Low-dose mifepristone for the management of refractory heavy menstrual bleeding in women with bleeding disorders: a retrospective study.

backgroundHeavy menstrual bleeding (HMB) is a common and debilitating condition in women with bleeding disorders (BD). The underlying hemostatic abnormalities in these patients often make standard treatments ineffective. Clinical management is complicated in women with moderate-to-severe thrombocytopenia or those receiving antithrombotic therapy. To date, research addressing these two patient groups remains limited, with few safe and effective treatment options available due to their heightened bleeding risk. Therefore, the study aimed to evaluate low-dose mifepristone as a bridge therapy for refractory HMB in women with BD.

methodsA retrospective cohort study was conducted among women aged 10-55 years with BD and refractory HMB, treated with mifepristone at the Department of Gynecology, Peking University People's Hospital, from 2010 to 2024. For some patients with acute severe bleeding, intrauterine balloon tamponade were initially used, followed by mifepristone therapy maintained for at least two months. In patients with chronic bleeding, mifepristone was initiated at any point during the menstrual cycle. The primary endpoint was change in pictorial blood assessment chart (PBAC) score. Secondary endpoints included hemoglobin levels, amenorrhea rate, Menorrhagia Multi-Attribute Scale (MMAS) scores, and safety parameters. Continuous paired comparisons were analyzed using the Wilcoxon signed-rank test, and repeated measurements using the Friedman test with Bonferroni correction.

resultsA total of 63 patients met the inclusion criteria, with the study cohort primarily comprising 48 cases of thrombocytopenia of diverse etiologies and 13 patients receiving antithrombotic therapy. Following one month of treatment, the median PBAC score decreased from 385 to 0, while median hemoglobin levels increased from 51 g/L to 84 g/L (Bonferroni-adjusted p<0.01). Amenorrhea was achieved in 92.1% of patients by one month, and in all evaluable patients by three months. MMAS scores improved. No thromboembolic events or clinically relevant coagulation abnormalities were observed. Liver function remained stable, with only transient enzyme elevations, which were attributed to chemotherapy.

conclusionsIn this retrospective cohort, low-dose mifepristone was associated with improved bleeding control and acceptable tolerability. It may serve as a potential bridge therapy for women with BD and refractory HMB. Prospective controlled studies are warranted.