title: One-year progression-free survival of therapy-naive patients with malignant pheochromocytoma and paraganglioma.
abstract: BACKGROUND
  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.
  
  
  METHODS
  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.
  
  
  METHODS
  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.affiliation: Department of Nuclear Medicine and Endocrine Tumors, Institut Gustave Roussy and University Paris-Sud, 114 rue Edouard Vaillant, 94805 Villejuif Cedex, France. baudin@igr.fr.
authors.forename: Ségolène
authors.initials: S
authors.lastname: Hescot
authors.forename: Sophie
authors.initials: S
authors.lastname: Leboulleux
authors.forename: Laurence
authors.initials: L
authors.lastname: Amar
authors.forename: Delphine
authors.initials: D
authors.lastname: Vezzosi
authors.forename: Isabelle
authors.initials: I
authors.lastname: Borget
authors.forename: Claire
authors.initials: C
authors.lastname: Bournaud-Salinas
authors.forename: Christelle
authors.initials: C
authors.lastname: de la Fouchardiere
authors.forename: Rossella
authors.initials: R
authors.lastname: Libé
authors.forename: Christine
authors.initials: C
authors.lastname: Do Cao
authors.forename: Patricia
authors.initials: P
authors.lastname: Niccoli
authors.forename: Antoine
authors.initials: A
authors.lastname: Tabarin
authors.forename: Isabelle
authors.initials: I
authors.lastname: Raingeard
authors.forename: Cécile
authors.initials: C
authors.lastname: Chougnet
authors.forename: Sophie
authors.initials: S
authors.lastname: Giraud
authors.forename: Anne-Paule
authors.initials: AP
authors.lastname: Gimenez-Roqueplo
authors.forename: Jacques
authors.initials: J
authors.lastname: Young
authors.forename: Francoise
authors.initials: F
authors.lastname: Borson-Chazot
authors.forename: Jérôme
authors.initials: J
authors.lastname: Bertherat
authors.forename: Jean-Louis
authors.initials: JL
authors.lastname: Wemeau
authors.forename: Xavier
authors.initials: X
authors.lastname: Bertagna
authors.forename: Pierre-Francois
authors.initials: PF
authors.lastname: Plouin
authors.forename: Martin
authors.initials: M
authors.lastname: Schlumberger
authors.forename: Eric
authors.initials: E
authors.lastname: Baudin
mesh_terms: D000310:Adrenal Gland Neoplasms / Q000401:mortality* / Q000473:pathology; D000328:Adult; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010235:Paraganglioma / Q000401:mortality* / Q000473:pathology; D010673:Pheochromocytoma / Q000401:mortality* / Q000473:pathology; D011379:Prognosis; D012189:Retrospective Studies