Girmens Jean François, Erginay Ali, Massin Pascale, Scigalla Paul, Gaudric Alain, Richard Stéphane
Service d'Ophtalmologie, Hôpital Lariboisière, Assistance Publique-Hôpitaux de Paris and Université Paris VII, Paris, France.
Am J Ophthalmol. 2003 Jul;136(1):194-6. doi: 10.1016/s0002-9394(03)00101-6.
To test the efficacy of the novel vascular endothelial growth factor (VEGF) receptor inhibitor SU5416, in a case of refractory von Hippel-Lindau (VHL) retinal hemangioblastoma (RHB).
Interventional case report.
Patient included in a multicenter phase II trial. A 30-year-old woman presenting with VHL disease and multiple RHB on her only eye, refractory to conventional treatments, had decreased visual acuity due to cystoid macular edema (CME). SU5416 was administered intravenously for 7 months. Best-corrected visual acuity (BCVA) and macular thickness were measured by optical coherence tomography.
Under treatment, the size of the RHB did not change, but CME improved significantly. Best-corrected visual acuity rose from 20/40 to 20/25. However, CME recurred after the end of the treatment.
The VEGF receptor inhibitor SU5416 failed to reduce the size of RHB but was very effective for the associated CME.
在一例难治性冯·希佩尔-林道（VHL）视网膜血管瘤（RHB）患者中测试新型血管内皮生长因子（VEGF）受体抑制剂SU5416的疗效。
介入性病例报告。
患者纳入一项多中心II期试验。一名30岁女性，患有VHL病，单眼有多个RHB，对传统治疗无效，因黄斑囊样水肿（CME）导致视力下降。静脉注射SU5416共7个月。通过光学相干断层扫描测量最佳矫正视力（BCVA）和黄斑厚度。
治疗期间，RHB的大小未改变，但CME显著改善。最佳矫正视力从20/40提高到20/25。然而，治疗结束后CME复发。
VEGF受体抑制剂SU5416未能减小RHB的大小，但对相关的CME非常有效。
