Joyce Teng, Jeff Martini, Michael Kelly, Megha Tollefson, Alexander Greer
{"title":"Sirolimus for Venous Malformations: A Systematic Review of Efficacy and Safety.","authors":"Joyce Teng, Jeff Martini, Michael Kelly, Megha Tollefson, Alexander Greer","doi":"10.1177/15578585251377562","DOIUrl":null,"url":null,"abstract":"<p><p><b><i>Background:</i></b> Venous malformations can cause substantial morbidity and long-term complications. There are no Food and Drug Administration (FDA)-approved therapies for the treatment of venous malformations. However, off-label use of sirolimus has demonstrated clinical benefit in these patients. This systematic review evaluates the efficacy and safety of sirolimus in the management of venous malformations. <b><i>Methods and Results:</i></b> A systematic literature search identified 26 studies comprising 98 patients treated with sirolimus for venous malformations. Most studies were case reports or case series (77%), with 15% prospective trials and 8% retrospective analyses. Sirolimus, primarily administered orally, yielded some level of clinical improvement in 72% of reported patients, with consistent symptomatic relief observed in coagulopathy, bleeding, anemia, pain, and improved function. However, reduction in the size of the malformation varied and treatment-limiting side effects were observed. <b><i>Conclusions:</i></b> Oral sirolimus is a promising treatment option for venous malformations, especially for those with deep venous malformations. A direct, targeted topical treatment could provide clinical benefit for the cutaneous manifestation without the risk of off-target effects as occur with oral sirolimus. Further prospective, controlled studies are warranted to elucidate sirolimus's role in managing venous malformations.</p>","PeriodicalId":18168,"journal":{"name":"Lymphatic research and biology","volume":" ","pages":""},"PeriodicalIF":1.9000,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Lymphatic research and biology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/15578585251377562","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}
引用次数: 0
Abstract
Background: Venous malformations can cause substantial morbidity and long-term complications. There are no Food and Drug Administration (FDA)-approved therapies for the treatment of venous malformations. However, off-label use of sirolimus has demonstrated clinical benefit in these patients. This systematic review evaluates the efficacy and safety of sirolimus in the management of venous malformations. Methods and Results: A systematic literature search identified 26 studies comprising 98 patients treated with sirolimus for venous malformations. Most studies were case reports or case series (77%), with 15% prospective trials and 8% retrospective analyses. Sirolimus, primarily administered orally, yielded some level of clinical improvement in 72% of reported patients, with consistent symptomatic relief observed in coagulopathy, bleeding, anemia, pain, and improved function. However, reduction in the size of the malformation varied and treatment-limiting side effects were observed. Conclusions: Oral sirolimus is a promising treatment option for venous malformations, especially for those with deep venous malformations. A direct, targeted topical treatment could provide clinical benefit for the cutaneous manifestation without the risk of off-target effects as occur with oral sirolimus. Further prospective, controlled studies are warranted to elucidate sirolimus's role in managing venous malformations.
期刊介绍:
Lymphatic Research and Biology delivers the most current peer-reviewed advances and developments in lymphatic biology and pathology from the world’s leading biomedical investigators. The Journal provides original research from a broad range of investigative disciplines, including genetics, biochemistry and biophysics, cellular and molecular biology, physiology and pharmacology, anatomy, developmental biology, and pathology.
Lymphatic Research and Biology coverage includes:
-Vasculogenesis and angiogenesis
-Genetics of lymphatic disorders
-Human lymphatic disease, including lymphatic insufficiency and associated vascular anomalies
-Physiology of intestinal fluid and protein balance
-Immunosurveillance and immune cell trafficking
-Tumor biology and metastasis
-Pharmacology
-Lymphatic imaging
-Endothelial and smooth muscle cell biology
-Inflammation, infection, and autoimmune disease