Analysis of the MILES cohort reveals determinants of disease progression and treatment response in lymphangioleiomyomatosis.

The Multicenter International Lymphangioleiomyomatosis (LAM) Efficacy of Sirolimus (MILES) trial revealed that sirolimus stabilised lung function in patients with moderately severe LAM. The purpose of this study was to further examine the MILES cohort for the effects of racial, demographic, clinical and physiological patient characteristics on disease progression and treatment response in LAM. MILES subjects were stratified on the basis of menopausal status (pre-menopausal/post-menopausal), race (Asian/Caucasian), bronchodilator responsiveness (present/absent), initial forced expiratory volume in 1 s (FEV In the MILES placebo group, pre-menopausal patients declined 5-fold faster than post-menopausal patients (mean±se FEV In LAM patients, treatment with sirolimus is beneficial regardless of menopausal status, race, bronchodilator responsiveness, baseline FEV

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Conflict of interest: N. Gupta has nothing to disclose. Conflict of interest: H-S. Lee has nothing to disclose. Conflict of interest: L.R. Young reports advisory board work for Boehringer Ingelheim and royalties for authorship from UpToDate, outside the submitted work; and has a patent Serum VEGF-D, no royalties issued. Conflict of interest: C. Strange reports grants for studies of LAM from Novartis, outside the submitted work. Conflict of interest: J. Moss has nothing to disclose. Conflict of interest: L.G. Singer has nothing to disclose. Conflict of interest: K. Nakata has nothing to disclose. Conflict of interest: A.F. Barker has nothing to disclose. Conflict of interest: J.T. Chapman has nothing to disclose. Conflict of interest: M.L. Brantly has nothing to disclose. Conflict of interest: J.M. Stocks has nothing to disclose. Conflict of interest: K.K. Brown reports grants from NHLBI, personal fees from AstraZeneca, Biogen, Galecto, MedImmune, Novartis, ProMetic, Patara, Third Pole, Galapagos, Boehringer Ingelheim, Theravance and Three Lakes Partners, conversation under CDA only from Genoa, other (submitted grant) from Roche/Genentech, outside the submitted work. Conflict of interest: J.P. Lynch has nothing to disclose. Conflict of interest: H.J. Goldberg has nothing to disclose. Conflict of interest: G.P. Downey has nothing to disclose. Conflict of interest: A.M. Taveira-DaSilva has nothing to disclose. Conflict of interest: J.P. Krischer has nothing to disclose. Conflict of interest: K. Setchell has nothing to disclose. Conflict of interest: B.C. Trapnell has nothing to disclose. Conflict of interest: Y. Inoue reports grants from Japanese Ministry of Health, Labor, and Welfare, during the conduct of the study. Conflict of interest: F.X. McCormack has a patent on serum VEGF-D testing. All royalties are waived to the parent institution, the University of Cincinnati.