Addressing heterogenous outcomes in uterine fibroid research: a call to action

Abstract

Uterine fibroid tumors are the most common benign pelvic tumors in women, with complications that include heavy menstrual bleeding, pelvic pain, reproductive complications, and bulk-related symptoms. Although the majority of uterine fibroid tumors are asymptomatic, those women who experience symptoms can experience substantial burdens on quality of life and daily functioning. Comparative effectiveness reviews of available medical, surgical, and radiologic treatments have found that a lack of high-quality data to inform treatment decisions is, in part, due to the use of heterogeneous outcomes and instruments in clinical studies. With multiple new interventions emerging, this call-to-action encourages the development and use of a core outcome set that will capture the most relevant, patient-important outcomes in late-phase and after-marketing therapeutic trials for uterine fibroid tumors. The core outcome set should be developed by a diverse, multistakeholder group comprised of key healthcare decision-makers. Development and uptake of a core outcome set ensures that a consistent, collaboratively vetted set of outcomes will be accessible across different studies and promotes transparency for innovators who seek to anticipate the evidence needs of patients, providers, payers, regulators, and other stakeholders.

Keywords: core outcome set; outcome measures; outcomes; uterine fibroids; uterine leiomyoma.