Efficacy and Safety of High-Specific-Activity 131I-MIBG Therapy in Patients with Advanced Pheochromocytoma or Paraganglioma.
Patients with metastatic or unresectable (advanced) pheochromocytoma and paraganglioma (PPGL) have poor prognoses and few treatment options. This multicenter, phase 2 trial evaluated the efficacy and safety of high-specific-activity 131I-meta-iodobenzylguanidine (HSA 131I-MIBG) in patients with advanced PPGL. Methods: In this open-label, single-arm study, 81 PPGL patients were screened for enrollment, and 74 received a treatment-planning dose of HSA 131I-MIBG. Of these patients, 68 received at least 1 therapeutic dose (∼18.5 GBq) of HSA 131I-MIBG intravenously. The primary endpoint was the proportion of patients with at least a 50% reduction in baseline antihypertensive medication use lasting at least 6 mo. Secondary endpoints included objective tumor response as assessed by Response Evaluation Criteria in Solid Tumors version 1.0, biochemical tumor marker response, overall survival, and safety. Results: Of the 68 patients who received at least 1 therapeutic dose of HSA 131I-MIBG, 17 (25%; 95% confidence interval, 16%-37%) had a durable reduction in baseline antihypertensive medication use. Among 64 patients with evaluable disease, 59 (92%) had a partial response or stable disease as the best objective response within 12 mo. Decreases in elevated (≥1.5 times the upper limit of normal at baseline) serum chromogranin levels were observed, with confirmed complete and partial responses 12 mo after treatment in 19 of 28 patients (68%). The median overall survival was 36.7 mo (95% confidence interval, 29.9-49.1 mo). The most common treatment-emergent adverse events were nausea, myelosuppression, and fatigue. No patients had drug-related acute hypertensive events during or after the administration of HSA 131I-MIBG. Conclusion: HSA 131I-MIBG offers multiple benefits, including sustained blood pressure control and tumor response in PPGL patients.
['Pryma|Daniel A|DA|', 'Chin|Bennett B|BB|', 'Noto|Richard B|RB|', 'Dillon|Joseph S|JS|', 'Perkins|Stephanie|S|', 'Solnes|Lilja|L|', 'Kostakoglu|Lale|L|', 'Serafini|Aldo N|AN|', 'Pampaloni|Miguel H|MH|', 'Jensen|Jessica|J|', 'Armor|Thomas|T|', 'Lin|Tess|T|', 'White|Theresa|T|', 'Stambler|Nancy|N|', 'Apfel|Stuart|S|', 'DiPippo|Vincent A|VA|', 'Mahmood|Syed|S|', 'Wong|Vivien|V|', 'Jimenez|Camilo|C|']
D019797:3-Iodobenzylguanidine D000293:Adolescent D000310:Adrenal Gland Neoplasms D014408:Biomarkers, Tumor D005260:Female D006801:Humans D008297:Male D010235:Paraganglioma D010673:Pheochromocytoma D012449:Safety D016019:Survival Analysis D016896:Treatment Outcome
high-specific-activity 131I-MIBG pheochromocytoma paraganglioma neuroendocrine tumors rare ultra-orphan disease