{"entity":{"id":"paper-pryma-high-specific-activity-i131-mibg-ppgl-jnm-2019","kind":"paper","name":"Efficacy and safety of high-specific-activity 131I-MIBG therapy in advanced pheochromocytoma or paraganglioma","aka":[],"tldr":"A purified radioactive form of MIBG, taken up by adrenaline-producing tumour cells, let a quarter of patients halve their blood pressure medication for at least six months and shrank tumours in about one in five, leading to the first approved radiopharmaceutical for these tumours.","summary":"Open-label single-arm multicentre phase 2 study of high-specific-activity iobenguane I-131 in patients with MIBG-avid unresectable or metastatic pheochromocytoma or paraganglioma, given as up to two therapeutic doses; 68 patients received at least one dose.\n\nAbout a quarter of patients achieved the primary endpoint of at least a 50 percent reduction in antihypertensive medication for six months or more, objective responses occurred in about 22 percent, and most patients had disease control; myelosuppression was the main toxicity. The FDA approved the product (Azedra) in 2018.","asOf":"2026-09-18","links":[{"label":"J Nucl Med 2019","url":"https://doi.org/10.2967/jnumed.118.217463"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/30291194/"}],"tags":[],"related":[],"cancers":["metastatic-ppgl"],"sections":[],"technologies":[],"targets":[],"drugs":["i131-mibg"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["journal-of-nuclear-medicine"],"dependsOn":[],"notes":[],"journal":"Journal of Nuclear Medicine","year":2019,"doi":"10.2967/jnumed.118.217463","pmid":"30291194","authors":"Pryma DA, Chin BB, Noto RB, et al.","paperType":"observational","findings":["At least a 50 percent reduction in antihypertensive medication for six months or longer in about a quarter of patients.","Objective tumour response in about 22 percent; myelosuppression was the main adverse effect."],"whatItMeans":"Radionuclide therapy is a standard option for MIBG-avid metastatic pheochromocytoma and paraganglioma; somatostatin receptor targeted lutetium therapy is the alternative for SSTR-avid disease.","caveats":["Single-arm study with a blood pressure medication endpoint rather than survival.","The commercial product was later withdrawn from the US market for business reasons, limiting access."],"changedPractice":true,"participants":68},"route":"/key-papers/paper-pryma-high-specific-activity-i131-mibg-ppgl-jnm-2019/","neighbours":{"cancer":[{"id":"metastatic-ppgl","kind":"cancer","name":"Metastatic pheochromocytoma and paraganglioma","route":"/cancers/metastatic-ppgl/"}],"drug":[{"id":"i131-mibg","kind":"drug","name":"131I-MIBG (iobenguane I-131) therapy","route":"/drugs/i131-mibg/"}],"journal":[{"id":"journal-of-nuclear-medicine","kind":"journal","name":"Journal of Nuclear Medicine","route":"/journals/journal-of-nuclear-medicine/"}]}}