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Title: Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience
Author: Revilla López, Eva
Berastegui, Cristina
Méndez, Alejandra
Sáez Giménez, Berta
Ruiz de Miguel, Victoria
López Meseguer, Manuel
Monforte, Victor
Bravo, Carlos
Pujana Genestar, M. Ángel
Ramon, Maria Antonia
Gómez Ollés, Susana
Roman, Antonio
Vall d’Hebron Multidisciplinary Cystic Lung Disease Group
Keywords: Malalties dels pulmons
Pulmonary diseases
Issue Date: 13-May-2021
Publisher: Springer Nature
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.
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It is part of: Scientific Reports, 2021, vol. 11
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Appears in Collections:Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL))

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