Fauroux B Why, when and how to propose noninvasive ventilation in cystic fibrosis? Minerva Anestesiol. 2011 Nov;77(11):1108-14. Epub 2011 May 20. Review.
Flume PA Pulmonary complications of cystic fibrosis. Respir Care. 2009 May;54(5):618-27. Review.
Granton JT, Kesten S The acute effects of nasal positive pressure ventilation in patients with advanced cystic fibrosis. Chest. 1998 Apr;113(4):1013-8.
Jones A, Bilton D, Evans TW, Finney SJ Predictors of outcome in patients with cystic fibrosis requiring endotracheal intubation. Respirology. 2013 May;18(4):630-6. doi: 10.1111/resp.12051.
Pittman JE, Cutting G, Davis SD, Ferkol T, Boucher R Cystic fibrosis: NHLBI Workshop on the Primary Prevention of Chronic Lung Diseases. Ann Am Thorac Soc. 2014 Apr;11 Suppl 3:S161-8. doi: 10.1513/AnnalsATS.201312-444LD. Review.
Ricard JD High flow nasal oxygen in acute respiratory failure. Minerva Anestesiol. 2012 Jul;78(7):836-41. Epub 2012 Apr 24. Review.
Rittayamai N, Tscheikuna J, Rujiwit P High-flow nasal cannula versus conventional oxygen therapy after endotracheal extubation: a randomized crossover physiologic study. Respir Care. 2014 Apr;59(4):485-90. doi: 10.4187/respcare.02397. Epub 2013 Sep 17.
Serra A, Polese G, Braggion C, Rossi A Non-invasive proportional assist and pressure support ventilation in patients with cystic fibrosis and chronic respiratory failure. Thorax. 2002 Jan;57(1):50-4.
Sood N, Paradowski LJ, Yankaskas JR Outcomes of intensive care unit care in adults with cystic fibrosis. Am J Respir Crit Care Med. 2001 Feb;163(2):335-8.
Young AC, Wilson JW, Kotsimbos TC, Naughton MT Randomised placebo controlled trial of non-invasive ventilation for hypercapnia in cystic fibrosis. Thorax. 2008 Jan;63(1):72-7. Epub 2007 Aug 3.
Randomized Cross-over Physiologic Study of High Flow Nasal Oxygen Cannula Versus Non-invasive Ventilation in Cystic Fibrosis. The HIFEN Study
Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.
Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.
Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.
Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.
Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.
