Methods for a multicenter randomized trial for mixed urinary incontinence: rationale and patient-centeredness of the ESTEEM trial.

Academic Article

Abstract

  • INTRODUCTION AND HYPOTHESIS: Mixed urinary incontinence (MUI) can be a challenging condition to manage. We describe the protocol design and rationale for the Effects of Surgical Treatment Enhanced with Exercise for Mixed Urinary Incontinence (ESTEEM) trial, designed to compare a combined conservative and surgical treatment approach versus surgery alone for improving patient-centered MUI outcomes at 12¬†months. METHODS: ESTEEM is a multisite, prospective, randomized trial of female participants with MUI randomized to a standardized perioperative behavioral/pelvic floor exercise intervention plus midurethral sling versus midurethral sling alone. We describe our methods and four challenges encountered during the design phase: defining the study population, selecting relevant patient-centered outcomes, determining sample size estimates using a patient-reported outcome measure, and designing an analysis plan that accommodates MUI failure rates. A central theme in the design was patient centeredness, which guided many key decisions. Our primary outcome is patient-reported MUI symptoms measured using the Urogenital Distress Inventory (UDI) score at 12¬†months. Secondary outcomes include quality of life, sexual function, cost-effectiveness, time to failure, and need for additional treatment. RESULTS: The final study design was implemented in November 2013 across eight clinical sites in the Pelvic Floor Disorders Network. As of 27 February 2016, 433 total/472 targeted participants had been randomized. CONCLUSIONS: We describe the ESTEEM protocol and our methods for reaching consensus for methodological challenges in designing a trial for MUI by maintaining the patient perspective at the core of key decisions. This trial will provide information that can directly impact patient care and clinical decision making.
  • Published In

    Keywords

  • Behavioral therapy, Clinical trials, Female, Mixed urinary incontinence, Sling, Female, Humans, Patient Selection, Patient-Centered Care, Quality of Life, Research Design, Surveys and Questionnaires, Urinary Incontinence
  • Digital Object Identifier (doi)

    Author List

  • Sung VW; Borello-France D; Dunivan G; Gantz M; Lukacz ES; Moalli P; Newman DK; Richter HE; Ridgeway B; Smith AL
  • Start Page

  • 1479
  • End Page

  • 1490
  • Volume

  • 27
  • Issue

  • 10