Efficacy of Sirolimus in Patients Requiring Tracheostomy for Life-Threatening Lymphatic Malformation of the Head and Neck : A Report From the European Reference Network

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dc.contributor.author Holm, Annegret
dc.contributor.author te Loo, Maroeska
dc.contributor.author Schultze Kool, Leo
dc.contributor.author Salminen, Päivi
dc.contributor.author Celis, Veronica
dc.contributor.author Baselga, Eulalia
dc.contributor.author Duignan, Sophie
dc.contributor.author Dvorakova, Veronika
dc.contributor.author Irvine, Alan D.
dc.contributor.author Boon, Laurence M.
dc.contributor.author Vikkula, Miikka
dc.contributor.author Ghaffarpour, Nader
dc.contributor.author Niemeyer, Charlotte M.
dc.contributor.author Rössler, Jochen
dc.contributor.author Kapp, Friedrich G.
dc.date.accessioned 2021-11-01T07:03:04Z
dc.date.available 2021-11-01T07:03:04Z
dc.date.issued 2021-09-30
dc.identifier.citation Holm , A , te Loo , M , Schultze Kool , L , Salminen , P , Celis , V , Baselga , E , Duignan , S , Dvorakova , V , Irvine , A D , Boon , L M , Vikkula , M , Ghaffarpour , N , Niemeyer , C M , Rössler , J & Kapp , F G 2021 , ' Efficacy of Sirolimus in Patients Requiring Tracheostomy for Life-Threatening Lymphatic Malformation of the Head and Neck : A Report From the European Reference Network ' , Frontiers in pediatrics , vol. 9 , 697960 . https://doi.org/10.3389/fped.2021.697960
dc.identifier.other PURE: 169959667
dc.identifier.other PURE UUID: 0915f54f-b5d2-455a-9532-e13359ddd8ab
dc.identifier.other Scopus: 85117148230
dc.identifier.other WOS: 000708464600001
dc.identifier.uri http://hdl.handle.net/10138/335886
dc.description Publisher Copyright: © Copyright © 2021 Holm, te Loo, Schultze Kool, Salminen, Celis, Baselga, Duignan, Dvorakova, Irvine, Boon, Vikkula, Ghaffarpour, Niemeyer, Rössler and Kapp.
dc.description.abstract 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. en
dc.format.extent 8
dc.language.iso eng
dc.relation.ispartof Frontiers in pediatrics
dc.rights cc_by
dc.rights.uri info:eu-repo/semantics/openAccess
dc.subject lymphatic malformation
dc.subject rapamycin
dc.subject sirolimus
dc.subject tracheostoma
dc.subject tracheostomy
dc.subject vascular anomaly
dc.subject 3123 Gynaecology and paediatrics
dc.title Efficacy of Sirolimus in Patients Requiring Tracheostomy for Life-Threatening Lymphatic Malformation of the Head and Neck : A Report From the European Reference Network en
dc.type Article
dc.contributor.organization HUS Children and Adolescents
dc.contributor.organization Lastenkirurgian yksikkö
dc.description.reviewstatus Peer reviewed
dc.relation.doi https://doi.org/10.3389/fped.2021.697960
dc.relation.issn 2296-2360
dc.rights.accesslevel openAccess
dc.type.version publishedVersion
dc.identifier.url http://www.scopus.com/inward/record.url?scp=85117148230&partnerID=8YFLogxK

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