Expert review of respiratory medicine
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Expert Rev Respir Med · Jan 2019
ReviewAcute and chronic non-pulmonary complications in adults with cystic fibrosis.
Cystic fibrosis (CF) is a genetic disease that primarily affects the respiratory system and often leads to respiratory failure and premature death. Although pulmonary complications contribute to 85% of deaths, non-pulmonary complications are responsible for significant morbidity and mortality in adults with CF. Areas covered: This review summarizes acute and chronic non-pulmonary complications in CF patients, with emphasis on emerging complications and in the context of the current growth and aging of the CF adult population. ⋯ Expert commentary: Acute non-pulmonary complications have been well described but should be recognized and managed carefully. Managing chronic non-pulmonary complications is an important and changing aspect of CF patient care, particularly with the emergence of novel complications in adults. Early detection of non-pulmonary complications is essential to the development of prevention and treatment strategies that aim to further improve the survival and health status of adult CF patients.
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Expert Rev Respir Med · Dec 2018
ReviewSolriamfetol for the treatment of daytime sleepiness in obstructive sleep apnea.
Introduction: Obstructive sleep apnea (OSA) is highly prevalent and constitutes a major health hazard. Current pharmacotherapy is ineffective in correcting sleep-disordered breathing and is used adjunctively to address residual sleepiness. A new drug, solriamfetol, a selective norepinephrine-dopamine reuptake inhibitor, is the first drug of its class that is being considered by the US Food and Drug Administration (FDA) to treat excessive sleepiness in OSA and narcolepsy patients. ⋯ Expert commentary: Solriamfetol shows promise as adjunctive therapy in OSA. It is well tolerated and effective in reducing sleepiness and is an alternative to modafinil or armodafinil. Unlike stimulants like methylphenidate or dextroamphetamine, it does not have cardiac effects, rebound hypersomnia, or withdrawal effects.
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Expert Rev Respir Med · Sep 2018
ReviewAssessing effects of inhaled antibiotics in adults with non-cystic fibrosis bronchiectasis--experiences from recent clinical trials.
Non-cystic fibrosis bronchiectasis (NCFB) results from a permanent and progressive destruction of the airways leading to poor lung function. NCFB is characterized by recurrent lung infection, sputum production, and cough, often requiring long-term antibiotic therapy and hospitalization. At present, there are no approved therapies available. Clinical trials of inhaled antibiotics have shown promise against sputum bacterial load, but mixed results on clinical outcomes. Areas covered: The objective of this review is to provide an overview of NCFB and critically evaluate the evidence supporting the outcome measures used in recent clinical trials of inhaled antibiotics. These include quantitative changes in bacterial load, sputum purulence and yield, inflammatory markers, and lung function, as well as clinical changes in exacerbations, exacerbation frequency, hospitalizations, and health-related quality of life. Expert commentary: Recently completed large trials of inhaled antibiotics in NCFB did not consistently meet pre-specified end points, suggesting that we have not yet found the best enrollment criteria or outcome measures to evaluate efficacy, although reduced exacerbation frequency may be clinically most meaningful. Future trials may focus on specific patient populations at high risk with new information obtained through analyses of large international patient registries. ⋯ 6-MWT: Six-Minute Walk Test; AIR-BX: Aztreonam for Inhalation Solution in Patients with Non-Cystic Fibrosis Bronchiectasis trial; BSI: Bronchiectasis Severity Index; CAT: COPD Assessment Test; CF: Cystic Fibrosis; CFTR: Cystic Fibrosis Transmembrane Conductance Regulator; CFU: Colony-Forming Units; COPD: Chronic Obstructive Pulmonary Disease; CRP: C-Reactive Protein; DPI: Dry Powder for Inhalation; EMA: European Medicines Agency; ERS: European Respiratory Society; FACED: FEV1, Age, Chronic colonization by P. aeruginosa, Extension of bronchiectasis and Dyspnea; FDA: US Food and Drug Administration; FEV1: Forced Expiration in 1 s; FVC: Forced Vital Capacity; HFCC: High-Frequency Chest Compression; HRCT: High-Resolution Computed Tomography; HRQoL: Health-Related Quality of Life; LCQ: Leicester Cough Questionnaire; MID: Minimal Important Difference; NCFB: Non-Cystic Fibrosis Bronchiectasis; NTM: Nontuberculous Mycobacteria; ORBIT: Once-daily Respiratory Bronchiectasis Inhalation Treatment trial; PRO: Patient-Reported Outcomes; QoL-B: Quality of Life-Bronchiectasis; SGRQ: St. George's Respiratory Questionnaire; SWT: Shuttle Walk Test; TORCH: Towards a Revolution in COPD Health trial; UPLIFT: Understanding Potential Long-term Impacts on Function with Tiotropium trial.
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Cystic fibrosis (CF) is the most common, life-limiting autosomal recessive disease in Caucasians, and is caused by defects in production of the CFTR ion channel. Until recently, there were no available treatments targeting the disease-causing defects in CFTR but newly developed CFTR modulators are changing the course of disease in CF. The newest modulator, tezacaftor, is a CFTR corrector that was recently approved by the FDA to be used in combination with the first approved CFTR potentiator, ivacaftor. ⋯ It is also approved for a large number of non-F508del, residual function mutations that are predicted to respond based on in vitro testing. The horizon for continued improvements in CFTR-targeted treatments is promising, with three-drug combinations currently in Phase 3 clinical trials, and other drugs with novel mechanisms of action being studied. Within the next 5 years, the vast majority of patients with CF are expected to have a modulator approved for their genotype.
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Expert Rev Respir Med · Aug 2018
ReviewThe use of bronchoscopy in critically ill patients: considerations and complications.
Flexible bronchoscopy has been well established for diagnostic and therapeutic purposes in critically ill patients. Areas covered: This review outlines the clinical evidence of the utility and safety of flexible bronchoscopy in the intensive care unit, as well as specific considerations, including practical points and potential complications, in critically ill patients. ⋯ The decision to perform flexible bronchoscopy can only be made by trade-offs between potential risks and benefits because of the fragile nature of the critically ill. Flexible bronchoscopy-associated serious adverse events are inevitable in cases of a lack of expertise or appropriate precautions.