title: Effect of Age on Clinical Outcomes in Phase 1 Trial Participants.
abstract: BACKGROUND
  Most persons with cancer living in the United States are older than 65 years of age; however, in general, elderly persons are under-represented in clinical trials and outcomes data are lacking.
  
  
  METHODS
  Outcomes data were analyzed of elderly participants (≥65 years of age) enrolled in phase 1 clinical trials and the results compared with those of younger patients. All consecutive, single-center, phase 1 oncology trials initiated and completed at the H. Lee Moffitt Cancer Center & Research Institute between 1997 and 2007 were included. Patient data (including survival, response, and toxicity rates) were extracted from a cancer registry database and electronic medical records at Moffitt Cancer Center.
  
  
  RESULTS
  After excluding multi-institution trials, we analyzed 39 trials for a total of 1,162 enrolled study participants, 32.7% of whom were elderly. Among patients who underwent transplantation, median survival rates were worse in those who were elderly compared with those who were younger (44.9 vs 32.9 months; P = .0037). However, in the no-transplantation setting, participants who were elderly had a median survival rate of 10.9 months (95% confidence interval [CI]: 8.9-13.1) compared with 8.8 months (95% CI: 7.9-10.3) in those who were younger (P = .15). Both groups had similar overall response rates (15.2% vs 13.1%) and similar treatment-related mortality rates (1% vs 0.9%, respectively). Adverse events occurring among the elderly and younger participants were not statistically significant.
  
  
  CONCLUSIONS
  Survival, response, toxicity, and treatment-related mortality rates were not significantly different between the elderly and younger phase 1 trial participants in the no-transplantation setting. Regardless of the complex pharmacological profiles and logistical issues involved in treating the elderly population, our data imply that elderly study participants do at least as well as their younger counterparts, contributing to the justification of increasing the phase 1 trial enrollment of elderly patients.
authors.affiliation: Clinical Research Unit, Moffitt Cancer Center, Tampa, FL 33612, USA. Amit.Mahipal@Moffitt.org.
authors.forename: Amit
authors.initials: A
authors.lastname: Mahipal
authors.forename: Aaron C
authors.initials: AC
authors.lastname: Denson
authors.forename: Benjamin
authors.initials: B
authors.lastname: Djulbegovic
authors.forename: Richard
authors.initials: R
authors.lastname: Lush
authors.forename: Ambuj
authors.initials: A
authors.lastname: Kumar
authors.forename: Tzu-Hua
authors.initials: TH
authors.lastname: Juan
authors.forename: Michael J
authors.initials: MJ
authors.lastname: Schell
authors.forename: Daniel M
authors.initials: DM
authors.lastname: Sullivan
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D017321:Clinical Trials, Phase I as Topic / Q000379:methods*; D006801:Humans; D008875:Middle Aged; D009369:Neoplasms / Q000188:drug therapy*; D017063:Outcome Assessment, Health Care; D018579:Patient Selection; D016019:Survival Analysis; D055815:Young Adult
chemical_list: D000970:Antineoplastic Agents