Edward S. Harkness Eye Institute, Columbia University Irving Medical Center, New York, New York.
Retin Cases Brief Rep. 2024 May 1;18(3):319-322. doi: 10.1097/ICB.0000000000001376.
To describe two cases of retinal hemangioblastoma regression following treatment with belzutifan in patients with von Hippel-Lindau syndrome.
Clinical information was extracted from the charts and tumor imaging of two patients with von Hippel-Lindau-associated retinal hemangioblastoma.
In Case 1, a 40-year-old man was treated with belzutifan for spine hemangioblastomas after diagnosis of a 2.0 × 2.0 × 1.3 mm left-eye retinal hemangioblastoma temporal to the macula associated with intraretinal edema, subretinal fluid, and mild retinal traction. In Case 2, a 66-year-old woman presented with a right eye 2.0 × 1.5 × 1.3 mm juxtapapillary lesion with subretinal fluid, intraretinal fluid, and nasal traction, and a 4.0 × 3.5 × 1.1 mm inferior midperiphery lesion with subretinal fluid, intraretinal fluid, and active exudation. She was treated for 2.5 years with belzutifan for renal cell carcinoma on the National Institutes of Health trial. The patient in case 1 demonstrated a 10% reduction in largest tumor diameter and 8% reduction in thickness, along with improving subretinal fluid, intraretinal edema, and retinal traction, after 4 weeks of treatment. After 2.5 years of treatment, the patient in Case 2 demonstrated similar margins of her now fibrotic-appearing juxtapapillary lesion with a 45% reduction in thickness, along with resolved subretinal fluid and greatly improved intraretinal fluid and traction. The inferior lesion demonstrated 12.5% reduction in largest diameter, 36% reduction in thickness, and was without active subretinal fluid or exudation. Neither patient demonstrated new lesions while on treatment.
Belzutifan is a promising treatment for retinal hemangioblastoma with the potential for both rapid and sustained tumor regression.
描述贝伐单抗治疗von Hippel-Lindau 综合征相关视网膜血管母细胞瘤后 2 例视网膜血管母细胞瘤消退的情况。
从 2 例 von Hippel-Lindau 相关性视网膜血管母细胞瘤患者的图表和肿瘤图像中提取临床信息。
在病例 1 中，一名 40 岁男性因左眼中部视网膜血管母细胞瘤（位于黄斑颞侧，大小为 2.0×2.0×1.3mm）伴有视网膜内水肿、视网膜下液和轻度视网膜牵引而被诊断为脊柱血管母细胞瘤后，接受了贝伐单抗治疗。在病例 2 中，一名 66 岁女性因右眼 2.0×1.5×1.3mm 视盘旁病变伴有视网膜下液、视网膜内液和鼻侧牵引，以及右眼 4.0×3.5×1.1mm 下中周边病变伴有视网膜下液、视网膜内液和活跃渗出物而就诊。该患者在 NIH 试验中因肾细胞癌接受了 2.5 年的贝伐单抗治疗。病例 1 患者在治疗 4 周后，最大肿瘤直径缩小了 10%，厚度缩小了 8%，同时视网膜下液、视网膜水肿和视网膜牵引情况也得到改善。在接受治疗 2.5 年后，病例 2 患者的视盘旁病变现在呈现出纤维化外观，其厚度缩小了 45%，同时视网膜下液已消失，视网膜内液和牵引情况得到了极大改善。下部病变的最大直径缩小了 12.5%，厚度缩小了 36%，且无活跃的视网膜下液或渗出物。在治疗期间，两名患者均未出现新的病变。
贝伐单抗是治疗视网膜血管母细胞瘤的一种很有前途的方法，具有快速和持续的肿瘤消退潜力。
