骨盆淋巴管平滑肌瘤用癌伏妥治療成功：病例報告
吳宗憲、Sharjil Wahid、江博暉
高雄長庚醫院泌尿科
Pelvic Lymphangioleiomyomatosis treated successfully with everolimus: Case report
Tsung Hsien Wu, Sharjil Wahid, Po Hui Chiang
Purpose: 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 3 cases of retroperitoneal LAM after treatment with everolimus, an mTOR inhibitor.
Materials and Methods: We enrolled 3 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 6-18 months of treatment with everolimus, all 3 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.