title: Impact of clinical trial on survival outcomes.
abstract: The number of patients with breast cancer who participate in therapeutic clinical trials remains low. One reason is a lack of opportunity; another is health care providers who do not recommend trials because they fear poorer outcome from the use of new drugs. Thus, we compared survival outcome in patients with metastatic breast cancer (MBC) who participated in first-line therapeutic clinical trials with outcome in patients who had never enrolled in a clinical trial and received only standard care. We hypothesized that first-line therapeutic clinical trials does not have a negative survival outcome. We reviewed the records of patients with MBC who were treated at MD Anderson Cancer Center between January 2000, and December 2010. The medical records of 5501 patients with MBC were screened, and 652 patients-285 in the trial arm and 367 in the control arm-met our specific eligible criteria. The median follow-up of our cohort was 7.16 years (95 % confidence interval [CI] 6.53-7.64 years). Among the global population, no significant differences in progression-free survival (PFS) or overall survival (OS) were observed between the treatment arms: for the clinical trial cohort, median PFS was 7 months (95 % CI 5.72-8.71 months), and median OS was 28.48 months (95 % CI 22.70-34.60 months). For the control cohort, median PFS was 10.02 months (95 % CI 7.13-11.99 months), and median OS was 28.71 months (95 % CI 24.41-31.31 months) (P = .089 and .335, respectively). Enrollment in first-line MBC therapeutic clinical trials does not result in less favorable survival outcome than that in MBC patients who never enrolled in a clinical trial.
authors.affiliation: Department of Breast Medical Oncology, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX, 77030-4009, USA.|Department of Medical Oncology, Eugène Marquis Cancer Center, Rennes, France.
authors.forename: Fanny
authors.initials: F
authors.lastname: Le Du
authors.affiliation: Department of Breast Medical Oncology, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX, 77030-4009, USA.|Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Takeo
authors.initials: T
authors.lastname: Fujii
authors.affiliation: Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Minjeong
authors.initials: M
authors.lastname: Park
authors.affiliation: Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Diane
authors.initials: D
authors.lastname: Liu
authors.affiliation: Department of Breast Medical Oncology, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX, 77030-4009, USA.
authors.forename: Limin
authors.initials: L
authors.lastname: Hsu
authors.affiliation: Department of Breast Medical Oncology, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX, 77030-4009, USA.|Department of Systems Biology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Ana M
authors.initials: AM
authors.lastname: Gonzalez-Angulo
authors.affiliation: Department of Breast Medical Oncology, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX, 77030-4009, USA. nueno@mdanderson.org.
authors.forename: Naoto T
authors.initials: NT
authors.lastname: Ueno
mesh_terms: D000328:Adult; D001943:Breast Neoplasms / Q000523:psychology / Q000628:therapy*; D002986:Clinical Trials as Topic / Q000523:psychology*; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D008875:Middle Aged; D009362:Neoplasm Metastasis; D010358:Patient Participation; D059039:Standard of Care; D016019:Survival Analysis
keywords: Breast neoplasms; Clinical trial; Disease-free survival; Overall survival; Survival; Triple-negative breast cancer