Martínez Arce Cristina A, Villegas Victor M, Di Nicola Maura, Williams Basil K, Murray Timothy G
School of Medicine, Universidad Central del Caribe, Bayamón 00960, Puerto Rico.
Department of Ophthalmology, University of Puerto Rico, Medical Sciences Campus, San Juan 00936, Puerto Rico.
Medicina (Kaunas). 2025 Jul 4;61(7):1219. doi: 10.3390/medicina61071219.
Retinoblastoma is a success story in pediatric oncology, evolving from life-saving interventions to approaches that preserve eyes and vision while minimizing complications. Initially managed with enucleation and radiotherapy, treatment now emphasizes eye preservation through chemotherapy as the cornerstone therapy. Various chemotherapy delivery methods-including intravenous (IVC), intraarterial (IAC), intravitreal, intracameral, and periocular-offer flexibility in treatment. Studies show nearly 100% eye salvage rates for groups A-C. For advanced cases (groups D and E), IAC has achieved outcomes that were not possible before. Intravitreal injections, when performed safely, may help avoid enucleation and radiotherapy in advanced cases, preserving vision, even in complex scenarios, with vitreous seeding. Each strategy may be tailored to tumor and patient characteristics that may help optimize outcomes. Recent innovations like liquid biopsy, prenatal diagnosis, prognostic biomarkers, and new surgical methods, such as tylectomy and chemoplaque, are paving the way for more personalized care. While advanced extraocular or metastatic retinoblastoma remains challenging, these advancements underscore a shift towards better outcomes and individualized management. The future holds promise for refining treatment strategies to maximize eye and vision preservation while ensuring patient survival.
视网膜母细胞瘤是儿科肿瘤学领域的一个成功范例，已从挽救生命的干预措施发展为在尽量减少并发症的同时保留眼球和视力的治疗方法。最初采用眼球摘除术和放射疗法进行治疗，如今治疗则强调以化疗作为基础疗法来保留眼球。包括静脉注射（IVC）、动脉内注射（IAC）、玻璃体内注射、前房内注射和眼周注射在内的各种化疗给药方法为治疗提供了灵活性。研究表明，A - C组的眼球挽救率接近100%。对于晚期病例（D组和E组），动脉内注射已取得了以前无法实现的治疗效果。在晚期病例中，安全进行玻璃体内注射有助于避免眼球摘除术和放射疗法，即使在存在玻璃体种植的复杂情况下也能保留视力。每种治疗策略都可根据肿瘤和患者的特征进行调整，这可能有助于优化治疗效果。液体活检、产前诊断、预后生物标志物以及睑板切除术和化学斑块等新手术方法等近期创新技术，正在为更个性化的治疗铺平道路。虽然晚期眼外或转移性视网膜母细胞瘤仍然具有挑战性，但这些进展突显了朝着更好的治疗效果和个体化管理的转变。未来有望改进治疗策略，在确保患者存活的同时最大限度地保留眼球和视力。
