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Efficacy and safety of sirolimus in lymphangioleiomyomatosis.
McCormack FX, Inoue Y, Moss J, Singer LG, Strange C, Nakata K, Barker AF, Chapman JT, Brantly ML, Stocks JM, Brown KK, Lynch JP 3rd, Goldberg HJ, Young LR, Kinder BW, Downey GP, Sullivan EJ, Colby TV, McKay RT, Cohen MM, Korbee L, Taveira-DaSilva AM, Lee HS, Krischer JP, Trapnell BC; National Institutes of Health Rare Lung Diseases Consortium; MILES Trial Group. McCormack FX, et al. N Engl J Med. 2011 Apr 28;364(17):1595-606. doi: 10.1056/NEJMoa1100391. Epub 2011 Mar 16. N Engl J Med. 2011. PMID: 21410393 Free PMC article. Clinical Trial.
St. George's Respiratory Questionnaire has longitudinal construct validity in lymphangioleiomyomatosis.
Swigris JJ, Lee HS, Cohen M, Inoue Y, Moss J, Singer LG, Young LR, McCormack FX; National Institutes of Health Rare Lung Diseases Consortium and the Multicenter International Lymphangioleiomyomatosis Efficacy and Safety of Sirolimus Trial Group. Swigris JJ, et al. Chest. 2013 Jun;143(6):1671-1678. doi: 10.1378/chest.12-0161. Chest. 2013. PMID: 23328755 Free PMC article.
Analysis of the MILES cohort reveals determinants of disease progression and treatment response in lymphangioleiomyomatosis.
Gupta N, Lee HS, Young LR, Strange C, Moss J, Singer LG, Nakata K, Barker AF, Chapman JT, Brantly ML, Stocks JM, Brown KK, Lynch JP 3rd, Goldberg HJ, Downey GP, Taveira-DaSilva AM, Krischer JP, Setchell K, Trapnell BC, Inoue Y, McCormack FX; NIH Rare Lung Disease Consortium. Gupta N, et al. Eur Respir J. 2019 Apr 4;53(4):1802066. doi: 10.1183/13993003.02066-2018. Print 2019 Apr. Eur Respir J. 2019. PMID: 30846465 Free PMC article.