Abstract：
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.
摘要：
在低收入和中等收入国家，缺乏对视网膜母细胞瘤患者的长期影响的研究。因此,我们在AntonioCandidodeCamargo癌症中心(ACCCC)接受治疗的视网膜母细胞瘤患者的回顾性队列中检测了病因特异性死亡率,圣保罗,巴西从1986年到2003年，随访至2018年12月31日。根据医疗记录和多个国家数据库确定生命状况和死亡原因。我们使用Kaplan-Meier生存方法估计总体和原因特异性生存，和估计的标准化死亡率（SMR）和绝对超额死亡风险（AER）。这项队列研究包括465例视网膜母细胞瘤患者(42%是遗传性的，58%非遗传性)，大多数（77％）患者诊断为晚期（IV或V）。经过11年的平均随访，80例死亡：70%是由于视网膜母细胞瘤，22％归因于随后的恶性肿瘤（SMN），5％归因于非癌症原因。遗传性和非遗传性患者的5年总生存率为88%（p=0.67）。遗传性视网膜母细胞瘤患者与普通人群相比，SMN相关死亡风险高86倍（N=16，SMR=86.1，95％CI52.7-140.5），相当于每10,000人年42.4例超额死亡。对于接受放疗和化疗（N=10，SMR=90.3，95％CI48.6-167.8）和仅接受化疗（N=6，SMR=80.0，95％CI35.9-177.9）的患者，这种风险保持一致。非遗传患者仅有2例SMN相关死亡（SMR=7.2，95％CI1.8-28.7）。在两种视网膜母细胞瘤形式中都没有非癌症相关死亡的额外风险。从这一队列中发现高比例的晚期患者和广泛使用的化疗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。
