Methods for a multicenter randomized trial for mixed urinary incontinence: rationale and patient-centeredness of the ESTEEM trial.
OBJECTIVE
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.
The Division of Urogynecology and Reconstructive Pelvic Surgery, Department of Obstetrics and Gynecology, Alpert Medical School of Brown University, Providence, RI, USA. vsung@wihri.org.|Women and Infants Hospital of Rhode Island Division of Urogynecology and Reconstructive Pelvic Surgery, Warren Alpert Medical School of Brown University, 101 Plain Street 5th floor, Providence, RI, 02903, USA. vsung@wihri.org.
Vivian W
VW
Sung
Department of Physical Therapy, Rangos School of Health Sciences, Duquesne University, Pittsburgh, PA, USA.
Diane
D
Borello-France
The Division of Urogynecology, Department of Obstetrics and Gynecology, University of New Mexico, Albuquerque, NM, USA.
Gena
G
Dunivan
Social, Statistical, & Environmental Sciences, RTI International, Research Triangle Park, NC, USA.
Marie
M
Gantz
The Division of Female Pelvic Medicine & Reconstructive Surgery, Department of Reproductive Medicine, UC San Diego Health System, San Diego, CA, USA.
Emily S
ES
Lukacz
Women's Center for Bladder and Pelvic Health, Department of Obstetrics, Gynecology and Reproductive Sciences, Division of Urogynecology and Reconstructive Pelvic Surgery, University of Pittsburgh Medical Center, Pittsburgh, PA, USA.
Pamela
P
Moalli
The Division of Urology, Department of Surgery, University of Pennsylvania Health System, Philadelphia, PA, USA.
Diane K
DK
Newman
Division of Urogynecology and Pelvic Reconstructive Surgery, Department of Obstetrics and Gynecology, University of Alabama at Birmingham, Birmingham, Alabama, USA.
Holly E
HE
Richter
Center for Urogynecology and Reconstructive Pelvic Surgery, Obstetrics, Gynecology and Women's Health Institute, Cleveland Clinic, Cleveland, OH, USA.
Beri
B
Ridgeway
The Division of Urology, Department of Surgery, University of Pennsylvania Health System, Philadelphia, PA, USA.
Ariana L
AL
Smith
Division of Urogynecology, Department of Obstetrics and Gynecology, Duke University Medical Center, Durham, NC, USA.
Alison C
AC
Weidner
Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Susan
S
Meikle
D005260:Female; D006801:Humans; D018579:Patient Selection; D018802:Patient-Centered Care; D011788:Quality of Life; D012107:Research Design; D011795:Surveys and Questionnaires; D014549:Urinary Incontinence / Q000628:therapy*
Behavioral therapy; Clinical trials; Female; Mixed urinary incontinence; Sling