Perspective review: lessons from successful clinical trials and real-world studies of systemic therapy for metastatic pheochromocytomas and paragangliomas

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dc.catalogadorpau
dc.contributor.authorJimenez, Camilo
dc.contributor.authorBaudrand, Rene
dc.contributor.authorUslar, Thomas
dc.contributor.authorBulzico, Daniel
dc.date.accessioned2025-04-10T19:57:12Z
dc.date.available2025-04-10T19:57:12Z
dc.date.issued2024
dc.description.abstractPheochromocytomas and paragangliomas (PPGLs) are orphan tumors with the potential to spread to distant organs such as the lymph nodes, the skeleton, the lungs, and the liver. These metastatic tumors exhibit high rates of morbidity and mortality due to their frequently large tumor burden, the progression of the disease, and the excessive secretion of catecholamines that lead to cardiovascular disease and gastrointestinal dysmotility. Several molecular drivers responsible for the development of PPGLs have been described over the last 30 years. Although therapeutic options are limited, substantial progress has been made in the recognition of effective systemic therapies for these tumors. Successful clinical trials with radiopharmaceuticals such as high-specific-activity meta-iodobenzylguanidine and tyrosine kinase inhibitors such as cabozantinib and sunitinib have been recently published. This review will discuss the results of these studies and their impact on current clinical practices. In addition, this review will provide valuable information on how to design clinical trials to treat patients with metastatic PPGLs with novel medications.
dc.description.funderTeam NAT Foundation
dc.fechaingreso.objetodigital2025-04-10
dc.fuente.origenWOS
dc.identifier.doi10.1177/17588359241301359
dc.identifier.eissn1758-8359
dc.identifier.issn1758-8340
dc.identifier.urihttps://doi.org/10.1177/17588359241301359
dc.identifier.urihttps://repositorio.uc.cl/handle/11534/103201
dc.identifier.wosidWOS:001360599600001
dc.information.autorucEscuela de Medicina; Baudrand, Rene; 0000-0002-8655-4957; 1024
dc.information.autorucEscuela de Medicina; Uslar, Thomas; S/I; 187161
dc.language.isoen
dc.nota.accesocontenido completo
dc.publisherSAGE PUBLICATIONS LTD
dc.revistaTHERAPEUTIC ADVANCES IN MEDICAL ONCOLOGY
dc.rights.uriacceso abierto
dc.subjectMetastatic pheochromocytoma and paraganglioma
dc.subjectRadiopharmaceuticals
dc.subjectTyrosine kinase inhibitors
dc.subject.ddc610
dc.subject.deweyMedicina y saludes_ES
dc.subject.ods03 Good health and well-being
dc.subject.odspa03 Salud y bienestar
dc.titlePerspective review: lessons from successful clinical trials and real-world studies of systemic therapy for metastatic pheochromocytomas and paragangliomas
dc.typereseña
dc.volumen16
sipa.codpersvinculados1024
sipa.codpersvinculados187161
sipa.trazabilidadWOS;2024-11-30
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