Azimi Fatemeh, Mirshahi Reza, Naseripour Masood
Eye Research Center, the Five Senses Institute, Iran University of Medical Sciences, Tehran, Iran.
Stem Cell and Regenerative Medicine Research Center, Iran University of Medical Sciences, Tehran, Iran.
Mol Vis. 2022 Jul 11;28:130-146. eCollection 2022.
Retinoblastoma (Rb) is a rare childhood intraocular malignancy with an incidence rate of approximately 9000 children per year worldwide. The management of Rb is inherently complex and depends on several factors. The orders of priorities in the treatment of Rb are saving life, globe salvage and vision salvage. Rarity and the young age at diagnosis impede conducting randomized clinical trials (RCTs) for new therapeutic options, and therefore pre-RCTs studies are needed. This review provides an overview of advances in Rb treatment options, focusing on the emergence of new small molecules to treat Rb. Articles related to the management and treatments of Rb were searched in different databases. Several studies and animal models discussing recent advances in the treatment of Rb were included to have a better grasp of the biological mechanisms of Rb. Over the years, the principles of management and treatment of Rb have changed significantly. Innovations in targeted therapies and molecular biology have led to improved patient and ocular survival. However, there is still a need for further evaluation of the long-term effects of these new treatments.
视网膜母细胞瘤(Rb)是一种罕见的儿童眼内恶性肿瘤,全球每年发病率约为每9000名儿童中有1例。Rb的治疗本质上很复杂,取决于多个因素。Rb治疗的优先顺序是挽救生命、挽救眼球和挽救视力。该病的罕见性以及诊断时患者年龄较小,阻碍了针对新治疗方案开展随机临床试验(RCT),因此需要进行RCT前研究。本综述概述了Rb治疗方案的进展,重点关注用于治疗Rb的新型小分子的出现。在不同数据库中检索了与Rb的管理和治疗相关的文章。纳入了几项讨论Rb治疗最新进展的研究和动物模型,以便更好地掌握Rb的生物学机制。多年来,Rb的管理和治疗原则发生了显著变化。靶向治疗和分子生物学方面的创新提高了患者生存率和眼球保留率。然而,仍需要进一步评估这些新治疗方法的长期效果。