Objective: To systematically review the effect of mifepristone on uterine leiomyoma size and symptoms and to summarize its adverse effects.
Data sources: A computerized search in MEDLINE, EMBASE, LILACS, and Cochrane databases from 1985 to 2002 and hand searches of conference proceedings from 1995 to 2002 were performed with the search terms "mifepristone" and "leiomyomata" and publication type "clinical trial."
Methods of study selection: Titles and abstracts were reviewed by 2 authors; there were no areas of disagreement. Inclusion criteria were clinical trials of daily mifepristone for uterine leiomyomata that measured uterine or leiomyoma volume before and after treatment.
Tabulation, integration, and results: Data from each article were abstracted by 2 reviewers. The search identified 6 before-and-after clinical trials involving a total of 166 women with symptomatic uterine leiomyomata. The subjects received 5 to 50 mg/d of mifepristone for 3 to 6 months. No study was placebo-controlled or blinded. Meta-analytic techniques were not performed due to variation in outcome and mifepristone dose. Daily treatment with all doses of mifepristone resulted in reductions in uterine and leiomyoma volumes ranging from 27% to 49% and 26% to 74%, respectively. Mifepristone treatment reduced the prevalence and severity of dysmenorrhea, menorrhagia, and pelvic pressure. Rates of amenorrhea ranged from 63% to 100%. Transient elevations in transaminases occurred in 4%. Endometrial hyperplasia was detected in 10 (28%) of 36 women screened by endometrial biopsy.
Conclusion: Published trials of mifepristone showed reduction in leiomyoma size and improvement in symptoms. A notable adverse effect of mifepristone was development of endometrial hyperplasia.