Efficacy and safety of transvaginal high-intensity focused ultrasound therapy in women with symptomatic uterine leiomyomas: A clinical trial

Objective: To evaluate the efficacy and safety of transvaginal high-intensity focused ultrasound (vHIFU) therapy in women with symptomatic uterine leiomyomas. Methods: This first-in-human, two-center, prospective, unblinded, single-arm trial was performed in the Republic of Korea from December 2017 to February 2019. Premenopausal women with symptomatic, contrast-enhanced uterine leiomyomas with a diameter ≤5 cm were eligible. Under sedation or monitored anesthesia, leiomyomas were ablated with vHIFU under ultrasound guidance. The primary endpoint was the non-perfused volume (NPV) ratio measured immediately after therapy. Secondary endpoints were changes in Uterine Fibroid Symptom-Quality of Life (UFS-QOL) scores, dysmenorrhea visual analog scale (VAS), uterine leiomyoma volume, rate of subsequent therapy, and treatment-emergent adverse events (TEAE). Results: Thirty-five women were screened; 13 women were enrolled and underwent vHIFU therapy for 33 uterine leiomyomas. NPV ratios were 0.76 ± 0.27 (mean ± SD); the lower limit of a one-sided 97.5 % confidence interval was 0.67, surpassing the non-inferiority cut-off of 0.50. UFS-QOL scores (symptom severity score, median, baseline: 66.60, 3-month follow-up: 32.85; p = 0.0010; health related quality of life score, median, baseline: 41.40, 3-month follow-up: 73.30; p = 0.0010) and dysmenorrhea VAS (mean, baseline: 50.92, 3-month follow-up: 20.67; p = 0.0019) improved significantly. Volume of uterine leiomyoma was reduced (median, baseline: 8.10 cm³, 3-month follow=-up: 5.30 cm³; p < 0.0001), and none received subsequent therapy. Twenty-six TEAEs from 8 participants were observed, and all TEAEs were resolved without sequelae. Conclusion: vHIFU therapy exhibited promising efficacy and safety and might be considered as a treatment option for women with symptomatic uterine leiomyomas. Registration: This trial was registered at: www.clinicaltrial.gov (NCT03328260)