PuSH - Publication Server of Helmholtz Zentrum München: Responses to systemic therapy in metastatic pheochromocytoma/paraganglioma: A retrospective multicenter cohort study.

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Fischer, A.* ; Kloos, S.* ; Remde, H.* ; Dischinger, U.* ; Pamporaki, C.* ; Timmers, H.J.L.M.* ; Robledo, M.* ; Fliedner, S.M.J.* ; Wang, K.* ; Maurer, J.* ; Reul, A.* ; Bechmann, N.* ; Hantel, C.* ; Mohr, H. ; Pellegata, N.S. ; Bornstein, S.* ; Kroiss, M.* ; Auernhammer, C.J.* ; Reincke, M.* ; Pacak, K.* ; Grossman, A.B.* ; Beuschlein, F.* ; Nölting, S.*

Responses to systemic therapy in metastatic pheochromocytoma/paraganglioma: A retrospective multicenter cohort study.

Eur. J. Endocrinol. 189, 546-565 (2023)

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Open Access Green as soon as Postprint is submitted to ZB.

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OBJECTIVE: The therapeutic options for metastatic pheochromocytomas/paragangliomas (mPPGLs) include chemotherapy with cyclophosphamide/vincristine/dacarbazine (CVD), temozolomide monotherapy, radionuclide therapies, and tyrosine kinase inhibitors such as sunitinib. The objective of this multicenter retrospective study was to evaluate and compare the responses of mPPGLs including those with pathogenic variants in succinate dehydrogenase subunit B (SDHB), to different systemic treatments. DESIGN: This is a retrospective analysis of treatment responses of mPPGL patients (n = 74) to systemic therapies. METHODS: Patients with mPPGLs treated at 6 specialized national centers were selected based on participation in the ENSAT registry. Survival until detected progression (SDP) and disease-control rates (DCRs) at 3 months were evaluated based on imaging reports. RESULTS: For the group of patients with progressive disease at baseline (83.8% of 74 patients), the DCR with first-line CVD chemotherapy was 75.0% (n = 4, SDP 11 months; SDHB [n = 1]: DCR 100%, SDP 30 months), with somatostatin peptide receptor-based radionuclide therapy (PPRT) 85.7% (n = 21, SDP 17 months; SDHB [n = 10]: DCR 100%, SDP 14 months), with 131I-meta-iodobenzylguanidine (131I-MIBG) 82.6% (n = 23, SDP 43 months; SDHB [n = 4]: DCR 100%, SDP 24 months), with sunitinib 100% (n = 7, SDP 18 months; SDHB [n = 3]: DCR 100%, SDP 18 months), and with somatostatin analogs 100% (n = 4, SDP not reached). The DCR with temozolomide as second-line therapy was 60.0% (n = 5, SDP 10 months; SDHB [n = 4]: DCR 75%, SDP 10 months). CONCLUSIONS: We demonstrate in a real-life clinical setting that all current therapies show reasonable efficacy in preventing disease progression, and this is equally true for patients with germline SDHB mutations.

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Publication type Article: Journal article

Document type Scientific Article

Corresponding Author

Keywords Sdhb ; Cvd ; Mibg ; Prrt ; Metastatic Pheochromocytoma ; Paraganglioma ; Systemic Therapy; Receptor Radionuclide Therapy; Malignant Pheochromocytoma; Paraganglioma; Management; Consensus; Cyclophosphamide; Vincristine; Combination; Lu-177-dotatate; Chemotherapy

ISSN (print) / ISBN 0804-4643

e-ISSN 1479-683X

Journal European Journal of Endocrinology

Quellenangaben Volume: 189, Issue: 5, Pages: 546-565

Publisher BioScientifica

Publishing Place Great Clarendon St, Oxford Ox2 6dp, England

Non-patent literature Publications

Reviewing status Peer reviewed

Institute(s) Institute of Diabetes and Cancer (IDC)

Grants University Medicine Zurich