Nishant Gupta1, Hye-Seung Lee2, Jay H Ryu3, Angelo M Taveira-DaSilva4, Gerald J Beck5, Jar-Chi Lee5, Kevin McCarthy6, Geraldine A Finlay7, Kevin K Brown8, Stephen J Ruoss9, Nilo A Avila10, Joel Moss4, Francis X McCormack11. 1. Division of Pulmonary, Critical Care and Sleep Medicine, University of Cincinnati, Cincinnati, OH; Medical Service, Veterans Affairs Medical Center, Cincinnati, OH. Electronic address: guptans@ucmail.uc.edu. 2. Department of Pediatrics, Health Informatics Institute, University of South Florida, Tampa, FL. 3. Division of Pulmonary and Critical Care Medicine, Mayo Clinic College of Medicine, Rochester, MN. 4. Cardiovascular and Pulmonary Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD. 5. Department of Quantitative Health Sciences, Cleveland Clinic, Cleveland, OH. 6. Formerly at Respiratory Institute, Cleveland Clinic, Cleveland, OH. 7. Division of Pulmonary, Critical Care and Sleep Medicine, Tufts Medical Center, Boston, MA. 8. Department of Medicine, National Jewish Health, Denver, CO. 9. Division of Pulmonary and Critical Care Medicine, Stanford University School of Medicine, Stanford, CA. 10. Radiology Service, Veterans Affairs Medical Center, Washington, DC. 11. Division of Pulmonary, Critical Care and Sleep Medicine, University of Cincinnati, Cincinnati, OH; Medical Service, Veterans Affairs Medical Center, Cincinnati, OH.
Abstract
BACKGROUND: The natural history of lymphangioleiomyomatosis (LAM) is mainly derived from retrospective cohort analyses, and it remains incompletely understood. A National Institutes of Health LAM Registry was established to define the natural history and identify prognostic biomarkers that can help guide management and decision-making in patients with LAM. METHODS: A linear mixed effects model was used to compute the rate of decline of FEV1 and to identify variables affecting FEV1 decline among 217 registry patients who enrolled from 1998 to 2001. Prognostic variables associated with progression to death/lung transplantation were identified by using a Cox proportional hazards model. RESULTS: Mean annual decline of FEV1 was 89 ± 53 mL/year and remained remarkably constant regardless of baseline lung function. FEV1 decline was more rapid in those with greater cyst profusion on CT scanning (P = .02) and in premenopausal subjects (118 mL/year) compared with postmenopausal subjects (74 mL/year) (P = .003). There were 26 deaths and 43 lung transplantations during the evaluation period. The estimated 5-, 10-, 15-, and 20-year transplant-free survival rates were 94%, 85%, 75%, and 64%, respectively. Postmenopausal status (hazard ratio, 0.30; P = .0002) and higher baseline FEV1 (hazard ratio, 0.97; P = .008) or diffusion capacity of lung for carbon monoxide (hazard ratio, 0.97; P = .001) were independently associated with a lower risk of progression to death or lung transplantation. CONCLUSIONS: The median transplant-free survival in patients with LAM is > 20 years. Menopausal status, as well as structural and physiologic markers of disease severity, significantly affect the rate of decline of FEV1 and progression to death or lung transplantation in LAM. Published by Elsevier Inc.
BACKGROUND: The natural history of lymphangioleiomyomatosis (LAM) is mainly derived from retrospective cohort analyses, and it remains incompletely understood. A National Institutes of Health LAM Registry was established to define the natural history and identify prognostic biomarkers that can help guide management and decision-making in patients with LAM. METHODS: A linear mixed effects model was used to compute the rate of decline of FEV1 and to identify variables affecting FEV1 decline among 217 registry patients who enrolled from 1998 to 2001. Prognostic variables associated with progression to death/lung transplantation were identified by using a Cox proportional hazards model. RESULTS: Mean annual decline of FEV1 was 89 ± 53 mL/year and remained remarkably constant regardless of baseline lung function. FEV1 decline was more rapid in those with greater cyst profusion on CT scanning (P = .02) and in premenopausal subjects (118 mL/year) compared with postmenopausal subjects (74 mL/year) (P = .003). There were 26 deaths and 43 lung transplantations during the evaluation period. The estimated 5-, 10-, 15-, and 20-year transplant-free survival rates were 94%, 85%, 75%, and 64%, respectively. Postmenopausal status (hazard ratio, 0.30; P = .0002) and higher baseline FEV1 (hazard ratio, 0.97; P = .008) or diffusion capacity of lung for carbon monoxide (hazard ratio, 0.97; P = .001) were independently associated with a lower risk of progression to death or lung transplantation. CONCLUSIONS: The median transplant-free survival in patients with LAM is > 20 years. Menopausal status, as well as structural and physiologic markers of disease severity, significantly affect the rate of decline of FEV1 and progression to death or lung transplantation in LAM. Published by Elsevier Inc.
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