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A Phase II Clinical Trial of an Aromatase Inhibitor for Postmenopausal Women with Lymphangioleiomyomatosis.

Calvin LuHye-Seung LeeGeorge P PappasDaniel F DillingCharles D BurgerAdrian ShifrenSrihari VeeraraghavanJeffrey T ChapmanJoseph ParambilStephen J RuossLisa R YoungStephen R HammesElizabeth J KoprasTammy RoadsJeffrey P KrischerFrancis X McCormacknull null
Published in: Annals of the American Thoracic Society (2018)
Letrozole treatment appears to be safe and well tolerated in postmenopausal patients with LAM, including those taking sirolimus. Enrollment in this trial was compromised by the publication of an effective treatment (sirolimus) in the same month as the study opened, resulting in limited power to detect treatment effects. Post hoc matched pairs exploration studies provide tentative support for additional studies of letrozole in LAM. Considering the reduced rate of lung function decline in postmenopausal patients, future studies will likely require enhanced study designs, such as selective enrollment of those with prognostic biomarkers predictive of decline. Clinical trial registered with www.clinicaltrials.gov (NCT01353209).
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