Vajzovic Lejla Mutapcic, Murray Timothy G, Aziz-Sultan Mohammad A, Schefler Amy C, Wolfe Stacey Quintero, Hess Ditte, Fernandes Cristina E, Dubovy Sander R
Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA;
Clin Ophthalmol. 2011;5:171-6. doi: 10.2147/OPTH.S12665. Epub 2011 Feb 10.
The purpose of this study is to report the complication profile and safety evaluation of supraselective intra-arterial melphalan chemotherapy in children undergoing treatment with advanced retinoblastoma.
Twelve eyes of 10 children with advanced retinoblastoma (Reese-Ellsworth Group Vb or International Classification Group D) were treated with supraselective intra-ophthalmic artery infusion of melphalan. Eleven eyes of nine children had previously failed traditional management with systemic chemotherapy and laser ablation and underwent intra-ophthalmic artery infusion of melphalan as an alternative to enucleation. Serial ophthalmic examinations, retinal photography, and ultrasonographic imaging were used to evaluate treatment regime.
Ophthalmic artery cannulation was successfully performed in 12 eyes of 10 patients (total 16 times). Striking regression of tumor, subretinal and vitreous seeds were seen early in each case. No severe systemic side effects occurred. Grade III neutropenia was seen in one patient. No transfusions were required. Three patients developed a vitreous hemorrhage obscuring tumor visualization. One patient developed periocular edema associated with inferior rectus muscle inflammation per orbital MRI. This same patient had scattered intraretinal hemorrhages and peripapillary cotton wool spots consistent with a Purtscher's-like retinopathy that resolved spontaneously. At the 6-month follow-up examination, nine eyes had no evidence of tumor progression, whereas three eyes were enucleated for tumor progression. In each enucleated case, viable tumor was identified on histopathologic examination.
Ophthalmic intra-arterial infusion with melphalan is an excellent globe-conserving treatment option in advanced retinoblastoma cases with minimal systemic side effects. Local toxicities include microemboli to the retina and choroid (1/12, 8%), vitreous hemorrhage (3/12, 25%), and myositis (1/12, 8%). Enucleation remained a definitive treatment for tumor progression in 3 of 12 eyes in this small case series with limited follow-up. Further studies are necessary to establish the role of supraselective intra-arterial melphalan chemotherapy for children with retinoblastoma.
本研究旨在报告超选择性眼动脉内美法仑化疗在晚期视网膜母细胞瘤患儿治疗中的并发症情况及安全性评估。
对10例晚期视网膜母细胞瘤（里斯 - 埃尔斯沃思分级Vb级或国际分级D级）患儿的12只眼进行超选择性眼动脉内美法仑灌注治疗。9例患儿的11只眼此前传统的全身化疗和激光消融治疗失败，接受眼动脉内美法仑灌注作为眼球摘除术的替代方案。采用系列眼科检查、视网膜摄影及超声成像评估治疗方案。
10例患者的12只眼（共16次）成功完成眼动脉插管。每例均早期观察到肿瘤、视网膜下及玻璃体内种植灶显著消退。未发生严重全身副作用。1例患者出现III级中性粒细胞减少。无需输血。3例患者发生玻璃体积血，影响肿瘤观察。1例患者眼眶MRI显示眶周水肿伴下直肌炎症。同一患者出现散在视网膜内出血及视乳头周围棉絮斑，符合类Purtscher视网膜病变，后自发消退。在6个月随访检查时，9只眼无肿瘤进展迹象，3只眼因肿瘤进展行眼球摘除术。在每例眼球摘除病例的组织病理学检查中均发现存活肿瘤。
眼动脉内美法仑灌注是晚期视网膜母细胞瘤病例中一种极佳的保眼球治疗选择，全身副作用极小。局部毒性包括视网膜和脉络膜微栓塞（1/12，8%）、玻璃体积血（3/12，25%）及肌炎（1/12，8%）。在这个随访有限的小病例系列中，12只眼中有3只眼因肿瘤进展眼球摘除术仍是最终治疗手段。有必要进一步研究以确定超选择性眼动脉内美法仑化疗在视网膜母细胞瘤患儿中的作用。
