Pelvic lymphangioleiomyomatosis treated successfully with everolimus
| Autor: | Ping Chia Chiang, Eing-Mei Tsai, Po Hui Chiang, Hao Lun Luo, Shun-Chen Huang, Sharjil Wahid |
|---|---|
| Rok vydání: | 2017 |
| Předmět: |
medicine.medical_specialty
Everolimus business.industry Disease progression Complete remission General Medicine bacterial infections and mycoses medicine.disease 03 medical and health sciences 0302 clinical medicine immune system diseases Response Evaluation Criteria in Solid Tumors hemic and lymphatic diseases 030220 oncology & carcinogenesis Lymphangioleiomyomatosis medicine Effective treatment lipids (amino acids peptides and proteins) 030212 general & internal medicine Radiology business Renal angiomyolipoma Rare disease medicine.drug |
| Zdroj: | Medicine. 96:e4562 |
| ISSN: | 0025-7974 |
| DOI: | 10.1097/md.0000000000004562 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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