Systemic therapy is effective in the management of leiomyomatosis

Objective: Leiomyomatosis is an intermediate neoplasm of uterine origin. There is limited understanding of its natural history and response to systemic therapy. The objective of this retrospective review is to describe the management and response to systemic therapy for leiomyomatosis. Methods: A retrospective chart review was performed from 1 January 2000 – 1 February 2021 for patients 18 years of age or older with leiomyomatosis treated at The Royal Marsden Hospital. Descriptive statistics were performed. Results: Fifteen female patients with a median age of 45 years (range 34–49) were identified. Patients were ethnically diverse (Black n = 4 [26%], Asian n = 1 [7%], Caucasian n = 6 [40%], other n = 3 [20%], Unknown n = 1 [7%]). Most patients presented with advanced disease (n = 12/15, 80%); common sites being abdomen (n = 9/12, 75%), abdominal veins (n = 6/12, 50%), including the IVC 4/12 (30%). At presentation, two patients (17%) had cardiac extension from the IVC. Eleven patients (73%) underwent surgery and 4 (27%)active surveillance as first treatment. All patients undergoing surgery had residual disease (n = 9/9 for those with available data). Ten patients received systemic therapy for advanced disease (median 3 treatments, range 1–5) and most patients (n = 9/10, 90%) were treated with endocrine therapy. Response rate (RR) and clinical benefit rate (CBR) was higher for GNRH agonist (RR 43%, CBR 86%) and GNRH agonist + aromatase inhibitor (AI) (RR/CBR 100%), than AI alone (RR 0%, CBR 66%). Patients were only treated with non-endocrine systemic therapy after failure of endocrine therapy, and no responses were seen. No patients had confirmed malignant transformation; two patients had clinical suspicion of transformation but no correlative features on repeat biopsy. Conclusions: Leiomyomatosis is a rare, but morbid condition. The mainstay of treatment for advanced disease is endocrine therapy, the choice of initial approach requires careful consideration within a multi-disciplinary team.

leiomyomatosis; intra-vascular leiomyomatosis; soft tissue neoplasm; systemic therapy; aromatase inhibitor

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