title: Pelvic lymphangioleiomyomatosis treated successfully with everolimus: Two case reports with literature review.
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.
authors.affiliation: Department of Urology, Kaohsiung Chang Gung Memorial Hospital and Chang Gung University College of Medicine Graduate Institute of Medicine, College of Medicine, Kaohsiung Medical University Department of Pathology, Kaohsiung Chang Gung Memorial Hospital and Chang Gung University College of Medicine Department of Obstetrics and Gynecology, Kaohsiung Medical University Hospital, Kaohsiung Medical University Graduate Institute of Medicine, Center of Excellence for Environmental Medicine, Kaohsiung Medical University, Kaohsiung, Taiwan College of Physicians and Surgeons, Pakistan School of Medicine, Kaohsiung Medical University, Kaohsiung, Taiwan.
authors.forename: Sharjil
authors.initials: S
authors.lastname: Wahid
authors.forename: Ping Chia
authors.initials: PC
authors.lastname: Chiang
authors.forename: Hao Lun
authors.initials: HL
authors.lastname: Luo
authors.forename: Shun-Chen
authors.initials: SC
authors.lastname: Huang
authors.forename: Eing-Mei
authors.initials: EM
authors.lastname: Tsai
authors.forename: Po Hui
authors.initials: PH
authors.lastname: Chiang
mesh_terms: D000328:Adult; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D000068338:Everolimus / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D018192:Lymphangioleiomyomatosis / Q000188:drug therapy* / Q000473:pathology; D008875:Middle Aged; D010386:Pelvic Neoplasms / Q000188:drug therapy* / Q000473:pathology; D010388:Pelvis / Q000473:pathology
chemical_list: D000970:Antineoplastic Agents; D000068338:Everolimus