PT  - JOURNAL ARTICLE
AU  - Marta Garcia Moyano
AU  - Amaia Urrutia
AU  - Leyre Serrano
AU  - Ainhoa Gómez
AU  - Sonia Castro
AU  - Imanol González
AU  - Jone Solorzano
AU  - Carmen Jaca
AU  - Pedro Ansola
AU  - Milagros Iriberri
TI  - LYMPHANGIOLEIOMYOMATOSIS AND SIROLIMUS: MEDIUM-TERM IMPACT ON PULMONARY FUNCTION
AID  - 10.1183/13993003.congress-2021.PA2372
DP  - 2021 Sep 05
TA  - European Respiratory Journal
PG  - PA2372
VI  - 58
IP  - suppl 65
4099  - http://erj.ersjournals.com/content/58/suppl_65/PA2372.short
4100  - http://erj.ersjournals.com/content/58/suppl_65/PA2372.full
SO  - Eur Respir J2021 Sep 05; 58
AB  - Introduction: Lymphangioleiomyomatosis (LAM) is a rare and progressive disease that usually leads to respiratory failure. Short-term treatment with sirolimus has shown to stabilize pulmonary function (PF) but there are few data on medium-term results.Aim: describe the impact on PF of sirolimus.Methods: 12 patients with LAM, from tertiary referral centre. October 2014-January 2021, 8 treated with sirolimus, retrospectively included. PF response to sirolimus at 1 and 2 years after treatment was evaluated. Negative response: decrease in FEV1 greater than 20 mL/year (physiological age-related decline).Results: 12 LAM women, mean age at diagnosis 47 (table 1): 8 treated with sirolimus. The other 4 were not treated (normal baseline PF tests and absence of angyomiolipoma). No side effects were reported (infections, diarrea or nausea). A positive response at 1 and 2nd year was observed in 100%. Mean PF test values at baselines, after 1 and 2nd years of treatment: table 2. Clinical improvement was noticed (mMRC dyspnea scale). Despite of taking sirolimus, due to respiratory impairment already noticed at diagnosis, 3 patients underwent pretrasplant study.Conclusions: 1. Sirolimus has a positive medium-term impact in LAM, with no serious side effects. 2. This study encourages to treat LAM with m-TOR inhibitors as no progression was shown in treated ones. 3. More studies at long term are needed to asses the impact of this treatment.FootnotesCite this article as: European Respiratory Journal 2021; 58: Suppl. 65, PA2372.This abstract was presented at the 2021 ERS International Congress, in session “Prediction of exacerbations in patients with COPD”.This is an ERS International Congress abstract. No full-text version is available. Further material to accompany this abstract may be available at www.ers-education.org (ERS member access only).