American journal of respiratory and critical care medicine
-
Am. J. Respir. Crit. Care Med. · Jan 2018
Randomized Controlled Trial Multicenter StudyTezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR.
Tezacaftor (formerly VX-661) is an investigational small molecule that improves processing and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) in vitro, and improves CFTR function alone and in combination with ivacaftor. ⋯ These results support continued clinical development of tezacaftor (100 mg every day) in combination with ivacaftor (150 mg every 12 h) in subjects with cystic fibrosis. Clinical trial registered with www.clinicaltrials.gov (NCT01531673).
-
Am. J. Respir. Crit. Care Med. · Jan 2018
Multicenter StudyQuantitative Evidence for Revising the Definition of Primary Graft Dysfunction After Lung Transplant.
Primary graft dysfunction (PGD) is a form of acute lung injury that occurs after lung transplantation. The definition of PGD was standardized in 2005. Since that time, clinical practice has evolved, and this definition is increasingly used as a primary endpoint for clinical trials; therefore, validation is warranted. ⋯ The PGD consensus definition can be simplified by combining lower PGD grades. Construct validity of grading was present regardless of transplant procedure type or use of mechanical ventilation. Additional severity categories had minimal impact on mortality or biomarker discrimination.
-
Am. J. Respir. Crit. Care Med. · Jan 2018
Comparative StudyMicrobial Lineages in Sarcoidosis: A Metagenomic Analysis Tailored for Low Microbial Content Samples.
The etiology of sarcoidosis is unknown, but microbial agents are suspected as triggers. ⋯ Metagenomic sequencing revealed enrichment of microbes in single types of sarcoidosis samples but limited concordance across sample types. Statistical analysis accounting for environmental contamination was essential to avoiding false positives.