Soni Sandeep, Pai Vinita, Gross Thomas G, Ranalli Mark
Division of Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, OH, USA.
Pediatr Transplant. 2014 Mar;18(2):217-20. doi: 10.1111/petr.12202. Epub 2013 Dec 16.
Bu-Mel as preparative therapy prior to autologous stem cell rescue was recently shown to be superior to the conventional CEM regimen for HR NBL in Europe. There are no data available on the feasibility and toxicity of Bu-Mel as consolidation therapy following the COG-type induction regimens used in North America. We report early complications and outcomes of patients with HR NBL who received Bu-Mel for consolidation following COG-based induction. Retrospective analysis of all patients who had received Bu-Mel as preparative regimen prior to stem cell rescue for HR NBL was carried out. Toxicity, outcomes, and any delays to receiving radiation or anti-GD2 antibody therapy were analyzed. Six patients undergoing PBSCT had received Bu-Mel. The treatment was well tolerated. Mucositis was the main toxicity; three patients had developed neutropenia fever and none developed pulmonary toxicity. One patient had developed moderate SOS that responded to conservative management. All patients were able to receive and tolerate post-transplant local radiotherapy and ch.14.18 anti-GD2 antibody therapy without any delays. All patients are alive with no disease recurrence. The Bu-Mel regimen is well tolerated and is feasible post-COG-type induction platform.
最近在欧洲,Bu-Mel作为自体干细胞救援前的预处理疗法,已被证明在高危神经母细胞瘤(HR NBL)方面优于传统的CEM方案。目前尚无关于在北美使用的COG型诱导方案后,将Bu-Mel作为巩固疗法的可行性和毒性的数据。我们报告了接受基于COG诱导方案后使用Bu-Mel进行巩固治疗的HR NBL患者的早期并发症和结局。对所有在HR NBL干细胞救援前接受Bu-Mel作为预处理方案的患者进行了回顾性分析。分析了毒性、结局以及接受放疗或抗GD2抗体治疗的任何延迟情况。6例接受 PBSCT 的患者接受了Bu-Mel治疗。该治疗耐受性良好。粘膜炎是主要毒性反应;3例患者出现中性粒细胞减少性发热,无患者出现肺部毒性。1例患者发生中度肝静脉闭塞病(SOS),经保守治疗后缓解。所有患者都能够接受并耐受移植后局部放疗和14.18抗GD2抗体治疗,且无任何延迟。所有患者均存活,无疾病复发。Bu-Mel方案耐受性良好,在基于COG型诱导平台后是可行的。