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Pelvic lymphangioleiomyomatosis treated successfully with everolimus: Two case reports with literature review

BACKGROUND: Lymphangioleiomyomatosis (LAM) is a rare disease affecting young women caused by abnormal proliferation of smooth muscle-like cells (LAM cells) in the lungs and extrapulmonary sites (extrapulmonary LAM). The objective of this case series is to demonstrate marked regression in 2 cases of...

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Detalles Bibliográficos
Autores principales: Wahid, Sharjil, Chiang, Ping Chia, Luo, Hao Lun, Huang, Shun-Chen, Tsai, Eing-Mei, Chiang, Po Hui
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Wolters Kluwer Health 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5348141/
https://www.ncbi.nlm.nih.gov/pubmed/28272193
http://dx.doi.org/10.1097/MD.0000000000004562
Descripción
Sumario:BACKGROUND: Lymphangioleiomyomatosis (LAM) is a rare disease affecting young women caused by abnormal proliferation of smooth muscle-like cells (LAM cells) in the lungs and extrapulmonary sites (extrapulmonary LAM). The objective of this case series is to demonstrate marked regression in 2 cases of retroperitoneal LAM after treatment with everolimus, an mTOR inhibitor. METHODS: We enrolled 2 cases with large volume, extrapulmonary pelvic LAM, and evaluated them with contrast-enhanced abdominal computed tomographic (CT) scans at presentation and serially during treatment with everolimus. Results were objectively quantified using the Response Evaluation Criteria in Solid Tumors, RECIST, Version 1.1. RESULTS: After 12 to 18 months of treatment with everolimus, both patients showed substantial reduction in the volume of their tumors. The first had about 50% regression of the pelvic LAM and renal angiomyolipoma (AML). The second patient had extensive abdomino-pelvic LAM which after treatment showed complete remission. Both patients have not demonstrated disease progression after nearly 4 and 2 years of follow-up, respectively. CONCLUSIONS: This case series demonstrates the enormous value of mTOR inhibitors (specifically everolimus) in the management of extrapulmonary pelvic LAM, of which there is no effective treatment currently available.