Pheochromocytoma and paraganglioma are neuroendocrine neoplasms, originating in the adrenal medulla and in parasympathetic and sympathetic autonomic nervous system ganglia, respectively. They usually present as localized tumours curable with surgery. However, these tumours may exhibit heterogeneous clinical course, ranging from no/minimal progression to aggressive (progressive/metastatic) behavior. For this setting of patients, current therapies are unsatisfactory. Immune checkpoint inhibitors have shown outstanding results for several types of solid cancers. We therefore aimed to summarize and discuss available data on efficacy and safety of current FDA-approved immune checkpoint inhibitors in patients with pheochromocytoma and paraganglioma. After an extensive search, we found 15 useful data sources (four full-published articles, four supplements of scientific journals, seven ongoing registered clinical trials). The data we detected, even with the limit of the small number of patients treated, make a great expectation on the therapeutic use of immune checkpoint inhibitors. Besides, the newly detected predictors of response will (hopefully) be of great helps in selecting the subset of patients that might benefit the most from this class of drugs. Finally, new trials are in the starting blocks, and they are expected to shed in the next future new light on a therapy, which is considered a milestone in oncology.

Emerging therapies in pheochromocytoma and paraganglioma. immune checkpoint inhibitors in the starting blocks / Fanciulli, Giuseppe; Di Molfetta, Sergio; Dotto, Andrea; Florio, Tullio; Feola, Tiziana; Rubino, Manila; de Cicco, Federica; Colao, Annamaria; Faggiano, Antongiulio; Nike, Group; Albertelli, Manuela; Altieri, Barbara; Barrea, Luigi; Bottiglieri, Filomena; Campione, Severo; Dicitore, Alessandra; Ferone, Diego; Ferraù, Francesco; Grossrubatscher, Erika; Gallo, Marco; Giannetta, Elisa; Grillo, Federica; Guadagno, Elia; Guarnotta, Valentina; Isidori, Andrea M; Lania, Andrea; Lenzi, Andrea; Lo Calzo, Fabio; Malandrino, Pasquale; Messina, Erika; Modica, Roberta; Muscogiuri, Giovanna; Pizza, Genoveffa; Pes, Luca; Pofi, Riccardo; Puliani, Giulia; Rainone, Carmen; Razzore, Paola; Rizza, Laura; Ruggieri, Rosa Maria; Sbardella, Emilia; Sesti, Franz; Venneri, Mary Anna; Vitale, Giovanni; Zatelli, Maria Chiara. - In: JOURNAL OF CLINICAL MEDICINE. - ISSN 2077-0383. - 10:1(2020), pp. 1-20. [10.3390/jcm10010088]

Emerging therapies in pheochromocytoma and paraganglioma. immune checkpoint inhibitors in the starting blocks

Fanciulli, Giuseppe
;
Feola, Tiziana;Faggiano, Antongiulio;Giannetta, Elisa;Grillo, Federica;Isidori, Andrea M;Lenzi, Andrea;Pofi, Riccardo;Puliani, Giulia;Rizza, Laura;Sbardella, Emilia;Sesti, Franz;Venneri, Mary Anna;Vitale, Giovanni;Zatelli, Maria Chiara
2020

Abstract

Pheochromocytoma and paraganglioma are neuroendocrine neoplasms, originating in the adrenal medulla and in parasympathetic and sympathetic autonomic nervous system ganglia, respectively. They usually present as localized tumours curable with surgery. However, these tumours may exhibit heterogeneous clinical course, ranging from no/minimal progression to aggressive (progressive/metastatic) behavior. For this setting of patients, current therapies are unsatisfactory. Immune checkpoint inhibitors have shown outstanding results for several types of solid cancers. We therefore aimed to summarize and discuss available data on efficacy and safety of current FDA-approved immune checkpoint inhibitors in patients with pheochromocytoma and paraganglioma. After an extensive search, we found 15 useful data sources (four full-published articles, four supplements of scientific journals, seven ongoing registered clinical trials). The data we detected, even with the limit of the small number of patients treated, make a great expectation on the therapeutic use of immune checkpoint inhibitors. Besides, the newly detected predictors of response will (hopefully) be of great helps in selecting the subset of patients that might benefit the most from this class of drugs. Finally, new trials are in the starting blocks, and they are expected to shed in the next future new light on a therapy, which is considered a milestone in oncology.
2020
atezolizumab; avelumab; cemiplimab; durvalumab; immune checkpoint inhibitors; ipilimumab; nivolumab; paraganglioma; pembrolizumab; pheochromocytoma
01 Pubblicazione su rivista::01g Articolo di rassegna (Review)
Emerging therapies in pheochromocytoma and paraganglioma. immune checkpoint inhibitors in the starting blocks / Fanciulli, Giuseppe; Di Molfetta, Sergio; Dotto, Andrea; Florio, Tullio; Feola, Tiziana; Rubino, Manila; de Cicco, Federica; Colao, Annamaria; Faggiano, Antongiulio; Nike, Group; Albertelli, Manuela; Altieri, Barbara; Barrea, Luigi; Bottiglieri, Filomena; Campione, Severo; Dicitore, Alessandra; Ferone, Diego; Ferraù, Francesco; Grossrubatscher, Erika; Gallo, Marco; Giannetta, Elisa; Grillo, Federica; Guadagno, Elia; Guarnotta, Valentina; Isidori, Andrea M; Lania, Andrea; Lenzi, Andrea; Lo Calzo, Fabio; Malandrino, Pasquale; Messina, Erika; Modica, Roberta; Muscogiuri, Giovanna; Pizza, Genoveffa; Pes, Luca; Pofi, Riccardo; Puliani, Giulia; Rainone, Carmen; Razzore, Paola; Rizza, Laura; Ruggieri, Rosa Maria; Sbardella, Emilia; Sesti, Franz; Venneri, Mary Anna; Vitale, Giovanni; Zatelli, Maria Chiara. - In: JOURNAL OF CLINICAL MEDICINE. - ISSN 2077-0383. - 10:1(2020), pp. 1-20. [10.3390/jcm10010088]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/1488791
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