Zhao Junyang, Feng Zhaoxun, Gallie Brenda L
Department of Ophthalmology, Chongqing Aier Children's Eye Hospital, Chongqing 400020, China.
Department of Ophthalmology, University of Ottawa, Ottawa, ON K1H 8M2, Canada.
Cancers (Basel). 2021 Jul 21;13(15):3646. doi: 10.3390/cancers13153646.
Treatment abandonment is a leading cause of death in children with retinoblastoma worldwide. We studied children who abandoned treatment upfront at diagnosis to delineate the natural history of untreated retinoblastoma. Studied were children who received no treatment, diagnosed between 2007 and 2017 at 29 Chinese centers. Data were retrospectively collected from medical chart reviews and interviews with each patient's family. During the study period, 44 children received no treatment after diagnosis of retinoblastoma. Clinical or radiologic evidence of orbital extension was available for 25 children, and radiologic evidence of systemic metastasis was available for 12 children. Median times from diagnosis of intraocular tumor to orbital disease was 13.7 months, orbital disease to metastasis was 2.6 months, and metastasis to death was 2.0 months. Children with brain metastasis had shorter survival than those with metastasis to other sites (median 1.0 vs. 3.1 months; = 0.015). Overall, 36% of patients died within 12 months of diagnosis, 77% within 24 months, 95% within 36 months and 100% within 48 months. While multiple factors influence refusal of treatment, insights into the natural history of retinoblastoma derived from real-world evidence can inform clinicians and parents that retinoblastoma is life-threatening and encourage urgent treatment at diagnosis.
在全球范围内，放弃治疗是视网膜母细胞瘤患儿的主要死因。我们对诊断时即直接放弃治疗的患儿进行了研究，以描绘未经治疗的视网膜母细胞瘤的自然病程。研究对象为2007年至2017年期间在中国29个中心确诊后未接受任何治疗的患儿。数据通过病历回顾以及与每位患儿家属的访谈进行回顾性收集。在研究期间，44例视网膜母细胞瘤患儿确诊后未接受治疗。25例患儿有眼眶扩展的临床或影像学证据，12例患儿有全身转移的影像学证据。从眼内肿瘤诊断到眼眶病变的中位时间为13.7个月，从眼眶病变到转移的中位时间为2.6个月，从转移到死亡的中位时间为2.0个月。发生脑转移的患儿比转移至其他部位的患儿生存时间更短（中位时间1.0个月对3.1个月；P = 0.015）。总体而言，36%的患者在诊断后12个月内死亡，77%在24个月内死亡，95%在36个月内死亡，100%在48个月内死亡。虽然多种因素影响治疗的拒绝，但从真实世界证据中获得的关于视网膜母细胞瘤自然病程的见解可以告知临床医生和家长，视网膜母细胞瘤会危及生命，并鼓励在诊断时进行紧急治疗。
