Camilo Jimenez1, Guofan Xu2, Jeena Varghese3, Paul H Graham4, Matthew T Campbell5, Yang Lu2. 1. Department of Endocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center, Unit 1461, 1515 Holcombe Blvd, Houston, TX, 77030, USA. cjimenez@mdanderson.org. 2. Department of Nuclear Medicine, The University of Texas MD Anderson Cancer Center, Unit 1483, 1515 Holcombe Blvd, Houston, TX, 77030, USA. 3. Department of Endocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center, Unit 1461, 1515 Holcombe Blvd, Houston, TX, 77030, USA. 4. Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Unit 1484, 1515 Holcombe Blvd, Houston, TX, 77030, USA. 5. Department of Genitourinary Medical Oncology, The University of Texas MD Anderson Cancer Center, Unit 1374, 1515 Holcombe Blvd, Houston, TX, 77030, USA.
Abstract
PURPOSE OF REVIEW: Multiple therapies with novel mechanisms have been explored in clinical trials for the treatment of metastatic pheochromocytomas and paragangliomas. We review current and future therapies for this disease and provide guidance on how and when to prescribe them based on tumor progression, clinical manifestations, molecular features, and social factors. RECENT FINDINGS: Approximately 60-70% of metastatic pheochromocytomas and paragangliomas express the noradrenaline transporter in their cell membranes. High specific activity iodine-131 metaiodobenzylguanidine has been recently approved by the US Food and Drug Administration for the treatment of metastatic pheochromocytomas and paragangliomas that express the noradrenaline transporter, in patients aged ≥ 12 years. More than 90% of patients treated with this medication exhibit clinical benefits. However, other therapies with novel mechanisms of action are needed to help all patients with this disease. Treatment of metastatic pheochromocytomas and paragangliomas is recommended based on the severity of symptoms, the progression of the disease, and the patient's performance status. Currently available therapies include surgery; systemic chemotherapy with cyclophosphamide, vincristine, and dacarbazine, or with temozolomide; high specific activity iodine-131 metaiodobenzylguanidine; peptide receptor radionuclide therapy; immunotherapy; tyrosine kinase inhibitors; and hypoxia-inducible factor 2 alpha inhibitors. Financial and social factors such as health insurance coverage and disparities also impact current clinical practice in the USA.
PURPOSE OF REVIEW: Multiple therapies with novel mechanisms have been explored in clinical trials for the treatment of metastatic pheochromocytomas and paragangliomas. We review current and future therapies for this disease and provide guidance on how and when to prescribe them based on tumor progression, clinical manifestations, molecular features, and social factors. RECENT FINDINGS: Approximately 60-70% of metastatic pheochromocytomas and paragangliomas express the noradrenaline transporter in their cell membranes. High specific activity iodine-131 metaiodobenzylguanidine has been recently approved by the US Food and Drug Administration for the treatment of metastatic pheochromocytomas and paragangliomas that express the noradrenaline transporter, in patients aged ≥ 12 years. More than 90% of patients treated with this medication exhibit clinical benefits. However, other therapies with novel mechanisms of action are needed to help all patients with this disease. Treatment of metastatic pheochromocytomas and paragangliomas is recommended based on the severity of symptoms, the progression of the disease, and the patient's performance status. Currently available therapies include surgery; systemic chemotherapy with cyclophosphamide, vincristine, and dacarbazine, or with temozolomide; high specific activity iodine-131 metaiodobenzylguanidine; peptide receptor radionuclide therapy; immunotherapy; tyrosine kinase inhibitors; and hypoxia-inducible factor 2 alpha inhibitors. Financial and social factors such as health insurance coverage and disparities also impact current clinical practice in the USA.
Authors: Segolene Hescot; Maria Curras-Freixes; Timo Deutschbein; Anouk van Berkel; Delphine Vezzosi; Laurence Amar; Christelle de la Fouchardière; Nuria Valdes; Fernando Riccardi; Christine Do Cao; Jerome Bertherat; Bernard Goichot; Felix Beuschlein; Delphine Drui; Letizia Canu; Patricia Niccoli; Sandrine Laboureau; Antoine Tabarin; Sophie Leboulleux; Bruna Calsina; Rossella Libé; Antongiulio Faggiano; Martin Schlumberger; Francoise Borson-Chazot; Massimo Mannelli; Anne-Paule Gimenez-Roqueplo; Philippe Caron; Henri J L M Timmers; Martin Fassnacht; Mercedes Robledo; Isabelle Borget; Eric Baudin Journal: J Clin Endocrinol Metab Date: 2019-06-01 Impact factor: 5.958
Authors: Alejandro Roman-Gonzalez; Shouhao Zhou; Montserrat Ayala-Ramirez; Chan Shen; Steven G Waguespack; Mouhammed A Habra; Jose A Karam; Nancy Perrier; Christopher G Wood; Camilo Jimenez Journal: Ann Surg Date: 2018-07 Impact factor: 12.969
Authors: Lauren Fishbein; Jaydira Del Rivero; Tobias Else; James R Howe; Sylvia L Asa; Debbie L Cohen; Patricia L M Dahia; Douglas L Fraker; Karyn A Goodman; Thomas A Hope; Pamela L Kunz; Kimberly Perez; Nancy D Perrier; Daniel A Pryma; Mabel Ryder; Aaron R Sasson; Michael C Soulen; Camilo Jimenez Journal: Pancreas Date: 2021-04-01 Impact factor: 3.327