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

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

Medicine (Baltimore). 2017 Mar;96(10):e4562

Authors: Wahid S, Chiang PC, Luo HL, Huang SC, Tsai EM, Chiang PH

Abstract
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.

PMID: 28272193 [PubMed - indexed for MEDLINE]

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