Abstract
BackgroundParagangliomas (PGLs) are neuroendocrine neoplasms arising from chromaffin cells of sympathetic or parasympathetic paraganglia. Systemic therapies have been used only in metastatic PGLs. Antiangiogenic agents, such as sunitinib, could be a viable therapeutic choice in the subgroup of patients with SDH-positive PGLs. We describe the case of a man with Familial Paraganglioma Syndrome type 1 (FPGL) related to a novel mutation in SDHD gene treated with sunitinib. Furthermore, we performed a systematic review of the literature aimed to address the following question: is sunitinib treatment effective in patients with advanced/progressive/metastatic PGL?MethodsWe performed a data search using MEDLINE, Cochrane Library, and Scopus between April 2019 and September 2020. We included studies reporting data on clinical or biological characteristics, or clinical outcomes of patients with PGLs treated with sunitinib.ResultsThe search leaded to the selection of 25 publications. Data from case reports and case series showed that disease control rate (DCR = stable disease + partial response + complete response) was achieved in 34.7% of cases under sunitinib treatment. In 39% of patients DCR was followed by progressive disease (PD) or tumor relapse, 26.1% patients showed PD. Data from clinical trials showed that DCR was 83%, and the median progression free survival was 13.4 months.DiscussionData from the present literature review suggested that sunitinib could be a viable therapeutic option in advanced/progressive/metastatic inoperable PGLs. However, further trials on the efficacy of sunitinib in FPGL and sporadic PGL are needed.
Highlights
Paragangliomas (PGLs) are neuroendocrine neoplasms (NENs) arising from chromaffin cells of sympathetic or parasympathetic paraganglia [1]
Of the 34 remaining publications, nine were excluded after full text assessment because they did not meet all the eligibility criteria. This process leaded to the selection of 25 publications (Figure 3) [16, 20,21,22,23,24,25,26,27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43]
In our review a significant radiographic DCR was achieved in 34.7% of cases (SD in 17.4%, partial response (PR) in 13%, and complete response (CR) in 4.3%)
Summary
Paragangliomas (PGLs) are neuroendocrine neoplasms (NENs) arising from chromaffin cells of sympathetic or parasympathetic paraganglia [1]. A germline mutation is found in approximatively 40% of these tumors [2] Both in sporadic and inherited PGLs, it is possible to evidence two different pathogenetic pathways: alterations in proteins associated to Krebs cycle and hypoxia signaling (cluster I), and alteration in kinase signaling (cluster II) [3]. Paragangliomas (PGLs) are neuroendocrine neoplasms arising from chromaffin cells of sympathetic or parasympathetic paraganglia. Systemic therapies have been used only in metastatic PGLs. Systemic therapies have been used only in metastatic PGLs Antiangiogenic agents, such as sunitinib, could be a viable therapeutic choice in the subgroup of patients with SDH-positive PGLs. We describe the case of a man with Familial Paraganglioma Syndrome type 1 (FPGL) related to a novel mutation in SDHD gene treated with sunitinib.
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