Long-term treatment of uterine fibroids with ulipristal acetate

ObjectiveTo investigate the efficacy and safety of ulipristal acetate (UPA) for long-term treatment of symptomatic uterine fibroids.DesignRepeated intermittent open-label UPA courses, each followed by randomized double-blind norethisterone acetate (NETA) or placebo.SettingEuropean clinical gynecology centers.Patient(s)Two hundred and nine women with symptomatic fibroids including heavy menstrual bleeding.Intervention(s)Patients received up to four 3-month courses of UPA 10 mg daily, immediately followed by 10-day double-blind treatment with NETA (10 mg daily) or placebo.Main Outcome Measure(s)Amenorrhea, fibroid volume, endometrial histology.Result(s)After the first UPA course, amenorrhea occurred in 79% of women, with median onset (from treatment start) of 4 days (interquartile range, 2-6 days). Median fibroid volume change was −45% (interquartile range, −66%; −25%). Amenorrhea rates were 89%, 88%, and 90% for the 131, 119, and 107 women who received treatment courses 2, 3, and 4, respectively. Median times to amenorrhea were 2, 3, and 3 days for treatment courses 2, 3, and 4, respectively. Median fibroid volume changes from baseline were −63%, −67%, and −72% after treatment courses 2, 3, and 4, respectively. All endometrial biopsies showed benign histology without hyperplasia; NETA did not affect fibroid volume or endometrial histology.Conclusion(s)Repeated 3-month UPA courses effectively control bleeding and shrink fibroids in patients with symptomatic fibroids.Clinical trial registrationClinicalTrials.gov (www.clinicaltrials.gov) registration numbers NCT01156857 (PEARL III) and NCT01252069 (PEARL III extension).