Efficacy outcomes by baseline prostate-specific antigen quartile in the AFFIRM trial

Eur Urol. 2015 Feb;67(2):223-30. doi: 10.1016/j.eururo.2014.08.025. Epub 2014 Aug 27.

Abstract

Background: Enzalutamide significantly prolonged the survival of men with metastatic castration-resistant prostate cancer (PCa) after docetaxel in the randomised, phase 3, double-blind, placebo-controlled, multinational Patients with Progressive Castration-Resistant Prostate Cancer Previously Treated with Docetaxel-Based Chemotherapy (AFFIRM) trial (NCT00974311). Prostate-specific antigen (PSA) is commonly used as a marker of PCa disease burden, and the relationship of baseline PSA level to consequent treatment effect is of clinical interest.

Objective: Exploratory analysis to evaluate any differences in patient characteristics and efficacy outcomes by baseline PSA level in the AFFIRM trial.

Design, setting, and participants: Post hoc subanalysis of all randomised patients (n=1199) from the AFFIRM trial.

Intervention: Participants were randomly assigned in a two-to-one ratio to receive oral enzalutamide 160 mg/d or placebo.

Outcome measurements and statistical analysis: The major clinical efficacy end points were overall survival (OS), radiographic progression-free survival (rPFS), and time to PSA progression (TTPP) versus placebo; baseline characteristics, treatment duration, and subsequent antineoplastic therapy were compared by baseline PSA quartile.

Results and limitations: Baseline PSA quartiles corresponded to the following PSA groups: <40 ng/ml (n=299), 40 to <111 ng/ml (n=300), 111 to <406 ng/ml (n=300), and ≥406 ng/ml (n=300). Enzalutamide consistently improved OS, rPFS, and TTPP compared with placebo across all subgroups, regardless of baseline PSA level. Hazard ratios for improvements in OS were 0.55 (95% confidence interval [CI], 0.36-0.85), 0.69 (95% CI, 0.47-1.02), 0.73 (95% CI, 0.53-1.01), and 0.53 (95% CI, 0.39-0.73) for PSA groups 1-4, respectively. The post hoc design of this analysis was not statistically powered to assess the relationship between baseline PSA and clinical efficacy outcomes.

Conclusions: This post hoc analysis of the AFFIRM trial demonstrates consistent benefits in OS, rPFS, and TTPP with enzalutamide regardless of baseline disease severity, as assessed by PSA.

Patient summary: Exploratory post hoc analysis of the AFFIRM trial showed that enzalutamide improves overall survival, radiographic progression-free survival, and time to prostate-specific antigen progression compared with placebo regardless of baseline disease severity, as assessed by prostate-specific antigen.

Trial registration: ClinicalTrials.gov identifier NCT00974311.

Keywords: AFFIRM trial; Androgen receptor inhibitor; Enzalutamide; Metastatic castration-resistant prostate cancer; Prostate-specific antigen.

Publication types

  • Clinical Trial, Phase III
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Antineoplastic Agents / therapeutic use*
  • Australia
  • Benzamides
  • Disease Progression
  • Disease-Free Survival
  • Double-Blind Method
  • Europe
  • Humans
  • Kallikreins / blood*
  • Kaplan-Meier Estimate
  • Male
  • Middle Aged
  • Nitriles
  • North America
  • Phenylthiohydantoin / analogs & derivatives*
  • Phenylthiohydantoin / therapeutic use
  • Proportional Hazards Models
  • Prostate-Specific Antigen / blood*
  • Prostatic Neoplasms, Castration-Resistant / blood
  • Prostatic Neoplasms, Castration-Resistant / drug therapy*
  • Prostatic Neoplasms, Castration-Resistant / mortality
  • Prostatic Neoplasms, Castration-Resistant / pathology
  • Risk Factors
  • South Africa
  • South America
  • Time Factors
  • Treatment Outcome

Substances

  • Antineoplastic Agents
  • Benzamides
  • Nitriles
  • Phenylthiohydantoin
  • enzalutamide
  • KLK3 protein, human
  • Kallikreins
  • Prostate-Specific Antigen

Associated data

  • ClinicalTrials.gov/NCT00974311