Progression-free survival without treatment of malignant pheochromocytoma and paraganglioma at one year
S. Hescot1, S. Leboulleux1, L. Amar2, I. Borget1, D. Vezzosi9, R. Libé3, P. Niccoli5, F. Borson-Chazot7, C. Do Cao6, A. Tabarin4, C. de la Fouchardiere10, I. Raingeard11, C. Chougnet1, A. Gimenez-Roqueplo2, J. Young8, J. Bertherat3, J. Wémeau6, X. Bertagna3, M. Schlumberger1, P. Plouin2 & E. Baudin1
Pheochromocytoma and paraganglioma are defined as malignant by the occurence of metastasis in extra paraganglionic sites. The natural evolution of these tumors remains unknown.
The aim of our retrospective study was to define the progression-free survival (PFS) of untreated Malignant Pheochromocytoma and Paraganglioma (MPP) at 12 months (12 m-PFS) and to look for prognostic markers of 12 m-PFS. We analyzed clinical parameters of patients followed within 8 centers of the INCa-COMETE and GTE-RENATEN French networks. Patients were included whether they had a diagnosis of metastasis between 01/01/2001 and 01/01/2011. Progression was defined according to RECIST 1.1 for morphologic imaging and by the appearance of new lesions on scintigraphic imaging.
Ninety-three files were reviewed on site. Among them, 57 untreated patients for whom a follow-up strategy was first proposed, were included (34 males, 23 females), with a median age of 51 years (1780). Median time since initial diagnosis was 17 months (0341). Primary tumors were 47% pheochromocytomas (n=27), 40% abdominal paragangliomas (n=23) and 13% head and neck paragangliomas (n=7). A pheochromocytoma/paraganglioma genetic test was performed in 54 patients. None mutation was identified in 27 of the patients (50%) whereas 20 patients (37%) carried a SDHB mutation, one patient had neurofibromatosis type 1, one von Hippel-Lindau disease, two a SDHD- and two a SDHC-hereditary paraganglioma. Median follow-up was 2.4 years, (range=0.45.7). 12 m-PFS without treatment was assessed to 46% (3359). No significant prognostic markers of 12m-PFS were found. Untreated patients presented more frequent peritoneal metastases and less frequent bone metastases than directly-treated patients (P<0.05).
Forty-six percent of MPP have a stable disease without treatment at 1 year. If patients are asymptomatic, we recommend a first imaging work-up at 3 months, a wait-and-see policy and/or local treatments in the absence of progression on imaging.
Declaration of interest: The authors declare that there is no conflict of interest that could be perceived as prejudicing the impartiality of the research project.
Funding: This research did not receive any specific grant from any funding agency in the public, commercial or not-for-profit sector.