Paediatric Haematology and Oncology, Department of Paediatrics and Child Health, Faculty of Medicine and Health Sciences, Stellenbosch University, Tygerberg Hospital, Cape Town, South Africa.
Faculty of Health Sciences, Division of Paediatric Haematology and Oncology, Department of Paediatrics and Child Health, University of the Witwatersrand, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.
Pediatr Hematol Oncol. 2020 May;37(4):300-313. doi: 10.1080/08880018.2020.1717698. Epub 2020 Feb 19.
Achieving remission after induction therapy in high-risk neuroblastoma (HR-NB) is of significant prognostic importance. This study investigated remission after induction-chemotherapy using three standard neuroblastoma protocols in the South African (SA) setting. Retrospective data of 261 patients with HR-NB diagnosed between January 2000 and December 2016, who completed induction chemotherapy with standard treatment protocols were evaluated. The treatment protocols were either OPEC/OJEC or the St Jude NB84 protocol (NB84) or rapid COJEC (rCOJEC). The postinduction metastatic complete remission (mCR) rate, 2-year overall survival (OS) and 2-year event free survival (EFS) were determined as comparative denominators. The majority (48.3%; = 126) received OPEC/OJEC, while 70 patients received (26.8%) rCOJEC and 65 (24.9%) NB84. Treatment with NB84 had the best mCR rate (36.9%), followed by OPEC/OJEC (32.5%) and rCOJEC (21.4%). The 2-year OS of treatment with NB84 was 41% compared to OPEC/OJEC (35%) and rCOJEC (24%) ( = 0.010). The 2-year EFS of treatment with NB84 was 37% compared to OPEC/OJEC (35%) and rCOJEC (18%) ( = 0.008). OPEC/OJEC had the least treatment-related deaths (1.6%) compared to rCOJEC (7.1%) and NB84 (7.5%) ( = 0.037). On multivariate analysis LDH ( = 0.023), ferritin ( = 0.002) and INSS stage ( = 0.006) were identified as significant prognostic factors for OS. The induction chemotherapy was not significant for OS ( = 0.18), but significant for EFS ( = 0.08) Treatment with NB84 achieved better mCR, OS and EFS, while OPEC/OJEC had the least treatment-related deaths. In resource-constrained settings, OPEC/OJEC is advised as induction chemotherapy in HR-NB due to less toxicity as reflected in less treatment-related deaths.
在高危神经母细胞瘤（HR-NB）中诱导治疗后达到缓解具有重要的预后意义。本研究调查了南非（SA）环境中使用三种标准神经母细胞瘤方案进行诱导化疗后的缓解情况。回顾性分析了 2000 年 1 月至 2016 年 12 月期间诊断为 HR-NB 的 261 例患者的数据，这些患者完成了标准治疗方案的诱导化疗。治疗方案为 OPEC/OJEC 或圣裘德 NB84 方案（NB84）或快速 COJEC（rCOJEC）。作为比较基准，确定诱导后转移性完全缓解（mCR）率、2 年总生存率（OS）和 2 年无事件生存率（EFS）。大多数患者（48.3%，n=126）接受 OPEC/OJEC 治疗，70 例患者（26.8%）接受 rCOJEC 治疗，65 例患者（24.9%）接受 NB84 治疗。NB84 治疗的 mCR 率最高（36.9%），其次是 OPEC/OJEC（32.5%）和 rCOJEC（21.4%）。NB84 治疗的 2 年 OS 为 41%，高于 OPEC/OJEC（35%）和 rCOJEC（24%）（p=0.010）。NB84 治疗的 2 年 EFS 为 37%，高于 OPEC/OJEC（35%）和 rCOJEC（18%）（p=0.008）。与 rCOJEC（7.1%）和 NB84（7.5%）相比，OPEC/OJEC 的治疗相关死亡率最低（1.6%）（p=0.037）。多变量分析显示，LDH（p=0.023）、铁蛋白（p=0.002）和 INSS 分期（p=0.006）是 OS 的显著预后因素。诱导化疗对 OS 无显著影响（p=0.18），但对 EFS 有显著影响（p=0.08）。NB84 治疗可获得更好的 mCR、OS 和 EFS，而 OPEC/OJEC 的治疗相关死亡率最低。在资源有限的环境中，由于治疗相关死亡率较低，OPEC/OJEC 被推荐作为 HR-NB 的诱导化疗方案。
