Concordance of real-world versus conventional progression-free survival from a phase 3 trial of endocrine therapy as first-line treatment for metastatic breast cancer

There is growing interest in leveraging real-world data to complement knowledge gained from randomized clinical trials and inform the design of prospective randomized studies in oncology. The present study compared clinical outcomes in women with metastatic breast cancer who received letrozole as fi...

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Bibliographic Details
Published in:PloS one 2020-04, Vol.15 (4), p.e0227256-e0227256
Main Authors: Huang Bartlett, Cynthia, Mardekian, Jack, Cotter, Matthew James, Huang, Xin, Zhang, Zhe, Parrinello, Christina M, Bourla, Ariel Bulua
Format: Article
Language:English
Subjects:
Adult
Antineoplastic Agents, Hormonal - therapeutic use
Aromatase Inhibitors - therapeutic use
Breast cancer
Breast Neoplasms - drug therapy
Breast Neoplasms - mortality
Breast Neoplasms - pathology
Clinical medicine
Clinical trials
Cyclin-dependent kinases
Datasets
Disease
Electronic health records
Electronic Health Records - statistics & numerical data
Electronic medical records
Endocrine therapy
Epidermal growth factor
Female
Health services
Humans
Letrozole - therapeutic use
Medical diagnosis
Medical research
Medicine and Health Sciences
Metastases
Metastasis
Oncology
Patients
Post-menopause
Progression-Free Survival
Randomization
Receptor, ErbB-2 - metabolism
Receptors, Estrogen - metabolism
Receptors, Progesterone - metabolism
Regression analysis
Research and Analysis Methods
Response Evaluation Criteria in Solid Tumors
Solid tumors
Statistical analysis
Survival
Tumors
United States - epidemiology
Weighting
Womens health
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Summary:There is growing interest in leveraging real-world data to complement knowledge gained from randomized clinical trials and inform the design of prospective randomized studies in oncology. The present study compared clinical outcomes in women with metastatic breast cancer who received letrozole as first-line monotherapy in oncology practices across the United States versus patients in the letrozole-alone cohort of the PALOMA-2 phase 3 trial. The real-world cohort (N = 107) was derived from de-identified patient data from the Flatiron Health electronic health record database. The clinical trial cohort (N = 222) comprised postmenopausal women in the letrozole-alone arm of PALOMA-2. Patients in the real-world cohort received letrozole monotherapy per labeling and clinical judgment; patients in PALOMA-2 received letrozole 2.5 mg/d, continuous. Real-world survival and response rates were based on evidence of disease burden curated from clinician notes, radiologic reports, and pathology reports available in the electronic health record. Progression-free survival and objective response rate in PALOMA-2 were based on Response Evaluation Criteria in Solid Tumors v1.1. Concordance of survival and response rates were retrospectively assessed using inverse probability of treatment weighting-adjusted Cox regression analysis. Inverse probability of treatment weighting-adjusted Cox regression results showed similar median progression-free survival in the real-world and PALOMA-2 cohorts (18.4 and 16.6 months, respectively): the hazard ratio using real-world data as reference was 1.04 (95% CI, 0.69-1.56). No significant difference was observed in response rates: 41.8% in the real-world cohort vs 39.4% in the PALOMA-2 cohort (odds ratio using real-world data as reference: 0.91 [95% CI, 0.57-1.44]). These findings indicate that data abstracted from electronic health records with proper quality controls can yield meaningful information on clinical outcomes. These data increase confidence in the use of real-world assessments of progression and response as efficacy endpoints. Trial registration NCT01740427; Funding: Pfizer.
ISSN:1932-6203
1932-6203
DOI:10.1371/journal.pone.0227256