A 42-year-old woman presents with heavy menstrual bleeding (HMB) and a 14-week-sized uterus with multiple intramural fibroids, largest 6 cm. She declines surgery. She is treated with ulipristal acetate (UPA). The PEARL III trial showed UPA is effective in controlling HMB; however, which key safety concern led to CHMP (European Medicines Agency) restricting its use?

• A Increased risk of endometrial carcinoma with long-term use
• B Cases of serious liver injury (hepatotoxicity) requiring liver transplantation ✓
• C Teratogenicity — UPA causes fetal anomalies if pregnancy occurs during treatment
• D Adrenal insufficiency due to cross-reactivity with glucocorticoid receptors

Explanation

Post-marketing pharmacovigilance following the PEARL trials identified rare but serious cases of hepatic injury (acute liver failure requiring transplantation) in women using ulipristal acetate (Esmya). The European Medicines Agency (EMA/CHMP) in 2020 suspended marketing authorisation for UPA for uterine fibroids pending safety review. Liver function monitoring was mandated. This remains the major safety signal that led to withdrawal from markets; endometrial changes (PAEC — progesterone receptor modulator-associated endometrial changes) with UPA are benign and reversible.

Reference: Shaw's Textbook of Gynaecology, 17th ed.

High-yield for: NEET PGINI-CETNExTFMGEUSMLEPLABMRCP

Written and medically reviewed by the StethoPrep medical team.