Department of Medical Oncology, Cancer Institute (WIA), Adyar, Chennai, Tamilnadu, India.
Department of Surgical Oncology, Cancer Institute (WIA), Adyar, Chennai, Tamilnadu, India.
Indian J Pediatr. 2019 May;86(5):417-426. doi: 10.1007/s12098-018-2834-6. Epub 2019 Feb 18.
Management of neuroblastoma, especially high-risk (HR) disease is difficult in a resource-limited setting. There is a paucity of literature on outcomes of patients treated in India. The present study was conducted to analyse the clinical profile, treatment, and outcomes of patients with neuroblastoma treated at authors' centre.
The study was a retrospective analysis of newly diagnosed patients with neuroblastoma treated at authors' centre between 2000 to 2017. The International Neuroblastoma Staging System and risk grouping were used to classify patients as low-risk (LR), intermediate-risk (IR) and high-risk (HR). Treatment was individualised and risk-adapted. Kaplan-Meier method was used to calculate the event-free survival (EFS) and overall survival (OS).
The study included 85 patients with a median age of 4 y and 67% were males. Malnutrition was observed in 55% of patients. Adrenal gland was the most common site in 75% patients followed by mediastinum in 12%. LR was observed in 7/85 (8%) patients, IR 20/85 (24%) and HR in 58/85 (68%) patients. The CCG-3891 protocol was used to treat 80% of the patients. Autologous stem cell transplantation (ASCT) was performed in 32% of HR patients. The median follow-up was 16.6 mo. The median EFS and OS for all patients were 19.2 mo and 26.9 mo respectively and the 3 y EFS and OS was 36% and 47% respectively. The 3y EFS for LR, IR and HR patients was 100%, 54%, and 18.9% respectively (P < 0.001) and for OS was 100%, 77%, and 34% respectively (P = 0.002). On multivariate analysis, a hemoglobin less than 10 g% predicted inferior EFS (P = 0.002) and OS (p = 0.005) for all patients. For patients with high-risk disease, on multivariate analysis, hemoglobin (P = 0.002) and 13-Cis Retinoic acid maintenance (P = 0.002) predicted EFS and only radiotherapy to the primary (P = 0.01) predicted OS. Only 4/19 (21%) are alive and in remission post ASCT.
Majority of patients with neuroblastoma presented to authors' centre with advanced disease. Survival outcomes of patients with LR disease are excellent. However, patients with HR disease have poor outcomes despite multimodality management. Non-availability of N-MYC testing in few patients could have falsely down-staged them to IR from HR. A low hemoglobin at diagnosis is a poor predictor of outcome.
神经母细胞瘤的治疗，尤其是高危（HR）疾病的治疗在资源有限的情况下较为困难。印度患者治疗结果的相关文献较少。本研究旨在分析作者所在中心治疗的神经母细胞瘤患者的临床特征、治疗方法和结果。
本研究为回顾性分析 2000 年至 2017 年在作者所在中心接受治疗的新诊断神经母细胞瘤患者。采用国际神经母细胞瘤分期系统和风险分组将患者分为低危（LR）、中危（IR）和高危（HR）。治疗方法个体化且适应风险。采用 Kaplan-Meier 法计算无事件生存率（EFS）和总生存率（OS）。
本研究共纳入 85 例患者，中位年龄为 4 岁，67%为男性。55%的患者存在营养不良。75%的患者肿瘤位于肾上腺，12%的患者位于纵隔。7/85（8%）例患者为 LR，20/85（24%）例患者为 IR，58/85（68%）例患者为 HR。80%的患者采用 CCG-3891 方案治疗。32%的 HR 患者接受了自体造血干细胞移植（ASCT）。中位随访时间为 16.6 个月。所有患者的中位 EFS 和 OS 分别为 19.2 个月和 26.9 个月，3 年 EFS 和 OS 分别为 36%和 47%。LR、IR 和 HR 患者的 3 年 EFS 分别为 100%、54%和 18.9%（P<0.001），OS 分别为 100%、77%和 34%（P=0.002）。多变量分析显示，血红蛋白<10g%预测所有患者 EFS（P=0.002）和 OS（P=0.005）不良。对于高危疾病患者，多变量分析显示，血红蛋白（P=0.002）和 13-顺式维甲酸维持（P=0.002）预测 EFS，仅放疗原发灶（P=0.01）预测 OS。仅有 4/19（21%）例患者在 ASCT 后仍存活且缓解。
大多数神经母细胞瘤患者就诊时已处于晚期疾病阶段。LR 疾病患者的生存结果非常好。然而，尽管采用了多模式治疗，HR 疾病患者的预后仍较差。少数患者未进行 N-MYC 检测，可能导致他们从 HR 降期为 IR。诊断时血红蛋白低是预后不良的预测因素。
