Table III.
Effectiveness and safety outcomes of ulipristal, studied in registration trials.
| PRETREATMENT | ||
|---|---|---|
| Enhancing preoperative parameters (Lethaby et al., 2017) | ||
| Outcome | Studied?* | Effect/commentary |
| Amenorrhoea rates/preoperative bleeding | Yes (+) | Majority reached amenorrhoea within 7–10 days after start treatment (Donnez et al., 2012a) |
| Increases preoperative haemoglobin (Hb) levels | Yes (±) | Improvement, but could be related to additional daily iron supplementation only (Donnez et al., 2012a) |
| Reduces fibroid volume | Yes (±) | Significant effect compared to placebo (Donnez et al., 2012a), and similar effect compared to GnRHa (Donnez et al., 2012b) |
| Reduces uterine volume | Yes (±) | Significant effect compared to placebo (Donnez et al., 2012a), but inferior to GnRHa (Donnez et al., 2012b) |
| Quality of life (symptom reduction by validated questionnaires/scales) | Yes (+) | Less pain (Donnez et al., 2012a) and similar effect of pain and quality of life (Donnez et al., 2012b) |
|
| ||
|
Enhancing per- and postoperative parameters (Lethaby et al., 2017) | ||
|
No | Trials focused on preoperative treatment but were not designed to evaluate possible treatment-related differences in surgical outcomes (Donnez et al., 2012a,b) |
|
| ||
|
Safety† | ||
| Endometrial changes | Yes (±) | Higher incidence than with placebo/GnRHa (Donnez et al., 2012a,b) |
| Laboratory values (e.g. Hb, serum hormone levels, lipids, glucose) | Yes (+) | Laboratory parameters did not change significantly during repeated courses (Donnez et al., 2012a,b) |
| Adverse effects | Yes (+) | Less hot flushes than GnRHa (Donnez et al., 2012b) |
|
| ||
|
INTERMITTENT TREATMENT | ||
|
Sustained effect (also in therapy free interval)‡ | ||
| Amenorrhoea rates/controlled bleeding | Yes (±) | Sustained effect with repeated courses (Donnez et al., 2014, 2015, 2016) |
| Fibroid volume | Yes (±) | Sustained effect with repeated courses (Donnez et al., 2014, 2015, 2016) |
| Uterine volume | Yes (±) | Sustained effect with repeated courses (Donnez et al., 2014, 2015) |
| Quality of life (symptom reduction by validated questionnaires/scales) | Yes (±) | Sustained effect with repeated courses (Donnez et al., 2015, 2016) Not all fibroids symptoms were assessed, e.g. pressure symptoms, abdominal distension |
| Fibroid recurrence | Yes (±) | No regrowth recurrence at follow-up 3 months after cessation of therapy (Donnez et al., 2016) |
|
| ||
|
Safety† | ||
| Endometrial changes | Yes (+) | Changes apparent, but no concerns regarding endometrial histology (Donnez et al., 2016; Fauser et al., 2017) |
| Adverse effects | Yes (+) | No concerns regarding laboratory safety (such as Hb, liver enzymes) (Donnez et al., 2016; Fauser et al., 2017) |
*
Colour meanings; Green: studied in specific trials; Yellow: partly studied or studied in a non-representative patient population; Red: not studied in specific trials.
†
Safety outcomes discussed in Section D: Longer-term safety data collected to show long-term or rare side effects.
‡
As described in Section B: Intended patient population is studied: the study population involved relatively small fibroids and mild fibroid symptoms.