Pasqualini C, Dufour C, Goma G, Raquin M-A, Lapierre V, Valteau-Couanet D
Department of Paediatric and Adolescent Oncology, Gustave Roussy Cancer Campus, Villejuif, France.
Biostatistics and Epidemiology Unit, Gustave Roussy Cancer Campus, Villejuif, France.
Bone Marrow Transplant. 2016 Feb;51(2):227-31. doi: 10.1038/bmt.2015.264. Epub 2015 Nov 2.
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.
高危神经母细胞瘤的特点是长期生存率低，尤其是对于极高危（VHR）患者（诱导治疗后转移灶反应不佳）。我们报告了一种强化大剂量化疗（HDC）策略改善VHR患者预后的结果。该策略基于硫替派和白消安-美法仑（Bu-Mel）的串联HDC，随后进行自体干细胞移植（ASCT）。所有数据均前瞻性记录于古斯塔夫·鲁西儿科ASCT数据库。2004年4月至2011年8月，26例患者符合串联HDC标准。诊断时的中位年龄为4.4岁（1-15.9岁）。所有患者均有转移性疾病。26例肿瘤中有5例MYCN基因扩增。尽管烷化剂有累积毒性，但强化HDC策略的毒性是可控的。硫替派相关毒性主要为消化系统毒性，而窦性阻塞综合征是Bu-Mel治疗后观察到的主要毒性。该队列的3年无事件生存率为37.3%（21.3-56.7）。在即将开展的SIOPEN VHR神经母细胞瘤方案中，将把该策略与联合（131）I-间碘苄胍/Bu-Mel进行比较。
