Ontology highlight
ABSTRACT:
SUBMITTER: El-Chemaly S
PROVIDER: S-EPMC6026235 | biostudies-literature | 2017 Jun
REPOSITORIES: biostudies-literature
El-Chemaly Souheil S Taveira-Dasilva Angelo A Goldberg Hilary J HJ Peters Elizabeth E Haughey Mary M Bienfang Don D Jones Amanda M AM Julien-Williams Patricia P Cui Ye Y Villalba Julian A JA Bagwe Shefali S Maurer Rie R Rosas Ivan O IO Moss Joel J Henske Elizabeth P EP
Chest 20170210 6
<h4>Background</h4>Animal and cellular studies support the importance of autophagy inhibition in lymphangioleiomyomatosis (LAM). In a cohort of subjects with LAM, we tested the hypothesis that treatment with sirolimus and hydroxychloroquine (an autophagy inhibitor) at two different dose levels is safe and well tolerated. Secondary end points included changes in lung function.<h4>Methods</h4>This 48-week, two-center phase I trial evaluated the safety of escalating oral hydroxychloroquine doses (1 ...[more]