Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience

Abstract There are few published data on long-term treatment with sirolimus in lymphangioleiomyomatosis (LAM). The objective of this study was to describe the long-term effect of sirolimus in a series of LAM patients followed up in a referral centre, focusing on pulmonary function. We retrospectivel...

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Autores principales:	Eva Revilla-López, Cristina Berastegui, Alejandra Méndez, Berta Sáez-Giménez, Victoria Ruiz de Miguel, Manuel López-Meseguer, Victor Monforte, Carlos Bravo, Miguel Angel Pujana, Maria Antonia Ramon, Susana Gómez-Ollés, Antonio Roman, The Vall d’Hebron Multidisciplinary Cystic Lung Disease Group
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/d41b30c353e24ea39210f9e0a6644bc1
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spelling	oai:doaj.org-article:d41b30c353e24ea39210f9e0a6644bc12021-12-02T16:50:31ZLong-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience10.1038/s41598-021-89562-02045-2322https://doaj.org/article/d41b30c353e24ea39210f9e0a6644bc12021-05-01T00:00:00Zhttps://doi.org/10.1038/s41598-021-89562-0https://doaj.org/toc/2045-2322Abstract There are few published data on long-term treatment with sirolimus in lymphangioleiomyomatosis (LAM). The objective of this study was to describe the long-term effect of sirolimus in a series of LAM patients followed up in a referral centre, focusing on pulmonary function. We retrospectively reviewed a series of 48 patients with LAM diagnosed, followed up and treated with sirolimus in a single centre. Response to sirolimus was evaluated at 1 and 5 years. A negative sirolimus response was defined as an FEV1 decline greater than − 75 ml/year. A mixed-effects model was used to estimate the longitudinal changes in FEV1 (average slope), both as absolute (ml/year) and as predicted values (%predicted/year). From a total of 48 patients, 9 patients underwent lung transplantation and 4 died during the study. Mean (95% CI) FEV1 slope over 5 years was − 0.14 (− 26.13 to 25.85) ml/year in the whole LAM group, 42.55 (14.87 to 70.22) ml/year in the responder group, − 54.00 (− 71.60 to − 36.39) ml/year in the partial responder group and − 84.19 (− 113.5 to − 54.0) ml/year in the non-responder group. After 5 years of sirolimus treatment 59% had a positive response, 30% had a partial response and 11% had a negative response. Our study found that sirolimus treatment had a positive long-term effect on most LAM patients.Eva Revilla-LópezCristina BerasteguiAlejandra MéndezBerta Sáez-GiménezVictoria Ruiz de MiguelManuel López-MeseguerVictor MonforteCarlos BravoMiguel Angel PujanaMaria Antonia RamonSusana Gómez-OllésAntonio RomanThe Vall d’Hebron Multidisciplinary Cystic Lung Disease GroupNature PortfolioarticleMedicineRScienceQENScientific Reports, Vol 11, Iss 1, Pp 1-9 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Eva Revilla-López Cristina Berastegui Alejandra Méndez Berta Sáez-Giménez Victoria Ruiz de Miguel Manuel López-Meseguer Victor Monforte Carlos Bravo Miguel Angel Pujana Maria Antonia Ramon Susana Gómez-Ollés Antonio Roman The Vall d’Hebron Multidisciplinary Cystic Lung Disease Group Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience
description	Abstract There are few published data on long-term treatment with sirolimus in lymphangioleiomyomatosis (LAM). The objective of this study was to describe the long-term effect of sirolimus in a series of LAM patients followed up in a referral centre, focusing on pulmonary function. We retrospectively reviewed a series of 48 patients with LAM diagnosed, followed up and treated with sirolimus in a single centre. Response to sirolimus was evaluated at 1 and 5 years. A negative sirolimus response was defined as an FEV1 decline greater than − 75 ml/year. A mixed-effects model was used to estimate the longitudinal changes in FEV1 (average slope), both as absolute (ml/year) and as predicted values (%predicted/year). From a total of 48 patients, 9 patients underwent lung transplantation and 4 died during the study. Mean (95% CI) FEV1 slope over 5 years was − 0.14 (− 26.13 to 25.85) ml/year in the whole LAM group, 42.55 (14.87 to 70.22) ml/year in the responder group, − 54.00 (− 71.60 to − 36.39) ml/year in the partial responder group and − 84.19 (− 113.5 to − 54.0) ml/year in the non-responder group. After 5 years of sirolimus treatment 59% had a positive response, 30% had a partial response and 11% had a negative response. Our study found that sirolimus treatment had a positive long-term effect on most LAM patients.
format	article
author	Eva Revilla-López Cristina Berastegui Alejandra Méndez Berta Sáez-Giménez Victoria Ruiz de Miguel Manuel López-Meseguer Victor Monforte Carlos Bravo Miguel Angel Pujana Maria Antonia Ramon Susana Gómez-Ollés Antonio Roman The Vall d’Hebron Multidisciplinary Cystic Lung Disease Group
author_facet	Eva Revilla-López Cristina Berastegui Alejandra Méndez Berta Sáez-Giménez Victoria Ruiz de Miguel Manuel López-Meseguer Victor Monforte Carlos Bravo Miguel Angel Pujana Maria Antonia Ramon Susana Gómez-Ollés Antonio Roman The Vall d’Hebron Multidisciplinary Cystic Lung Disease Group
author_sort	Eva Revilla-López
title	Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience
title_short	Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience
title_full	Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience
title_fullStr	Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience
title_full_unstemmed	Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience
title_sort	long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/d41b30c353e24ea39210f9e0a6644bc1
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Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience

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