Background:Intra-arterial chemotherapy (IAC) is increasingly useful for treating intraocular retinoblastoma (Rb). It offers targeted delivery of chemotherapy with reduced systemic exposure. In this study, we evaluate management outcomes and identify predictive factors for globe salvage following IAC in children with Rb.Methods:This retrospective study included 20 eyes of 20 melphalan-based IAC-treated patients (67 sessions) between 2015 and 2023 in a tertiary cancer center (King Hussein Cancer Center) in Jordan. Data collection included patients’ demographics, tumor staging, eye salvage, complications, and survival, followed by statistical comparisons between eye salvage rates and clinical factors.Results:The median age of IAC initiation was 38 months (range: 6–78 months). IAC was used as a primary treatment in 35% (7/20) of eyes and as a secondary treatment following systemic chemotherapy in 65% (13/20) of eyes. Nineteen (95%) eyes showed initial tumor regression, 15 (75%) eyes showed short term tumor control, and long-term eye salvage was achieved in 11 (55%) eyes. Poor prognostic factors for eye salvage included advanced tumor stage (Group D/E: 43% salvage rate vs. Group C: 83%;p= 0.047), vitreous seeding at the time of IAC (38% with seeding vs. 75% without;p= 0.046), use of IAC as a secondary rather than a primary treatment (46% vs. 71%;p= 0.047), and the need for >3 IAC cycles (20% success with >3 cycles vs. 67% with ≤3 cycles;p= 0.034). Complications were notable: systemic adverse effects were seen in five (25%) patients, including neutropenia (20%) and bronchospasm (6%). Procedure-related complications were seen with 22% of injections, including failure of the procedure (7%), ophthalmic artery spasm (6%), and intra-procedural stroke (3%). Five (25%) eyes developed ocular complications, including vitreous hemorrhage (15%), retinal detachment (10%), optic atrophy (10%), and retinal or choroidal ischemia (10%). Notably, all infants under 12 months of age (4/4) developed complications, including the two events of stroke. At a median follow-up of 60 months, eye salvage was achieved in 11 (55%) eyes, and none of the 9 (45%) enucleated eyes showed high-risk pathological features. There was no orbital recurrence, and one (5%) child developed CNS metastasis and passed away.Conclusion:IAC achieves long-term globe salvage in 55% of Rb cases; however, outcomes are poorer with Group D/E tumors, vitreous seeds, prior IVC failure, or requiring >3 IAC cycles. While reducing systemic chemotherapy toxicity, IAC carries significant risks of vision- and life-threatening complications. Infants and single-eyed patients require particularly cautious consideration. Though IAC remains crucial for globe preservation, optimal implementation demands improved patient selection criteria, multicenter collaboration, and long-term outcome studies to maximize safety and efficacy.
背景：动脉内化疗（IAC）在治疗眼内视网膜母细胞瘤（Rb）中的应用日益广泛。该方法可实现化疗药物的靶向递送，并减少全身暴露。本研究旨在评估IAC治疗Rb患儿的眼球保留效果，并识别影响眼球保留的预测因素。 方法：这项回顾性研究纳入2015年至2023年间在约旦三级癌症中心（侯赛因国王癌症中心）接受以马法兰为基础的IAC治疗的20例患者（共20只眼，67次治疗）。数据收集包括患者人口统计学特征、肿瘤分期、眼球保留情况、并发症及生存状态，随后对眼球保留率与临床因素进行统计学比较。 结果：开始IAC治疗的中位年龄为38个月（范围：6-78个月）。35%（7/20）的眼将IAC作为初始治疗，65%（13/20）的眼在全身化疗失败后将其作为二线治疗。19只眼（95%）显示初期肿瘤消退，15只眼（75%）实现短期肿瘤控制，11只眼（55%）获得长期眼球保留。影响眼球保留的不良预后因素包括：晚期肿瘤分期（D/E组：保留率43% vs. C组：83%；p=0.047）、IAC时存在玻璃体种植（有种植者38% vs. 无种植者75%；p=0.046）、IAC作为二线而非初始治疗（46% vs. 71%；p=0.047）以及需要>3个IAC周期（>3周期成功率20% vs. ≤3周期67%；p=0.034）。并发症值得关注：5例患者（25%）出现全身不良反应，包括中性粒细胞减少（20%）和支气管痉挛（6%）。22%的注射操作出现相关并发症，包括操作失败（7%）、眼动脉痉挛（6%）和术中卒中（3%）。5只眼（25%）发生眼部并发症，包括玻璃体积血（15%）、视网膜脱离（10%）、视神经萎缩（10%）以及视网膜或脉络膜缺血（10%）。值得注意的是，所有12月龄以下婴儿（4/4）均出现并发症，其中包括2例卒中事件。中位随访60个月后，11只眼（55%）实现眼球保留，9只摘除眼（45%）均未显示高危病理特征。无眼眶复发，1例患儿（5%）发生中枢神经系统转移并死亡。 结论：IAC在55%的Rb病例中实现长期眼球保留；但对于D/E组肿瘤、存在玻璃体种植、既往静脉化疗失败或需要>3个IAC周期的病例，疗效较差。尽管IAC降低了全身化疗毒性，但其存在导致视力损害及危及生命的严重并发症风险。婴幼儿和单眼患者需特别谨慎评估。虽然IAC在眼球保留治疗中仍至关重要，但为最大化安全性与疗效，需优化患者选择标准、加强多中心协作并开展长期结局研究。
肿瘤(癌症)患者之家