Departments of Endocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center.
Excel Diagnostics and Nuclear Oncology Center.
Curr Opin Endocrinol Diabetes Obes. 2020 Jun;27(3):162-169. doi: 10.1097/MED.0000000000000544.
Pheochromocytomas and paragangliomas represent less than 1% of all endocrine tumors. Approximately 15-20% of these tumors are malignant. The definition of malignancy relies on the presence of metastasis. Metastatic pheochromocytomas and paragangliomas are usually advanced, incurable tumors with limited therapeutic options. About 50-60% of these tumors express the noradrenaline transporter in their cell membranes. Recently, the United States Food and Drug Administration approved high-specific-activity iodine 131 metaiodobenzylguanidine (HSA-I-131-MIBG) for the treatment of metastatic pheochromocytomas and paragangliomas that express the noradrenaline transporter. This review reports the benefits and toxicity of HSA-I-131-MIBG, its physical and dosimetric aspects, and radiation safety precautions, as well as its potential therapeutic value for other malignancies (neuroblastoma, gastroenteropancreatic neuroendocrine tumors, and medullary thyroid carcinoma).
A phase 2 clinical trial with HSA-I-131-MIBG reported an impressive clinical benefit rate, acceptable toxicity and long-term benefits.
HSA-I-131-MIBG is an effective medication for metastatic pheochromocytomas and paragangliomas that express the noradrenaline transporter.
嗜铬细胞瘤和副神经节瘤占所有内分泌肿瘤的比例不到 1%。这些肿瘤中约有 15-20%是恶性的。恶性肿瘤的定义依赖于转移的存在。转移性嗜铬细胞瘤和副神经节瘤通常是晚期、无法治愈的肿瘤，治疗选择有限。这些肿瘤中约有 50-60%在其细胞膜上表达去甲肾上腺素转运体。最近，美国食品和药物管理局批准了高比活碘 131 间碘苄胍（HSA-I-131-MIBG）用于治疗表达去甲肾上腺素转运体的转移性嗜铬细胞瘤和副神经节瘤。本综述报告了 HSA-I-131-MIBG 的益处和毒性、物理和剂量学方面以及辐射安全预防措施，以及其对其他恶性肿瘤（神经母细胞瘤、胃肠胰神经内分泌肿瘤和甲状腺髓样癌）的潜在治疗价值。
一项使用 HSA-I-131-MIBG 的 2 期临床试验报告了令人印象深刻的临床获益率、可接受的毒性和长期获益。
HSA-I-131-MIBG 是一种有效的治疗表达去甲肾上腺素转运体的转移性嗜铬细胞瘤和副神经节瘤的药物。
