One-year progression-free survival of therapy-naive patients with malignant pheochromocytoma and paraganglioma.

Abstract

CONTEXT:

The natural history of malignant pheochromocytoma or paragangliomas (MPP) remain unknown.

OBJECTIVE:

The primary aim of this study was to define progression-free survival at 1 year in therapy-naive patients with MPP. Secondary objectives were to characterize MPP and to look for prognostic parameters for progression at 1 year.

DESIGN AND SETTING:

The files of MPP followed up between January 2001 and January 2011 in two French Endocrine Networks were retrospectively reviewed. Therapy-naive patients were enrolled.

MAIN OUTCOME MEASURES:

The main outcome was progression-free survival at 1 year in therapy-naive MPP patients according to Response Evaluation Criteria In Solid Tumors 1.1 criteria.

RESULTS:

Ninety files (46 men, 44 women, mean age of 47.5 ± 15 years) were reviewed on site by one investigator. MPP characteristics were as follows: presence of an adrenal primary, a mitotic count exceeding 5 per high power field, hypertension, inherited disease, and presence of bone metastases in 50%, 22%, 60%, 49%, and 56% patients, respectively. Fifty-seven of the 90 patients with MPP (63%) were classified as therapy-naive. The median follow-up of these 57 patients was 2.4 years (range, 0.4-5.7). At 1 year, progression-free survival was 46% (CI 95: 33-59). Twenty-six of 30 (87%) patients with progression at 1 year had exhibited progressive disease at the first imaging workup performed after a median of 5.7 months. No prognostic parameter was identified.

CONCLUSIONS:

Half of the therapy-naive patients with MPP achieved stable disease at 1 year. In symptom-free patients with MPP, a wait-and-see antitumor policy seems appropriate as first line. Modality for a prospective follow-up is proposed.

• Authors: Ségolène Hescot, Sophie Leboulleux, Laurence Amar, Delphine Vezzosi, Isabelle Borget, Claire Bournaud-Salinas, Christelle de la Fouchardiere, Rossella Libé, Christine Do Cao, Patricia Niccoli, Antoine Tabarin, Isabelle Raingeard, Cécile Chougnet, Sophie Giraud, Anne-Paule Gimenez-Roqueplo, Jacques Young, Francoise Borson-Chazot, Jérôme Bertherat, Jean-Louis Wemeau, Xavier Bertagna, Pierre-Francois Plouin, Martin Schlumberger, Eric Baudin, French group of Endocrine and Adrenal tumors (Groupe des Tumeurs Endocrines-REseau NAtional des Tumeurs ENdocrines and COrtico-MEdullo Tumeurs Endocrines networks)
• Journal: The Journal of clinical endocrinology and metabolism (J Clin Endocrinol Metab) Vol. 98 Issue 10 Pg. 4006-12 (Oct 2013) ISSN: 1945-7197 [Electronic] United States
• PMID: 23884775 (Publication Type: Journal Article)

Topics

• Adrenal Gland Neoplasms (mortality, pathology)
• Adult
• Disease-Free Survival
• Female
• Humans
• Male
• Middle Aged
• Paraganglioma (mortality, pathology)
• Pheochromocytoma (mortality, pathology)
• Prognosis
• Retrospective Studies