Back to Search
Start Over
Sirolimus in the treatment of kaposiform lymphangiomatosis.
- Source :
-
Orphanet Journal of Rare Diseases . 6/8/2021, Vol. 16 Issue 1, p1-9. 9p. - Publication Year :
- 2021
-
Abstract
- <bold>Background: </bold>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.<bold>Results: </bold>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.<bold>Conclusions: </bold>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. [ABSTRACT FROM AUTHOR]
- Subjects :
- *RAPAMYCIN
*PROGNOSIS
*DISEASE progression
*RARE diseases
*MTOR protein
Subjects
Details
- Language :
- English
- ISSN :
- 17501172
- Volume :
- 16
- Issue :
- 1
- Database :
- Academic Search Index
- Journal :
- Orphanet Journal of Rare Diseases
- Publication Type :
- Academic Journal
- Accession number :
- 150767086
- Full Text :
- https://doi.org/10.1186/s13023-021-01893-3