Paediatric Haematology and Oncology, Department of Paediatrics and Child Health, Faculty of Medicine and Health Sciences, Stellenbosch University, Tygerberg Hospital, Cape Town, South Africa.
Haematology Oncology Service, Red Cross War Memorial Children's Hospital, Department of Paediatrics and Child Health, Faculty of Health Sciences, University of Cape Town, South Africa.
Pediatr Blood Cancer. 2019 Nov;66(11):e27944. doi: 10.1002/pbc.27944. Epub 2019 Jul 31.
Outcome data for neuroblastoma in sub-Saharan Africa are minimal, whereas poor outcome is reported in low- and middle-income countries. A multi-institutional retrospective study across South Africa was undertaken to determine outcome.
Patients treated between January 2000 and December 2014 in nine South African pediatric oncology units were included. Kaplan-Meier curves and Cox regression models were employed to determine two-year survival rates and to identify prognostic factors.
Data from 390 patients were analyzed. The median age was 39.9 months (range, 0-201 months). The majority presented with stage 4 disease (70%). The main chemotherapy regimens were OPEC/OJEC (44.8%), St Jude NB84 protocol (28.96%), and Rapid COJEC (22.17%). Only 44.4% had surgery across all risk groups, whereas only 16.5% of high-risk patients received radiotherapy. The two-year overall survival (OS) for the whole cohort was 37.6%: 94.1%, 81.6%, and 66.7%, respectively, for the very-low-risk, low-risk, and intermediate-risk groups and 27.6% for the high-risk group (P < 0.001, 95% CI). The median survival time for the whole group was 13 months (mean, 41.9 months; range, 0.1-209 months). MYCN-nonamplified patients had a superior two-year OS of 51.3% in comparison with MYCN-amplified patients at 37.3% (P = 0.002, 95% CI).
Limited disease had an OS comparable with high-income countries, but advanced disease had a poor OS. South Africa should focus on early diagnosis and implementation of a national protocol with equitable access to treatment.
撒哈拉以南非洲地区的神经母细胞瘤预后数据很少，而中低收入国家则报告预后较差。本研究通过在南非的九个儿科肿瘤单位进行了一项多机构回顾性研究，以确定预后情况。
纳入了 2000 年 1 月至 2014 年 12 月期间在南非 9 个儿科肿瘤单位接受治疗的患者。采用 Kaplan-Meier 曲线和 Cox 回归模型来确定两年生存率并确定预后因素。
共分析了 390 例患者的数据。中位年龄为 39.9 个月（范围 0-201 个月）。大多数患者处于 4 期疾病（70%）。主要的化疗方案为 OPEC/OJEC（44.8%）、圣裘德 NB84 方案（28.96%）和快速 COJEC（22.17%）。仅 44.4%的所有风险组患者接受了手术，而只有 16.5%的高危患者接受了放疗。整个队列的两年总生存率（OS）为 37.6%：极低危、低危和中危组分别为 94.1%、81.6%和 66.7%，高危组为 27.6%（P<0.001，95%CI）。全组的中位生存时间为 13 个月（平均 41.9 个月；范围 0.1-209 个月）。与 MYCN 扩增患者的 37.3%相比，无 MYCN 扩增患者的两年 OS 为 51.3%，具有显著优势（P=0.002，95%CI）。
局限性疾病的 OS 与高收入国家相当，但进展性疾病的 OS 较差。南非应注重早期诊断，并实施全国性方案，确保公平获得治疗。
