Expression of Androgen Receptor Splice Variant 7 or 9 in Whole Blood Does Not Predict Response to Androgen-Axis-targeting Agents in Metastatic Castration-resistant Prostate Cancer

Eur Urol. 2018 Jun;73(6):818-821. doi: 10.1016/j.eururo.2018.01.007.

Abstract

In 2014, a landmark study was published demonstrating that the expression of androgen receptor splice variant (AR-V) 7 was a negative predictive biomarker for response to abiraterone acetate and enzalutamide in metastatic castration-resistant prostate cancer (mCRPC) patients. However, these results were not supported by the recently reported ARMOR3-SV phase III clinical trial, which employed an identical circulating tumour cell assay to assess AR-V7 expression. Therefore, the predictive utility of AR-V7 expression in mCRPC remains uncertain, as does any potential association between other AR-Vs and treatment response. To further investigate, we designed a highly sensitive and specific whole blood assay for detecting AR-V7 and AR-V9. We then examined for a correlation between baseline AR-V7/V9 status and treatment outcome in 37 mCRPC patients commencing abiraterone or enzalutamide. Of the patients, 24% (9/37) were AR-V-positive. Notably, prostate-specific antigen (PSA) response rates did not significantly differ between AR-V-positive (6/9) and AR-V-negative (18/28) patients (66% vs 64%, p=0.9). Likewise, median PSA progression-free survival was not significantly different between AR-V-positive and AR-V-negative patients (9.2 mo vs not reached; p=0.9). These data, which support the findings of the pivotal ARMOR3-SV clinical trial, suggest that baseline AR-V expression does not predict outcomes in mCRPC patients receiving abiraterone or enzalutamide.

Patient summary: Detection of androgen receptor splice variants (AR-Vs) in circulating tumour cells of advanced prostate cancer patients has been linked to resistance to abiraterone and enzalutamide. We designed a blood test to detect AR-Vs that can be performed more routinely than tests involving circulating tumour cells and found that patients with AR-Vs still benefit from these effective treatments.

Keywords: Abiraterone; Androgen receptor splice variant; Biomarker; Castration resistant; Enzalutamide; Prostate cancer.

Publication types

  • Letter
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Abiraterone Acetate / therapeutic use*
  • Antineoplastic Agents / therapeutic use*
  • Benzamides
  • Biomarkers, Tumor / blood*
  • Humans
  • Male
  • Neoplasm Metastasis
  • Nitriles
  • Phenylthiohydantoin / analogs & derivatives*
  • Phenylthiohydantoin / therapeutic use
  • Predictive Value of Tests
  • Progression-Free Survival
  • Prostate-Specific Antigen / blood
  • Prostatic Neoplasms, Castration-Resistant / blood*
  • Prostatic Neoplasms, Castration-Resistant / drug therapy*
  • Prostatic Neoplasms, Castration-Resistant / pathology
  • Protein Isoforms / blood
  • Receptors, Androgen / blood*

Substances

  • Antineoplastic Agents
  • Benzamides
  • Biomarkers, Tumor
  • Nitriles
  • Protein Isoforms
  • Receptors, Androgen
  • Phenylthiohydantoin
  • enzalutamide
  • Prostate-Specific Antigen
  • Abiraterone Acetate