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Demographic, tumor and clinical features of clinical trials versus clinical practice patients with HER2-positive early breast cancer: results of a prospective study

  • Original Article – Clinical Oncology
  • Published:
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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.

Patients and 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.

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Abbreviations

RCT:

Randomized clinical trials

CT:

Clinical trials

HER2:

Human epidermal growth factor receptor 2

BC:

Breast cancer

CP:

Clinical practice

ER:

Estrogen receptor

PgR:

Progesterone receptor

HR:

Hormone receptors

T:

Tumor size

N:

Nodal status

G:

Grading

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Acknowledgments

We gratefully thank CR technology S.r.l. in the person of Luciano Di Pierno, for designing the online database and extracting all patient’s data for statistical analysis. We also thank Ms. Jane Gilder for her punctual revision and correction of the paper draft.

Author’s contributions

G.A., M.E.C., S.D.P., L.B., A.M. and C.Z. conceived of the study and participated in its design. All authors contributed in enrolling patients and collecting data. D.B. performed the statistical analysis. G.A., M.G. and F.S. wrote the manuscript. All authors read and approved the final manuscript.

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Authors and Affiliations

  1. Department of Clinical Medicine and Surgery, University of Naples Federico II, Via S. Pansini 5, 80131, Naples, Italy

    Grazia Arpino, Francesco Schettini, Mario Giuliano & Sabino De Placido

  2. Medical Oncology, “Santa Chiara” Hospital, University of Pisa, Pisa, Italy

    Andrea Michelotti

  3. Department of Pathology, National Institute for Cancer Research, Genoa, Italy

    Mauro Truini

  4. Investigative Clinical Oncology, Candiolo Cancer Institute, Candiolo, Italy

    Filippo Montemurro

  5. Medical Oncology, University Hospital, Udine, Italy

    Stefania Russo

  6. Service of Preventive Oncology, IRCCS Fondazione “Salvatore Maugeri”, Pavia, Italy

    Raffaella Palumbo

  7. Medical Oncology Unit, “S.Orsola-Malpighi” Hospital, Bologna, Italy

    Claudio Zamagni

  8. National Cancer Institute, Bari, Italy

    Agnese Latorre

  9. Preventive Medical Sciences, University of Naples “Federico II”, Naples, Italy

    Dario Bruzzese

  10. Medical Oncology, “A. Cardarelli” Hospital, Naples, Italy

    Ferdinando Riccardi

  11. National Cancer Institute Fondazione “G.Pascale”, Naples, Italy

    Michelino De Laurentiis

  12. Presidio Evangelico Valdese-ASL TO1, Area Medica, Turin, Italy

    Alessandra Beano

  13. Department of Medical Oncology, Hospital of Prato, Istituto Toscano Tumori, Prato, Italy

    Laura Biganzoli

  14. Department of Medical Oncology, Casa di Cura Poliambulanza, Brescia, Italy

    Alberto Zaniboni

  15. Medical Oncology, Ospedale F. Renzetti, Lanciano, Italy

    Lucio Laudadio

  16. Oncology, Ospedali Riuniti di Trieste, Trieste, Italy

    Maria Malagoli

  17. Oncology, “San Carlo” Hospital, Potenza, Italy

    Domenico Bilancia

  18. Medical Oncology, “San Gerardo” Hospital, Monza, Italy

    Marina Elena Cazzaniga

Authors
  1. Grazia Arpino
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  2. Andrea Michelotti
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  3. Mauro Truini
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  4. Filippo Montemurro
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  5. Stefania Russo
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  6. Raffaella Palumbo
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  7. Claudio Zamagni
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  8. Agnese Latorre
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  9. Dario Bruzzese
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  10. Ferdinando Riccardi
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  11. Michelino De Laurentiis
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  12. Alessandra Beano
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  13. Laura Biganzoli
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  14. Alberto Zaniboni
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  15. Lucio Laudadio
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  16. Maria Malagoli
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  17. Domenico Bilancia
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  18. Francesco Schettini
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  19. Mario Giuliano
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  20. Marina Elena Cazzaniga
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  21. Sabino De Placido
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Corresponding author

Correspondence to Grazia Arpino.

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Conflict of interest

The authors themselves financed this study and declared no conflict of interests.

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Arpino, G., Michelotti, A., Truini, M. et al. Demographic, tumor and clinical features of clinical trials versus clinical practice patients with HER2-positive early breast cancer: results of a prospective study. J Cancer Res Clin Oncol 142, 669–678 (2016). https://doi.org/10.1007/s00432-015-2033-z

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  • DOI: https://doi.org/10.1007/s00432-015-2033-z

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