ECE2024 Poster Presentations Endocrine-Related Cancer (40 abstracts)
Effectiveness of temozolomide treatment in SDHx mutant and wildtype metastatic pheochromocytoma and paraganglioma – results of a European restrospective multicentre study
Hanna Remde 1 , Julien Hadoux 2 , Joakim Crona 3 , Rossella Libe 4 , Eugénie Lim 5 , Guido Di Dalmazi 6 , Emanuela Arvat 7 , Magalie Haissaguerre 8 , Letizia Canu 9 , Gregory Kaltsas 10 , Peter Igaz 11 , Henri Timmers 12 , Svenja Noelting 13 , Marcus Quinkler 14 , Timo Deutschbein 15 , Martin Fassnacht 1 & Kroiss Matthias 16
1University Hospital Würzburg, Würzburg, Germany; 2Institut Gustave Roussy, Villejuif, France; 3University of Uppsala, Uppsala, Sweden; 4Hôpital Cochin-Port Royal, Paris, France; 5St. Bartholomew’s Hospital, London, UK; 6IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy; 7University of Turin, Torino, Italy; 8Bordeaux University, Bordeaux, France; 9University of Florence, Firenze, Italy; 10National and Kapodistrian University of Athens, Athens, Greece; 11Semmelweis University, Budapest, Hungary; 12Radboud University Medical Centre, Nijmegen, Netherlands; 13University Hospital Zürich, Zürich, Switzerland; 14Endokrinologie in Charlottenburg, Berlin, Germany; 15Medicover MVZ Oldenburg, Oldenburg, Germany; 16LMU University Hospital, München, Germany
Background: Pheochromocytomas and paragangliomas (mPPGL) are rare neuroendocrine tumors. Therapeutic options in advanced and irresectable mPPGL are limited. Two small retrospective studies demonstrated the effectiveness of temozolomide in patients with mPPGL and suggested that patients with mutation in the succinate dehydrogenase B (SDHB) gene might benefit more than SDHB wildtype cases.
Aim: To re-evaluate safety and effectiveness of temozolomide in a larger cohort of patients with mPPGL and identify predictors of clinical response.
Methods: Data from patients with mPPGL who had received treatment with temozolomide were collected in an international retrospective setting at 15 reference centres in Europe. The objective response rate (ORR) and non-progression rate (NPR) were the primary outcome measures. The association of SDHB mutation status in and treatment response was assessed using Fisher’s exact test.
Results: Radiological outcome assessment was available for 67 (94.3%) of 71 patients included into the study at data cut-off. Best response was partial response (PR) in 18 patients and stable disease (SD) in 29. The ORR was 27% and the NPR 70%. Germline SDHB mutation was available for 56 patients including 24 (43%) patients with an SDHB mutation and 32 (57%) patients with SDHB wildtype. Objective treatment response was observed in both groups but was significantly more frequent in patients with SDHB mutation (41.6% vs 12.5%, P=0.027). Accordingly, non-progression rate was higher in patients with SDHB mutation compared to those with SDHB wildtype (83% vs 56%, P=0.044).
Conclusion: Treatment with temozolomide in mPPGL can be effective in both patients with SDHB mutation and those with SDHB wildtype. However, objective response rates and non-progression rates are higher in patients carrying an SDHB mutation.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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