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Advances in and Limitations of Up-and-down Methodology: A Précis of Clinical Use, Study Design, and Dose Estimation in Anesthesia Research
Sequential design methods for binary response variables exist for determination of the concentration or dose associated with the 50% point along the dose–response curve; the up-and-down method ofExpand
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Progressive preclinical interstitial lung disease in rheumatoid arthritis.
BACKGROUND Early detection and treatment for interstitial lung disease (ILD) in patients with rheumatoid arthritis (RA) may ameliorate disease progression. The objective of this study was to identifyExpand
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Risk factors for hospital morbidity and mortality after the Norwood procedure: A report from the Pediatric Heart Network Single Ventricle Reconstruction trial.
OBJECTIVES We sought to identify risk factors for mortality and morbidity during the Norwood hospitalization in newborn infants with hypoplastic left heart syndrome and other single right ventricleExpand
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Dose finding using the biased coin up-and-down design and isotonic regression.
We are interested in finding a dose that has a prespecified toxicity rate in the target population. In this article, we investigate five estimators of the target dose to be used with the up-and-downExpand
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Atenolol versus losartan in children and young adults with Marfan's syndrome.
BACKGROUND Aortic-root dissection is the leading cause of death in Marfan's syndrome. Studies suggest that with regard to slowing aortic-root enlargement, losartan may be more effective thanExpand
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Rationale and design of a randomized clinical trial of beta-blocker therapy (atenolol) versus angiotensin II receptor blocker therapy (losartan) in individuals with Marfan syndrome.
BACKGROUND Cardiovascular disease, including aortic root dilation, dissection, and rupture, is the leading cause of mortality in patients with Marfan syndrome (MFS). The maximal aortic root diameterExpand
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Changes in lung function and chylous effusions in patients with lymphangioleiomyomatosis treated with sirolimus.
BACKGROUND Lymphangioleiomyomatosis (LAM) is a disorder that affects women and is characterized by cystic lung destruction, chylous effusions, lymphangioleiomyomas, and angiomyolipomas. It is causedExpand
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Bacterial colonization of the respiratory tract following tracheal intubation—Effect of gravity: An experimental study*
ObjectiveTo explore the role of the horizontal orientation of endotracheal tube and neck on bacterial colonization of the respiratory tract in anesthetized sheep on mechanical ventilation, withoutExpand
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Estimating the probability of toxicity at the target dose following an up-and-down design.
One of the most important aspects of a phase I trial or other acute toxicity study is estimating accurately the probability of toxicity that is associated with the recommended dose. We use the biasedExpand
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Reversible airflow obstruction, proliferation of abnormal smooth muscle cells, and impairment of gas exchange as predictors of outcome in lymphangioleiomyomatosis.
Lymphangioleiomyomatosis (LAM) is a rare disease, occurring in women, characterized by cystic degeneration of the lungs, abdominal tumors, and proliferation of abnormal smooth muscle cells. LungExpand
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