Autor: | Claudia Pasqualini, M-A Raquin, G. Goma, Dominique Valteau-Couanet, Valérie Lapierre, Christelle Dufour |
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| Rok vydání: | 2015 |
| Předmět: |
Oncology
Male medicine.medical_specialty Adolescent medicine.medical_treatment ThioTEPA Disease-Free Survival 03 medical and health sciences Neuroblastoma 0302 clinical medicine Autologous stem-cell transplantation immune system diseases hemic and lymphatic diseases Internal medicine Antineoplastic Combined Chemotherapy Protocols medicine Humans Progenitor cell Autografts Child neoplasms Busulfan Melphalan Retrospective Studies Transplantation Chemotherapy business.industry Infant Hematology medicine.disease Surgery Survival Rate surgical procedures operative Graft-versus-host disease 030220 oncology & carcinogenesis Child Preschool Female Stem cell business Thiotepa 030215 immunology medicine.drug Stem Cell Transplantation |
| Zdroj: | Bone marrow transplantation. 51(2) |
| ISSN: | 1476-5365 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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