High-specific-activity iodine 131 metaiodobenzylguanidine for the treatment of metastatic pheochromocytoma or paraganglioma: a novel therapy for an orphan disease

• Published in: Current opinion in endocrinology, diabetes, and obesity Vol. 27; no. 3; pp. 162 - 169
• Main Authors: Jimenez, Camilo, Núñez, Rodolfo, Wendt, Richard
• Format: Journal Article
• Language: English
• Published: England Wolters Kluwer Health, Inc. All rights reserved 01-06-2020
• Subjects: 3-Iodobenzylguanidine - therapeutic use; Adrenal Gland Neoplasms - pathology; Adrenal Gland Neoplasms - radiotherapy; Carcinoma, Neuroendocrine - pathology; Carcinoma, Neuroendocrine - radiotherapy; Humans; Intestinal Neoplasms - pathology; Intestinal Neoplasms - radiotherapy; Iodine Radioisotopes - therapeutic use; Neoplasm Metastasis; Neuroblastoma - pathology; Neuroblastoma - radiotherapy; Neuroendocrine Tumors - pathology; Neuroendocrine Tumors - radiotherapy; Pancreatic Neoplasms - pathology; Pancreatic Neoplasms - radiotherapy; Paraganglioma - pathology; Paraganglioma - radiotherapy; Pheochromocytoma - pathology; Pheochromocytoma - radiotherapy; Rare Diseases - radiotherapy; Stomach Neoplasms - pathology; Stomach Neoplasms - radiotherapy; Therapies, Investigational - methods; Thyroid Neoplasms - pathology; Thyroid Neoplasms - radiotherapy
• ISSN: 1752-296X; 1752-2978
• 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.