Department of Pediatrics and Child Health, Faculty of Medicine and Health Sciences, Stellenbosch University, Cape Town, South Africa.
Tygerberg Hospital, Cape Town, South Africa.
JCO Glob Oncol. 2024 Aug;10:e2400034. doi: 10.1200/GO.24.00034.
Retinoblastoma, a curable childhood cancer, has been identified as a tracer cancer in the WHO Global Initiative for Childhood Cancer. To document the outcomes of children with retinoblastoma in South Africa, treated as per the first prospective standard national treatment guidelines for childhood cancer in South Africa.
All children diagnosed with retinoblastoma between 2012 and 2016 in five South African pediatric oncology units were treated with a standard treatment on the basis of the International Society of Pediatric Oncology-Pediatric Oncology in Developing Countries guidelines for high-income settings. Treatment included focal therapy with/without chemotherapy, or enucleation with/without chemotherapy, and orbital radiotherapy, depending on enucleated eye histology. The end point was survival at 24 months, using Kaplan-Meier curves with log-rank (Mantel-Cox) and chi-square (χ2) tests with respective values reported.
A total of 178 children were included in the study; 68% presented with unilateral disease. The median age was 27 months (range 0-118 months) with a male:female ratio of 1:0.75. The overall survival was 79% at 24 months with significant association with stage at diagnosis ( < .001) and older age over 2 years as opposed to younger than 2 years ( < .001). Causes of death were disease progression/relapses in 90% (34 of 38) and unknown in 2% (1 of 38), whereas treatment abandonment was 1.7% (3 of 178).
Efficacy with national treatment guidelines was confirmed, and feasibility of implementing standard national childhood cancer treatment guidelines was documented, involving multidisciplinary teams in South Africa. Outcome was significantly associated with stage at diagnosis and age.
视网膜母细胞瘤是一种可治愈的儿童癌症，已被世界卫生组织全球儿童癌症倡议确定为示踪癌症。本研究旨在记录在南非，根据南非首个前瞻性儿童癌症标准国家治疗指南，接受治疗的视网膜母细胞瘤患儿的结局。
2012 年至 2016 年期间，5 家南非儿科肿瘤学单位诊断为视网膜母细胞瘤的所有儿童，均根据国际小儿肿瘤学会-发展中国家小儿肿瘤学的高收入国家环境下的儿童肿瘤标准治疗指南进行标准治疗。治疗包括根据眼球组织学进行局部治疗加/或不加化疗、眼球摘除加/或不加化疗以及眼眶放疗。使用 Kaplan-Meier 曲线和对数秩（Mantel-Cox）检验以及卡方（χ2）检验进行生存分析，终点为 24 个月的生存率，并报告相应的 P 值。
共有 178 名患儿纳入本研究，其中 68%为单侧疾病。中位年龄为 27 个月（范围 0-118 个月），男女比例为 1:0.75。24 个月时的总生存率为 79%，与诊断时的分期（P<0.001）和 2 岁以上年龄（与 2 岁以下年龄相比，P<0.001）显著相关。死亡原因是疾病进展/复发 90%（34/38）和未知 2%（1/38），而放弃治疗的比例为 1.7%（3/178）。
本研究证实了国家治疗指南的疗效，并记录了在南非实施标准国家儿童癌症治疗指南的可行性，涉及多学科团队。结局与诊断时的分期和年龄显著相关。
