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Evaluating treatment efficacy by multiple end points in phase II acute heart failure clinical trials: analyzing data using a global method.

Publication ,  Journal Article
Sun, H; Davison, BA; Cotter, G; Pencina, MJ; Koch, GG
Published in: Circ Heart Fail
November 2012

BACKGROUND: To assess concomitant simultaneous effects on multiple end points using global statistical methods in phase II acute heart failure studies. METHODS AND RESULTS: Using simulations we have assessed different statistical methods to evaluate concomitant effects of a new intervention on dyspnea relief (using 2 measures), length of hospital stay, worsening heart failure to 5 days, mortality, and heart failure readmission to 30 days. Treatment effect scenarios included large (20% to 28% relative improvements) and very large (30% to 43% relative improvements) effects among others. Placebo responses and correlations among end points typical in recent acute heart failure clinical trials were used. Powers for the average Z score exceeded 70% with ≥75 patients per group for 35% relative improvement across all 6 end points. Assessing dyspnea alone generally provides lower power than the average Z score approach, with power deducted ≈50% under most of scenarios. Other approaches generally provide lower power than the average Z score method. CONCLUSIONS: Assessing the effects of new therapies on multiple clinical end points using the average Z score enables detection of therapeutic efficacy using sample sizes of 100 to 150 patients per group, approximately double the power achievable assessing the effects on dyspnea alone.

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Published In

Circ Heart Fail

DOI

EISSN

1941-3297

Publication Date

November 2012

Volume

5

Issue

6

Start / End Page

742 / 749

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Statistics as Topic
  • Patient Readmission
  • Length of Stay
  • Humans
  • Hospital Mortality
  • Heart Failure
  • Endpoint Determination
  • Dyspnea
  • Disease Management
 

Citation

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Sun, H., Davison, B. A., Cotter, G., Pencina, M. J., & Koch, G. G. (2012). Evaluating treatment efficacy by multiple end points in phase II acute heart failure clinical trials: analyzing data using a global method. Circ Heart Fail, 5(6), 742–749. https://doi.org/10.1161/CIRCHEARTFAILURE.112.969154
Sun, Hengrui, Beth A. Davison, Gad Cotter, Michael J. Pencina, and Gary G. Koch. “Evaluating treatment efficacy by multiple end points in phase II acute heart failure clinical trials: analyzing data using a global method.Circ Heart Fail 5, no. 6 (November 2012): 742–49. https://doi.org/10.1161/CIRCHEARTFAILURE.112.969154.
Sun, Hengrui, et al. “Evaluating treatment efficacy by multiple end points in phase II acute heart failure clinical trials: analyzing data using a global method.Circ Heart Fail, vol. 5, no. 6, Nov. 2012, pp. 742–49. Pubmed, doi:10.1161/CIRCHEARTFAILURE.112.969154.

Published In

Circ Heart Fail

DOI

EISSN

1941-3297

Publication Date

November 2012

Volume

5

Issue

6

Start / End Page

742 / 749

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Statistics as Topic
  • Patient Readmission
  • Length of Stay
  • Humans
  • Hospital Mortality
  • Heart Failure
  • Endpoint Determination
  • Dyspnea
  • Disease Management