Ulipristal acetate for treatment of uterine leiomyomas: A randomized controlled trial

James H. Liu*, David Soper, Andrea Lukes, Phyllis Gee, Thomas Kimble, Robin Kroll, Madhuja Mallick, Anna Chan, Patrick Gillard, Amanda Harrington, Vilma Sniukiene, Lee P. Shulman

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

32 Scopus citations


OBJECTIVE: To assess the efficacy and tolerability of ulipristal acetate, a selective progesterone receptor modulator, for treatment of symptomatic uterine leiomyomas. METHODS: This phase 3, double-blind, double-dummy, placebo-controlled trial randomized premenopausal women (18–50 years) with uterine leiomyomas and abnormal uterine bleeding to once-daily 5 mg ulipristal, 10 mg ulipristal, or placebo in two 12-week treatment courses separated by a drug-free interval of two menses. Coprimary end points were rates of and time to amenorrhea during course 1. Change from baseline to end of course 1 in the Revised Activities subscale of the Uterine Fibroid Symptom and Health-Related Quality of Life questionnaire was a secondary end point. A sample size of 400 was planned to compare separately each ulipristal dose with placebo. RESULTS: From January 2014 through November 2016, 432 women were randomized. Demographic characteristics were similar across treatment groups. In course 1, 68 of 162 (42.0% [97.5% CI 33.3–51.1]) and 86 of 157 (54.8% [97.5% CI 45.5–63.8]) patients treated with 5 mg and 10 mg ulipristal, respectively, compared with 0 of 113 (0.0% [97.5% CI 0.0–3.8]) patients treated with placebo achieved amenorrhea (P,.001 for each dose); most women who achieved amenorrhea did so within 10 days (time to amenorrhea, P,.001 for each dose). Significantly greater improvements in Uterine Fibroid Symptom and Health-Related Quality of Life Revised Activities subscale scores were reported with 5 mg and 10 mg ulipristal compared with placebo (least squares mean change from baseline: 48.3, 56.7, and 13.0, respectively; P,.001 for each dose). Both ulipristal doses were well tolerated; in course 1, hot flush occurred in 7.5%, 11.6%, and 1.7%

Original languageEnglish (US)
Pages (from-to)1241-1251
Number of pages11
JournalObstetrics and gynecology
Issue number5
StatePublished - 2018
Externally publishedYes

ASJC Scopus subject areas

  • Obstetrics and Gynecology


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