RT Journal Article
SR Electronic
T1 LYMPHANGIOLEIOMYOMATOSIS AND SIROLIMUS: MEDIUM-TERM IMPACT ON PULMONARY FUNCTION
JF European Respiratory Journal
JO Eur Respir J
FD European Respiratory Society
SP PA2372
DO 10.1183/13993003.congress-2021.PA2372
VO 58
IS suppl 65
A1 Marta Garcia Moyano
A1 Amaia Urrutia
A1 Leyre Serrano
A1 Ainhoa Gómez
A1 Sonia Castro
A1 Imanol González
A1 Jone Solorzano
A1 Carmen Jaca
A1 Pedro Ansola
A1 Milagros Iriberri
YR 2021
UL http://erj.ersjournals.com/content/58/suppl_65/PA2372.abstract
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).