Abstract
OBJECTIVE: To evaluate novel hormonal therapies in patients with unresectable benign metastasizing leiomyoma (BML) disease. DESIGN: Case series. SETTING: National Institutes of Health (NIH). PATIENT(S): Five subjects with the diagnosis of BML based on imaging and/or histopathologic diagnosis. INTERVENTION(S): Four patients were treated with single or combination therapy of leuprolide acetate and/or an aromatase inhibitor. One patient was treated with an antiprogestin (CDB-2914). MAIN OUTCOME MEASURE(S): Response to therapy was measured by tumor burden on cross-sectional imaging employing RECIST (Response Evaluation Criteria in Solid Tumors) 1.1 guidelines. RESULT(S): Four patients treated with single or combination therapy of leuprolide acetate and/or an aromatase inhibitor demonstrated stable disease with reduction in tumor burden. The fifth patient treated with antiprogestin (CDB-2914) had degeneration of her tumor, progression of its size, and an improvement in symptoms. CONCLUSION(S): Hormone treatment with GnRH agonist and/or aromatase inhibition may be a therapeutic option to reduce tumor burden in unresectable BML disease or for those patients who wish to avoid surgical intervention. RECIST 1.1 guidelines, while traditionally used to evaluate tumor response to cancer therapeutics, may be useful in evaluating BML tumor burden response to hormone therapy.