Table 2.
Efficacy Endpoint Results (Intent-to-Treat Population)
| Placebo (n=56) | 5 mg Ulipristal (n=53) | 10 mg Ulipristal (n=48) | |
|---|---|---|---|
| Coprimary endpoint | |||
| Absence of bleeding responder on treatment* | |||
| Responder rate | 1 (1.8) | 25 (47.2) | 28 (58.3) |
| Responder rate difference | 45.4 (29.5–61.3) | 56.5 (40.1–73.0) | |
| P | <.001 | <.001 | |
| Secondary endpoints | |||
| Absence of bleeding responder by day 11† | |||
| Responder rate | 0 (0.0) | 23 (43.4) | 28 (58.3) |
| Responder rate difference | 43.4 (28.1–58.7) | 58.3 (42.4–74.3) | |
| P | <.001 | <.001 | |
| UFS-QOL Revised Activities subscale score‡ | |||
| LS change from baseline | 21.2±63.8 | 52.163.7 | 59.064.0 |
| LS mean difference | 30.9 (19.2–42.6) | 37.7 (25.5–49.9) | |
| P | <.001 | <.001 | |
| Additional endpoints | |||
| Volume of up to 3 largest uterine leiomyomas | |||
| LS mean % change from baseline to end of treatment§ | 7.2 | −9.6 | −16.3 |
| LS mean difference | −16.8 (−48.1 to 14.0) | −23.5 (−54.9 to 7.3) | |
| P | .218 | .086 | |
| LS mean % change from baseline to end of follow-up§ | 5.7 | −2.3 | −17.4 |
| LS mean difference | −8.0 (−43.7 to 27.3) | −23.1 (−57.8 to 10.8) | |
| P | .605 | .124 | |
| Control of bleeding∥ | |||
| n1 | 53 | 46 | 38 |
| Responder rate | 1 (1.9) | 27 (58.7) | 31 (81.6) |
| Responder rate difference | 56.8 (40.0–73.6) | 79.7 (65.0–94.4) | |
| P | <.001 | <.001 | |
UFS-QOL, Uterine Fibroid Symptom and Health-Related Quality of Life questionnaire; LS, least squares; n1, patients with 66 days or greater of treatment exposure.
Data are n (%), % (97.5% CI), or mean±standard error unless otherwise specified.
Cochran-Mantel-Haenszel tests controlling for study site were used for dichotomous endpoints; log-rank tests were used for time to amenorrhea; analysis of covariance controlling for baseline score and pooled study site was used for change from baseline in UFS-QOL Revised Activity subscale score.
Proportion of patients achieving amenorrhea for the last 35 consecutive days of treatment; results for coprimary endpoint of time to amenorrhea are shown in Figure 3.
Proportion of patients achieving amenorrhea by day 11 of treatment and did not report bleeding for the duration of treatment.
HRQOL Revised Activities subscale scores range from 0 to 100; higher scores indicate better health-related quality of life.
Represents log-transformed analyses of percent reduction in leiomyoma volume; positive values represent increase in volume and negative values represent reduction in volume.
Proportion of patients with 66 days of treatment or greater exposure achieving 0 days of heavy bleeding and 8 days of bleeding or less within the last 56 days of treatment.