Oral medication treats uterine fibroids
Uterine fibroids are common benign tumors that cause bleeding problems, pain, and infertility. The only 100% cure is a hysterectomy: a procedure that is devastating to a women who desires to preserve her fertility.
Two new studies were published online on February 2 in The New England Journal of Medicine, which reported that ulipristal was highly effective for the treatment of these tumors. Both studies reported that the medication, which is classified as a progesterone receptor modulator, rapidly reduced excessive bleeding, reduced the size of the tumors and was well tolerated. (Progesterone receptor modulators negate the effect of the female hormone progesterone.)
In 1910, ulipristal was approved for emergency contraception in 2010. The medication works by interrupting ovulation; it is known as ella (HRA Pharma) in the U.S. and ellaOne in Europe. A 30 mg dose is administered for emergency contraception; however, the new studies evaluated a 5 mg and 10 mg daily dose for fibroid treatment. These studies were designed to evaluate the effectiveness of the medication on controlling bleeding and shrinking the tumor size so that the women could more safely undergo a myomectomy (less blood loss and complications). A myomectomy is the surgical removal of a fibroid tumor with preservation of the uterus.
In the first of the two studies, women with symptomatic fibroids and excessive uterine bleeding from fibroids were treated for up to 13 weeks with ulipristal at a dose of 5 mg per day (96 women) or 10 mg per day (98 women) or to receive placebo (48 women). All patients received an iron supplement. The outcome measures were the control of uterine bleeding and reduction of fibroid volume at week 13, after which patients could undergo a myomectomy.
At 13 weeks, uterine bleeding was controlled in 91% of the women receiving 5 mg of ulipristal acetate, 92% of those receiving 10 mg of ulipristal acetate, and 19% of those receiving a placebo. The rates of amenorrhea (no menses were 73% (5 mg), 82% (10 mg), and 6% (placebo); amenorrhea occurred within 10 days in the majority of patients receiving ulipristal acetate. The median changes in total fibroid volume were −21% (5 mg), −12% (10 mg), and +3% (placebo). One patient receiving 10 mg ulipristal experienced a uterine hemorrhage and one woman receiving the placebo suffered from a fibroid that protruded through her cervix. Headache and breast tenderness were the most common adverse events experienced by those that received the active medication; however, the difference between those groups and the placebo group was not statistically significant.
The authors concluded that treatment with ulipristal acetate for 13 weeks effectively controlled excessive bleeding due to uterine fibroids and reduced the size of the fibroids.
In the second study, 307 women with symptomatic fibroids and excessive uterine bleeding were randomly assigned to receive three months of daily therapy with ulipristal (either 5 mg or 10 mg) or once-monthly intramuscular injections of leuprolide acetate (Lupron) 3.75 mg. The study was termed a noninferiority trial, meaning that the authors attempted to determine whether ulipristal was noninferior to leuperolide.
The authors reported that uterine bleeding was controlled in 90% of women who received 5 mg of ulipristal, in 98% of those who received 10 mg of ulipristal, and in 89% of women who received leuprolide. The median times to amenorrhea were seven days for the women who received five mg of ulipristal acetate, five days for those who received 10 mg of ulipristal, and 21 days for those receiving leuprolide acetate. The most common side-effect was hot flashes, which were reported for 11% of women who received 5 mg of ulipristal, for 10% of those who received 10 mg of ulipristal, and for 40% of women who received leuprolide.
The authors concluded that both the 5 mg and 10 mg daily doses of ulipristal were noninferior to once-monthly leuprolide acetate in controlling uterine bleeding and were significantly less likely to cause hot flashes.
Of note, the primary author of both studies was Jacques Donnez, M.D., Ph.D. The studies were supported by ulipristal’s manufacturer, PregLem; PregLem is a Swiss-based specialty biopharmaceutical company focused on women's reproductive medicine. Dr. Donnez disclosed participation in PregLem’s scientific advisory board and payment from stocks sold in October 2010 from the acquisition by Gedeon.