title: The Complementary Nature of Patient-Reported Outcomes and Adverse Event Reporting in Cooperative Group Oncology Clinical Trials: A Pooled Analysis (NCCTG N0591).
abstract: BACKGROUND
  Clinical trials use clinician-graded adverse events (AEs) and patient-reported outcomes (PROs) to describe symptoms.
  
  
  OBJECTIVE
  The aim of the study was to examine the agreement between PROs and AEs in the clinical trial setting.
  
  
  METHODS
  Patient-level data were pooled from seven North Central Cancer Treatment Group, two Southwest Oncology Group, and three Radiation Therapy Oncology Group lung studies that included both PROs and AE data. Ten-point changes (on a 0-100 scale) in PRO scores were considered clinically significant differences (CSDs). PRO score changes were compared to AE grade (Gr) categories (2+ yes vs. no and 3+ yes vs. no) using Wilcoxon rank-sum or two-sample t-tests between Gr categories. Incidence rates and concordance of CSD in PRO scores and AE Gr categories were compiled. Spearman correlations were computed between PRO scores and AE severity.
  
  
  RESULTS
  PROs completed by patients (n = 1013) were the Uniscale, Lung Cancer Symptom Scale (LCSS), Functional Assessment of Cancer Therapy-Lung (FACT-L), Symptom Distress Scale, and/or Functional Living Index-Cancer. Significantly worse PRO score changes were found for the FACT-L in patients with Gr 2+ AEs. Worse scores were seen for the Uniscale for patients with Gr 2+ AEs (P = 0.07) and LCSS for patients with Gr 3+ AEs (P = 0.09). Agreement between incidence of any Gr 2+ (Gr 3+) AE and a CSD in PROs ranged from 27% to 67% (36%-61%). Correlations between PRO scores and AE severity were low: -0.06 Uniscale, -0.03 LCSS, 0.10 FACT-L, -0.11 Symptom Distress Scale, and -0.51 Functional Living Index-Cancer.
  
  
  CONCLUSIONS
  These results support previous work and an a priori hypothesis that AEs and PROs measure differing aspects of the disease experience and are complementary.
authors.affiliation: Alliance Statistics and Data Center, Mayo Clinic, Rochester, Minnesota, USA. Electronic address: atherton@mayo.edu.
authors.forename: Pamela J
authors.initials: PJ
authors.lastname: Atherton
authors.affiliation: Nell Hodgson Woodruff School of Nursing, Emory University, Atlanta, Georgia, USA.
authors.forename: Deborah W
authors.initials: DW
authors.lastname: Watkins-Bruner
authors.affiliation: School of Population and Public Health, University of British Columbia, Vancouver, British Columbia, Canada.
authors.forename: Carolyn
authors.initials: C
authors.lastname: Gotay
authors.affiliation: Public Health Sciences Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.
authors.forename: Carol M
authors.initials: CM
authors.lastname: Moinpour
authors.affiliation: Alliance Statistics and Data Center, Mayo Clinic, Rochester, Minnesota, USA.
authors.forename: Daniel V
authors.initials: DV
authors.lastname: Satele
authors.affiliation: Statistical Department, Radiation Therapy Oncology Group, Philadelphia, Pennsylvania, USA.
authors.forename: Kathryn A
authors.initials: KA
authors.lastname: Winter
authors.affiliation: Toledo Community Hospital Oncology Program, Toledo, Ohio, USA.
authors.forename: Paul L
authors.initials: PL
authors.lastname: Schaefer
authors.affiliation: Department of Radiation Oncology, Henry Ford Medical Center, Detroit, Michigan, USA.
authors.forename: Benjamin
authors.initials: B
authors.lastname: Movsas
authors.affiliation: Alliance Statistics and Data Center, Mayo Clinic, Rochester, Minnesota, USA.
authors.forename: Jeff A
authors.initials: JA
authors.lastname: Sloan
mesh_terms: D000368:Aged; D002986:Clinical Trials as Topic / Q000379:methods*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D015994:Incidence; D008297:Male; D008875:Middle Aged; D009369:Neoplasms / Q000453:epidemiology / Q000628:therapy*; D063868:Patient Outcome Assessment; D010820:Physicians; D057566:Self Report; D012720:Severity of Illness Index; D016896:Treatment Outcome
keywords: Patient-reported outcomes; adverse events; clinical trials