Long-term results of sirolimus treatment in lymphangioleiomyomatosis : a single referral centre experience
Revilla-López, Eva (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Berastegui García, Cristina (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Méndez, Alejandra (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Sáez-Giménez, Berta (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Ruiz de Miguel, Victoria (Hospital Universitari Vall d'Hebron. Institut de Recerca)
López-Meseguer, Manuel (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Monforte, Víctor (Centro de Investigación Biomédica en Red de Enfermedades Respiratorias)
Bravo Masgoret, Carles (Centro de Investigación Biomédica en Red de Enfermedades Respiratorias)
Pujana, Miguel Angel (Institut d'Investigació Biomèdica de Bellvitge)
Ramon, Maria Antonia (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Gómez-Ollés, Susana (Centro de Investigación Biomédica en Red de Enfermedades Respiratorias)
Roman, Antonio (Centro de Investigación Biomédica en Red de Enfermedades Respiratorias)
Universitat Autònoma de Barcelona

Date:	2021
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 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.
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Subject:	Respiratory tract diseases ; Diagnosis ; Drug therapy
Published in:	Scientific reports, Vol. 11 (may 2021) , ISSN 2045-2322

DOI: 10.1038/s41598-021-89562-0
PMID: 33986388

9 p, 1.2 MB

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