期刊
FRONTIERS IN PEDIATRICS
卷 9, 期 -, 页码 -出版社
FRONTIERS MEDIA SA
DOI: 10.3389/fped.2021.697960
关键词
sirolimus; rapamycin; lymphatic malformation; vascular anomaly; tracheostomy; tracheostoma
类别
资金
- Center for Vascular Anomalies, Freiburg Center for Rare Diseases
- EXCEL-Fellowship of the Faculty of Medicine, University of Freiburg - Else-Kroner-Fresenius-Stiftung
- Baden-Wuerttemberg Ministry of Science, Research and Art
- University of Freiburg
Sirolimus can be considered as an effective and safe salvage treatment for patients with extensive lymphatic malformations even after tracheostomy, as it can successfully improve respiratory conditions and reduce reliance on tracheostomy. Further research is needed to better understand when to initiate sirolimus therapy, the duration of treatment, and factors predicting treatment response.
Extensive lymphatic malformations (LMs) of the head and neck region may require tracheostomy to secure the airway. Treatment of these life-threatening LMs is usually multimodal and includes sclerotherapy and surgery, among others. Recently, systemic therapy with sirolimus has been introduced as an effective treatment for venous and lymphatic malformations; its efficacy and safety profile in patients with extensive LM requiring tracheostomy are, however, as yet not fully known. We performed a retrospective, multicenter review and identified 13 patients with an extensive LM of the head and neck region, who previously underwent placement of tracheostomy and subsequently received sirolimus treatment with the aim to improve the local respiratory situation and remove the tracheostomy. Under sirolimus therapy, tracheostomy could be reversed in 8/13 (62%) patients, a further 2/13 (15%) patients improved markedly, and removal of the tracheostomy was planned at the time of writing, while 3/13 (23%) patients showed insufficient or absent response to sirolimus, rendering tracheostomy reversal not feasible. The median duration of sirolimus treatment until removal of tracheostomy was 18 months (range, 8 months to 5.6 years). Adverse events of sirolimus therapy were common [10/13 (77%) patients], yet the majority of these were mild [9/10 (90%) patients] and only one severe adverse event was recorded, with ulceration and necrosis at a catheter insertion site. In conclusion, sirolimus can be considered an effective and safe salvage treatment in patients with extensive LM even after placement of a tracheostomy, as closure of the latter was possible in the majority of patients (62%) of our retrospective cohort. A better understanding of when to start sirolimus therapy, of the duration of treatment, and of factors allowing the prediction of treatment response will require further investigation.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据