OBJECTIVE: To investigate the efficacy and safety of ulipristal acetate (UPA) for long-term treatment of symptomatic uterine fibroids.
DESIGN: Repeated intermittent open-label UPA courses, each followed by randomized double-blind norethisterone acetate (NETA) or placebo.
SETTING: European 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 REGISTRATION: ClinicalTrials.gov (www.clinicaltrials.gov) registration numbers NCT01156857 (PEARL III) and NCT01252069 (PEARL III extension).
- Administration, Oral
- Antineoplastic Agents, Hormonal
- Antineoplastic Combined Chemotherapy Protocols
- Double-Blind Method
- Drug Administration Schedule
- Middle Aged
- Pelvic Pain
- Quality of Life
- Time Factors
- Treatment Outcome
- Tumor Burden
- Uterine Neoplasms