Developing an objective marker to optimize patient selection and predict survival benefit in early-phase cancer trials

Cancer. 2014 Jan 15;120(2):262-70. doi: 10.1002/cncr.28381. Epub 2013 Oct 8.

Abstract

Background: Several prognostic indices have been devised to optimize patient selection for phase 1 oncology trials with no consensus as to the optimal score and none qualifying as a marker of treatment response.

Methods: Multivariate predictors of overall survival (OS) were tested on 118 referred patients to develop the Hammersmith Score (HS). The score's ability to predict OS, progression-free survival (PFS), and 90-day mortality (90DM) was compared with other prognostic indices. Changes in HS were recalculated during treatment.

Results: Albumin<35 g/L, lactate dehydrogenase>450 U/L, and sodium<135 mmol/L emerged as independent prognostic factors. These were used with equal weighting to devise the HS, a compound prognostic index ranging from 0 to 3. High (HS=2-3) score predicted worse OS (hazard ratio [HR]=6.5, P<.001), PFS (HR=2.8, P=.01), and 90DM (OR=9.0, P<.001). HS was a more accurate multivariate predictor of OS (HR=6.4, P<.001, C-index=0.72), PFS (HR=2.7, P=.03), and 90DM (area under the ROC curve 0.703) compared with other scores. Worsening of the HS during treatment predicted for shorter OS (P<.001). HS retained prognostic and predictive ability following external validation.

Conclusions: HS is a simple, validated index to optimize patient selection and predict survival benefit from phase 1 oncology treatments. Prospective validation is ongoing.

Keywords: albumin; outcome; phase 1 trial; prognostic score; survival.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Albumins / analysis
  • Clinical Trials, Phase I as Topic*
  • Disease-Free Survival
  • Female
  • Humans
  • L-Lactate Dehydrogenase / blood
  • Male
  • Middle Aged
  • Multivariate Analysis
  • Neoplasms / mortality*
  • Neoplasms / therapy
  • Patient Selection*
  • Predictive Value of Tests*
  • Prognosis
  • Reproducibility of Results
  • Sodium / blood

Substances

  • Albumins
  • Sodium
  • L-Lactate Dehydrogenase