title: Demographic, tumor and clinical features of clinical trials versus clinical practice patients with HER2-positive early breast cancer: results of a prospective study.
abstract: BACKGROUND
  Several randomized clinical trials (RCTs) have demonstrated the efficacy of trastuzumab-based adjuvant therapy in HER2-positive breast cancer (BC). However, RCT patients may not invariably be representative of patients routinely seen in clinical practice (CP). To address this issue, we compared the clinical and tumor features of RCT and CP patients with HER2-positive BC.
  
  
  METHODS
  From January to December 2012, 650 consecutive patients with HER2-positive early BC, treated in 36 different types of Italian healthcare facilities, were enrolled in this study. Age, treatment, tumor size (T), nodes (N), grade (G), estrogen receptor (ER) and progesterone receptor (PgR) status were prospectively collected in these CP patients. The same data were extracted from the main adjuvant trastuzumab RCTs and pooled using the random-effects model of DerSimonian and Laird. RCT and CP patients were compared by using the Cochran Q statistics.
  
  
  RESULTS
  Versus RCT patients, CP patients were more likely to be older than 50 years (65 vs. 49 %; p < 0.0001) and to have HR (ER and/or PgR)-positive (72 vs. 54 %; p < 0.0001) BC and less likely to have tumor >2 cm (T ≥ 2 cm 39 vs. 59 %; p < 0.0001), positive N (47 vs. 89 %; p < 0.0001) and a high G (61 vs. 67 %; p = 0.0241). CP patients more frequently received adjuvant endocrine therapy (70 vs. 57 %; p < 0.0003) and less frequently adjuvant chemotherapy (97 vs. 99.7 %; p < 0.0001).
  
  
  CONCLUSIONS
  Most tumor and clinical features differed significantly between CP and RCT patients. These data raise concerns about the applicability of RCT results to CP patients.
authors.affiliation: Department of Clinical Medicine and Surgery, University of Naples Federico II, Via S. Pansini 5, 80131, Naples, Italy. grazia.arpino@unina.it.
authors.forename: Grazia
authors.initials: G
authors.lastname: Arpino
authors.affiliation: Medical Oncology, "Santa Chiara" Hospital, University of Pisa, Pisa, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Michelotti
authors.affiliation: Department of Pathology, National Institute for Cancer Research, Genoa, Italy.
authors.forename: Mauro
authors.initials: M
authors.lastname: Truini
authors.affiliation: Investigative Clinical Oncology, Candiolo Cancer Institute, Candiolo, Italy.
authors.forename: Filippo
authors.initials: F
authors.lastname: Montemurro
authors.affiliation: Medical Oncology, University Hospital, Udine, Italy.
authors.forename: Stefania
authors.initials: S
authors.lastname: Russo
authors.affiliation: Service of Preventive Oncology, IRCCS Fondazione "Salvatore Maugeri", Pavia, Italy.
authors.forename: Raffaella
authors.initials: R
authors.lastname: Palumbo
authors.affiliation: Medical Oncology Unit, "S.Orsola-Malpighi" Hospital, Bologna, Italy.
authors.forename: Claudio
authors.initials: C
authors.lastname: Zamagni
authors.affiliation: National Cancer Institute, Bari, Italy.
authors.forename: Agnese
authors.initials: A
authors.lastname: Latorre
authors.affiliation: Preventive Medical Sciences, University of Naples "Federico II", Naples, Italy.
authors.forename: Dario
authors.initials: D
authors.lastname: Bruzzese
authors.affiliation: Medical Oncology, "A. Cardarelli" Hospital, Naples, Italy.
authors.forename: Ferdinando
authors.initials: F
authors.lastname: Riccardi
authors.affiliation: National Cancer Institute Fondazione "G.Pascale", Naples, Italy.
authors.forename: Michelino
authors.initials: M
authors.lastname: De Laurentiis
authors.affiliation: Presidio Evangelico Valdese-ASL TO1, Area Medica, Turin, Italy.
authors.forename: Alessandra
authors.initials: A
authors.lastname: Beano
authors.affiliation: Department of Medical Oncology, Hospital of Prato, Istituto Toscano Tumori, Prato, Italy.
authors.forename: Laura
authors.initials: L
authors.lastname: Biganzoli
authors.affiliation: Department of Medical Oncology, Casa di Cura Poliambulanza, Brescia, Italy.
authors.forename: Alberto
authors.initials: A
authors.lastname: Zaniboni
authors.affiliation: Medical Oncology, Ospedale F. Renzetti, Lanciano, Italy.
authors.forename: Lucio
authors.initials: L
authors.lastname: Laudadio
authors.affiliation: Oncology, Ospedali Riuniti di Trieste, Trieste, Italy.
authors.forename: Maria
authors.initials: M
authors.lastname: Malagoli
authors.affiliation: Oncology, "San Carlo" Hospital, Potenza, Italy.
authors.forename: Domenico
authors.initials: D
authors.lastname: Bilancia
authors.affiliation: Department of Clinical Medicine and Surgery, University of Naples Federico II, Via S. Pansini 5, 80131, Naples, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Schettini
authors.affiliation: Department of Clinical Medicine and Surgery, University of Naples Federico II, Via S. Pansini 5, 80131, Naples, Italy.
authors.forename: Mario
authors.initials: M
authors.lastname: Giuliano
authors.affiliation: Medical Oncology, "San Gerardo" Hospital, Monza, Italy.
authors.forename: Marina Elena
authors.initials: ME
authors.lastname: Cazzaniga
authors.affiliation: Department of Clinical Medicine and Surgery, University of Naples Federico II, Via S. Pansini 5, 80131, Naples, Italy.
authors.forename: Sabino
authors.initials: S
authors.lastname: De Placido
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001943:Breast Neoplasms / Q000453:epidemiology* / Q000235:genetics / Q000473:pathology* / Q000628:therapy*; D005260:Female; D006801:Humans; D007558:Italy / Q000453:epidemiology; D015201:Meta-Analysis as Topic; D008875:Middle Aged; D010818:Practice Patterns, Physicians' / Q000706:statistics & numerical data*; D016032:Randomized Controlled Trials as Topic; D018719:Erb-b2 Receptor Tyrosine Kinases / Q000235:genetics / Q000378:metabolism
chemical_list: D018719:Erb-b2 Receptor Tyrosine Kinases
keywords: Adjuvant therapy; Clinical practice; Early breast cancer; HER2; Trastuzumab