Pilot study of treatment of patients with deep infiltrative endometriosis with methotrexate carried in lipid nanoparticles

Objective Previously, lipid nanoparticles (LDE) injected in women with endometriosis were shown to concentrate in the lesions. Here, the safety and feasibility of LDE carrying methotrexate (MTX) to treat deep infiltrating endometriosis was tested. Design Prospective pilot study. Setting Perola Byington Hospital Reference for Women’s Health. Subjects Eleven volunteers (aged 30–47 years, BMI 26.15 ± 6.50 kg/m 2 ) with endometriosis with visual analog scale pelvic pain scores (VAS) > 7 and rectosigmoid lesions were enrolled in the study. Intervention Three patients were treated with LDE–MTX at single intravenous 25 mg/m 2 dose of MTX and eight patients with two 25 mg/m 2 doses with 1-week interval. Main outcome measures Clinical complaints, blood count, and biochemistry were analyzed before treatment and on days 90, 120, and 180 after LDE–MTX administration. Endometriotic lesions were evaluated by pelvic and transvaginal ultrasound (TVUS) before treatment and on days 30 and 180 after LDE–MTX administration. Results No clinical complaints related with LDE–MTX treatment were reported by the patients, and no hematologic, renal, or hepatic toxicities were observed in the laboratorial exams. FSH, LH, TSH, free T4, anti-Müllerian hormone, and prolactin levels were also within normal ranges during the observation period. Scores for deep dyspareunia ( p