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Lung function response and side effects to rapamycin for lymphangioleiomyomatosis: a prospective national cohort study

Bee, Janet; Fuller, Sharon; Miller, Suzanne; Johnson, Simon R.

Authors

Janet Bee

Sharon Fuller

Suzanne Miller

Simon R. Johnson Simon.Johnson@nottingham.ac.uk



Abstract

Rationale

Mechanistic target of rapamycin inhibitors reduce loss of lung function in lymphangioleiomyomatosis (LAM), although their benefit varies between individuals. We examined lung function response and side effects to rapamycin in a national cohort.

Methods

Subjects were receiving rapamycin for progressive lung disease. Clinical evaluation, detailed phenotyping, serial lung function, rapamycin and safety monitoring were performed according to a clinical protocol. Lung function change, measured as FEV1 slope (ΔFEV1), was reported for those treated for 1 year or longer.

Results

Rapamycin was associated with improved ΔFEV1 in 21 individuals where pretreatment data were available (p

Journal Article Type Article
Journal Thorax
Print ISSN 0040-6376
Electronic ISSN 0040-6376
Publisher BMJ Publishing Group
Peer Reviewed Peer Reviewed
APA6 Citation Bee, J., Fuller, S., Miller, S., & Johnson, S. R. (in press). Lung function response and side effects to rapamycin for lymphangioleiomyomatosis: a prospective national cohort study. Thorax, doi:10.1136/thoraxjnl-2017-210872
DOI https://doi.org/10.1136/thoraxjnl-2017-210872
Publisher URL https://doi.org/10.1136/thoraxjnl-2017-210872
Copyright Statement Copyright information regarding this work can be found at the following address: http://eprints.nottingh.../end_user_agreement.pdf
Additional Information Copyright ©2017 BMJ Publishing Group Ltd & British Thoracic Society

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