Sirolimus in the treatment of kaposiform lymphangiomatosis
Open Access
- 8 June 2021
- journal article
- research article
- Published by Springer Science and Business Media LLC in Orphanet Journal of Rare Diseases
- Vol. 16 (1), 1-9
- https://doi.org/10.1186/s13023-021-01893-3
Abstract
Background: Kaposiform lymphangiomatosis (KLA), which is a new subtype of generalized lymphatic anomaly, is a rare disease with a poor prognosis. Currently, there is no standard treatment due to the poor understanding of KLA. Sirolimus, which is an inhibitor of mammalian target of rapamycin, has been shown to have promising potential in the treatment of complicated vascular anomalies. The aim of this study was to introduce the use of sirolimus for the treatment of KLA and to highlight the challenges of managing this refractory disease. Results: We reported seven patients with KLA who received sirolimus therapy in our center. Combined with previously reported cases, 58.3% achieved a partial response, 25.0% had stable disease, and 16.7% experienced disease progression. No severe sirolimus-related adverse events occurred during treatment. Conclusions: This study suggests that sirolimus is currently an option for the treatment of KLA, and it is hoped that more specific therapies will be developed in the future. Rapid advances in basic science and clinical practice may facilitate the development of important new treatments for KLA.Keywords
Funding Information
- National Natural Science Foundation of China (81400862, 81401606)
- Key Project in the Science & Technology Program of Sichuan Province (2019YFS0322)
- Science Foundation for The Excellent Youth Scholars of Sichuan University (2015SCU04A15)
- 1·3·5 Project for Disciplines of Excellence-Clinical Research Incubation Project, West China Hospital of Sichuan University (2019HXFH056, 2020HXFH048)
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