title: FOLFOX-4 chemotherapy for patients with unresectable or relapsed peritoneal pseudomyxoma.
abstract: OBJECTIVE
  The standard treatment of peritoneal pseudomyxoma is based on cytoreductive surgery combined with hyperthermic intraperitoneal chemotherapy (HIPEC). The establishment of newer systemic treatments is an unmet clinical need for unresectable or relapsed peritoneal pseudomyxoma. The aim of our study was to assess the activity of chemotherapy with 5-fluorouracil and oxaliplatin (FOLFOX-4 regimen) in terms of response rate in this subset of patients.
  
  
  METHODS
  Patients were included in a single-center, observational study and treated with FOLFOX-4 administered every 2 weeks for up to 12 cycles or until progressive disease or unacceptable toxicity.
  
  
  RESULTS
  Twenty consecutive patients were reviewed from July 2011 to September 2013. Only partial responses were observed, with an objective response rate of 20%. Median progression-free survival and overall survival were 8 months and 26 months, respectively. Two patients were able to undergo laparotomy with complete cytoreduction and HIPEC in one case. Safety data for FOLFOX-4 were consistent with the literature. By means of a mutant enriched polymerase chain reaction, KRAS mutation was found in 16 of 19 cases (84%), and MGMT promoter methylation was found in 8 (42%, all KRAS mutant).
  
  
  CONCLUSIONS
  FOLFOX-4 chemotherapy is tolerable and active in patients with peritoneal pseudomyxoma when disease is deemed unresectable or relapsed after peritonectomy and HIPEC. The identification of predictive biomarkers, such as KRAS for resistance to anti-epidermal growth factor receptor monoclonal antibodies and MGMT for response to temozolomide, is a priority for the development of evidence-based treatment strategies for peritoneal pseudomyxoma.
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy filippo.pietrantonio@istitutotumori.mi.it.
authors.forename: Filippo
authors.initials: F
authors.lastname: Pietrantonio
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Claudia
authors.initials: C
authors.lastname: Maggi
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Fanetti
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Roberto
authors.initials: R
authors.lastname: Iacovelli
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Maria
authors.initials: M
authors.lastname: Di Bartolomeo
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Ricchini
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Marcello
authors.initials: M
authors.lastname: Deraco
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Federica
authors.initials: F
authors.lastname: Perrone
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Dario
authors.initials: D
authors.lastname: Baratti
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Shigeki
authors.initials: S
authors.lastname: Kusamura
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Tamborini
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Alessandra
authors.initials: A
authors.lastname: Castano
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Paola Valentina
authors.initials: PV
authors.lastname: Consonni
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Ilaria
authors.initials: I
authors.lastname: Bossi
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Cecilia
authors.initials: C
authors.lastname: Gavazzi
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Massimo
authors.initials: M
authors.lastname: Milione
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Pelosi
authors.affiliation: Medical Oncology Department, Surgery Department, Pathology and Molecular Biology Department, Scientific Directorate, and Clinical Nutrition Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Filippo
authors.initials: F
authors.lastname: de Braud
mesh_terms: D000328:Adult; D000368:Aged; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage* / Q000009:adverse effects; D019175:DNA Methylation / Q000235:genetics; D015254:DNA Modification Methylases / Q000235:genetics; D045643:DNA Repair Enzymes / Q000235:genetics; D018572:Disease-Free Survival; D005260:Female; D005472:Fluorouracil / Q000008:administration & dosage / Q000009:adverse effects; D005500:Follow-Up Studies; D006801:Humans; D002955:Leucovorin / Q000008:administration & dosage / Q000009:adverse effects; D008297:Male; D008875:Middle Aged; D009944:Organoplatinum Compounds / Q000008:administration & dosage / Q000009:adverse effects; D010534:Peritoneal Neoplasms / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D011518:Proto-Oncogene Proteins / Q000235:genetics; D016283:Proto-Oncogene Proteins p21(ras); D011553:Pseudomyxoma Peritonei / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D016896:Treatment Outcome; D025521:Tumor Suppressor Proteins / Q000235:genetics; D018631:ras Proteins / Q000235:genetics
chemical_list: C117307:KRAS protein, human; D009944:Organoplatinum Compounds; D011518:Proto-Oncogene Proteins; D025521:Tumor Suppressor Proteins; D015254:DNA Modification Methylases; C515491:MGMT protein, human; D016283:Proto-Oncogene Proteins p21(ras); D018631:ras Proteins; D045643:DNA Repair Enzymes; D002955:Leucovorin; D005472:Fluorouracil
keywords: Appendiceal cancer; Chemotherapy; FOLFOX-4; Peritoneal pseudomyxoma