BioMed Research International

Research Article

Selective Progesterone Receptor Modulators for the Medical Treatment of Uterine Fibroids with a Focus on Ulipristal Acetate

Table 1

Most important results from studies PEARL I–III [11, 23, 24].
 Study designStudy drug/- durationInclusion criteriaStudy populationPrimary endpoints and resultsSecondary endpoints and results
PEARL I [23]double-blind, randomized, 
placebo controlled, multicenter, parallel group		UPA 5 mg/d 
vs. 
UPA 10 mg/d 
vs.  
placebo 
Duration:  
12 Weeks 
All patients 
received iron 
supplementation		women with uterine fibroids,hypermenorrhea 
(PBAC> 100), anemia (Hb < 10,2 g/dl), 
eligible to undergo fibroid surgery after the end of the treatment period		UPA 5 mg:  
N = 96 
UPA 10 mg: 
N = 98 
Placebo: 
N = 48		Percendays of patients with control of uterine bleeding (PBAC 
< 75) at week 13: 
UPA 5 mg: 91  
UPA 10 mg: 92  
Placebo: 19  
P < 0,001 
Reduction of fibroid volume compared to baseline 
(median) at week 13: 
UPA 5 mg: -21,2  
UPA 10 mg: -12,3 ∗∗ 
Placebo: +3  
P = 0,002 
P = 0,006		Percendays of patients in amenorrhea at week 13: 
UPA 5 mg: 73 % 
UPA 10 mg: 82 % 
Placebo: 6 % 
P < 0,001 
Menstrual bleeding: 
PBAC at baseline (median) 
UPA 5 mg: 386 
UPA 10 mg: 330 
Placebo: 376 vs. at week 13: 
UPA 5 mg: -329 UPA 10 mg: -326 
Placebo: -59 
P < 0,001
PEARL II [11]double-blind, randomized 
parallel group, 
double-dummy, noninferiority trial		UPA 5 mg/d 
vs. 
UPA 10 mg/d 
vs. 
Leuprorelin 
Acetate 
(LA) 3,75 mg i.m. 
/1x Month 
Duration: 12 weeks		Women with uterine 
fibroids, 
Hypermenorrhea, 
all patients eligible for 
uterine fibroid surgery		UPA 5 mg: 
N = 93 
UPA 10 mg: 
N = 95 
LA: 
N = 93		Percendays of patients with 
controlled bleeding (PBAC < 75) 
at week 13: 
UPA 5 mg: 90  
UPA 10 mg: 98 ∗∗ 
LA: 89  
1,2 (95 CI: -9,3 to 11,8) 
8,8 (95 CI: 0,4 to 18,3)		Percendays of patients with 
amenorrhea at week 13: 
UPA 5 mg: 75 % 
UPA 10 mg: 89 % 
LA: 80 % 
Median change in fibroid 
volume of the 3 largest 
fibroids at baseline and at week 13: 
UPA 5 mg: -36 % 
UPA 10 mg: -42 % 
LA: -53 %
PEARL III [24]Long-term phase III 
UPA treatment 
Courses 
Open 
NETA treatment corses: 
double-blind, placebo controlled, randomized		Per treatment 
course: 
UPA 10 mg/d 
for 12 weeks 
followed by 
NETA 10 mg/d 
for 10 days or 
placebo 
for a total of 4 treatment courses		Women with 
symptomatic uterine 
fibroids, eligible for 
uterine fibroid surgery		UPA treatment 
Treatment course 1 
N = 132 
Treatment course 2 
N = 131 
Treatment course 3 
N = 119 
Treatment course 4 
N = 107		Patients in amenorrhea after each 
treatment course 
Treatment course 1: 79,5 % 
Treatment course 2: 88,5 % 
Treatment course 3: 88,2 % 
Treatment course 4: 89,7 %		Median change in fibroid 
volume of the 3 largest 
fibroids at baseline 
Treatment course 1: -45 % 
Treatment course 2: -63  
Treatment course 3: -67 % 
Treatment course 4: -72  
time to amenorrhea 
Treatment course 1: 4 Days 
Treatment course 2: 2 Days 
Treatment course 3: 3 Days 
Treatment course 4: 3 Days
PBAC: pictorial blood-loss assessment chart; UPA: ulipristal acetate; LA: leuprorelin acetate; NETA: norethisterone acetate.