Ulipristal is a safe and effective option for women with uterine fibroids, according to two new studies published in the New England Journal of Medicine. In both studies, the oral selective progesterone(Drug information on progesterone) receptor modulator was well-tolerated, rapidly reduced excessive bleeding, and decreased the size of uterine fibroids. Dr. Jacques Donnez, head of the gynecology and andrology department at Brussels Saint-Luc University Hospital, Belgium, and president of the International Society for Preserving Fertility, was lead author for both studies.
In one study, Donnez and colleagues compared the efficacy and side effect profile of ulipristal acetate with those of leuprolide acetate for the treatment of symptomatic uterine fibroids before surgery in a double-blind noninferiority trial. The researchers randomly assigned women (N=307) who had symptomatic fibroids and excessive uterine bleeding to one of two treatment groups: one received 3 months of daily oral ulipristal acetate (at a dose of either 5 mg or 10 mg) and the other group received once-monthly intramuscular injections of leuprolide acetate (at a dose of 3.75 mg).
Compared with those patients who received leuprolide, women who received ulipristal were more likely to achieve controlled uterine bleeding. Specifically, uterine bleeding was controlled in 90% and 98% of women on ulipristal at 5 mg and 10 mg, respectively; uterine bleeding was controlled in just 89% of those who received leuprolide. Similarly, median time to amenorrhea was shorter among those women who receive ulipristal as compared leuprolide; median time was 7 days, 5 days, and 21 days for women receiving 5 mg ulipristal, 10 mg ulipristal, and leuprolide, respectively. Leuprolide was more often associated with hot flashes than ulipristal; while 40% of all patients on leuprolide reported moderate to severe hot flashes, only about 10% of women on either dose of ulipristal experienced hot flashes.
In a second study published in NEJM, Donnez and colleagues compared ulipristal with placebo by randomly assigning women with symptomatic fibroids, excessive uterine bleeding, and anemia to placebo (N=48) or ulipristal acetate (5 mg per day [N=96] or 10 mg per day [N=98]). After 13 weeks, bleeding was controlled in about 90% of patients receiving ulipristal at either dose but in only 19% of women on placebo. Median changes in fibroid volume decreased among women who received ulipristal (-21% and -12% for 5 mg and 10 mg, respectively) and increased (+3%) in the women in the placebo treatment group. Headache and breast tenderness were the most common adverse events reported among women in the ulipristal group; however, these events did not occur significantly more frequently than that in the placebo group.
While higher doses (30 mg) of ulipristal are used for emergency contraception (and known as ella), doses for fibroid treatment are significantly lower (ie, 5 mg and 10 mg).
Both studies concluded that ulipristal appears to be a safe and effective treatment option for women with fibroids and excessive bleeding and may prove to be an important tool in treating such women.