Long-term results of sirolimus treatment in lymphangioleiomyomatosis: a single referral centre experience
Eva Revilla‐López, Cristina Berastegui, Alejandra Méndez, Berta Sáez, Victoria Ruiz de Miguel, Manuel López‐Meseguer, Vı́ctor Monforte, Carlos Bravo, Miguel Ángel Pujana, María Antonia Ramón, Susana Gómez‐Ollés, Antonio Román, The Vall d’Hebron Multidisciplinary Cystic Lung Disease Group, Irene Bello, R. Burgos, Roser Escobar, Carla Ferrándiz‐Pulido, Alba Gómez, Esther Pallisa, Gloria Palomares, Sabina Salicrú, A. L. Sánchez Martinez, Irene Sansano, Joana Sellarés
Abstract
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 1 decline greater than − 75 ml/year. A mixed-effects model was used to estimate the longitudinal changes in FEV 1 (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 1 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.