Title:
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Long-term results of sirolimus treatment in lymphangioleiomyomatosis : a single referral centre experience
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Author:
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Revilla-López, Eva; Berastegui García, Cristina; Méndez, Alejandra; Sáez-Giménez, Berta; Ruiz de Miguel, Victoria; López-Meseguer, Manuel; Monforte, Víctor; Bravo, Carlos; Pujana, Miguel Angel; Ramon, Maria Antonia; Gómez-Ollés, Susana; Roman, Antonio; Universitat Autònoma de Barcelona
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Abstract:
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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 FEV decline greater than − 75 ml/year. A mixed-effects model was used to estimate the longitudinal changes in FEV (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) FEV 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 |
Subject(s):
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-Respiratory tract diseases -Diagnosis -Drug therapy |
Rights:
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open access
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https://creativecommons.org/licenses/by/4.0/ |
Document type:
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Article |
Published by:
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Share:
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Uri:
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https://ddd.uab.cat/record/255568
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