학술논문
Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience.
Document Type
Article
Author
Revilla-López, Eva; Berastegui, Cristina; Méndez, Alejandra; Sáez-Giménez, Berta; Ruiz de Miguel, Victoria; López-Meseguer, Manuel; Monforte, Victor; Bravo, Carlos; Pujana, Miguel Angel; Ramon, Maria Antonia; Gómez-Ollés, Susana; Roman, Antonio; The Vall d'Hebron Multidisciplinary Cystic Lung Disease Group; Bello, Irene; Burgos, Rosa; Escobar, Roser; Ferrándiz-Pulido, Carla; Gómez, Alba; Pallisa, Esther; Palomares, Gloria
Source
Subject
*RAPAMYCIN
*LYMPHANGIOMYOMATOSIS
*LUNG transplantation
*DRUG efficacy
*MEDICAL statistics
*
*
*
*
Language
ISSN
2045-2322
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. [ABSTRACT FROM AUTHOR]