缺乏对低收入和中等收入国家视网膜母细胞瘤患者的长期影响的研究。因此，我们检查了 1986 年至 2003 年在巴西圣保罗安东尼奥·坎迪多·德卡马戈癌症中心 (ACCCC) 接受治疗并随访至 2018 年 12 月 31 日的视网膜母细胞瘤患者的回顾性队列的具体原因死亡率。 生命状况和死亡原因是根据医疗记录和多个国家数据库确定的。我们使用 Kaplan-Meier 生存法估计了总体生存率和特定原因生存率，并估计了死亡的标准化死亡率 (SMR) 和绝对超额风险 (AER)。该队列研究包括 465 名视网膜母细胞瘤患者（42% 为遗传性，58% 为非遗传性），其中大多数 (77%) 患者在晚期（IV 或 V）诊断时。在平均 11 年的随访中，发生了 80 例死亡：70% 死于视网膜母细胞瘤，22% 死于随后的恶性肿瘤 (SMN)，5% 死于非癌症原因。遗传性和非遗传性患者的总体 5 年生存率一致为 88% (p = .67)。与普通人群相比，遗传性视网膜母细胞瘤患者面临的 SMN 相关死亡风险高出 86 倍（N = 16，SMR = 86.1，95% CI 52.7-140.5），相当于每 10,000 人年有 42.4 例超额死亡。对于接受放疗和化疗的患者（N = 10，SMR = 90.3，95% CI 48.6-167.8）和单独化疗的患者（N = 6，SMR = 80.0，95% CI 35.9-177.9），这种风险保持一致。非遗传性患者仅发生 2 例 SMN 相关死亡（SMR = 7.2，95% CI 1.8-28.7）。两种视网膜母细胞瘤均不存在非癌症相关死亡的过高风险。该队列中晚期患者比例较高且化疗使用广泛，其结果可能有助于指导政策和医疗保健规划，强调需要加强欠发达国家的早期诊断和治疗机会。© 2024 作者。约翰·威利出版的《国际癌症杂志》

Studies are lacking on long-term effects among retinoblastoma patients in low- and middle-income countries. Therefore, we examined cause-specific mortality in a retrospective cohort of retinoblastoma patients treated at Antonio Candido de Camargo Cancer Center (ACCCC), São Paulo, Brazil from 1986 to 2003 and followed up through December 31, 2018. Vital status and cause of death were ascertained from medical records and multiple national databases. We estimated overall and cause-specific survival using the Kaplan-Meier survival method, and estimated standardized mortality ratios (SMRs) and absolute excess risk (AER) of death. This cohort study included 465 retinoblastoma patients (42% hereditary, 58% nonhereditary), with most (77%) patients diagnosed at advanced stages (IV or V). Over an 11-year average follow-up, 80 deaths occurred: 70% due to retinoblastoma, 22% due to subsequent malignant neoplasms (SMNs) and 5% to non-cancer causes. The overall 5-year survival rate was 88% consistent across hereditary and nonhereditary patients (p = .67). Hereditary retinoblastoma patients faced an 86-fold higher risk of SMN-related death compared to the general population (N = 16, SMR = 86.1, 95% CI 52.7-140.5), corresponding to 42.4 excess deaths per 10,000 person-years. This risk remained consistent for those treated with radiotherapy and chemotherapy (N = 10, SMR = 90.3, 95% CI 48.6-167.8) and chemotherapy alone (N = 6, SMR = 80.0, 95% CI 35.9-177.9). Nonhereditary patients had only two SMN-related deaths (SMR = 7.2, 95% CI 1.8-28.7). There was no excess risk of non-cancer-related deaths in either retinoblastoma form. Findings from this cohort with a high proportion of advanced-stage patients and extensive chemotherapy use may help guide policy and healthcare planning, emphasizing the need to enhance early diagnosis and treatment access in less developed countries.© 2024 The Author(s). International Journal of Cancer published by John Wiley & Sons Ltd on behalf of UICC. This article has been contributed to by U.S. Government employees and their work is in the public domain in the USA.