Abstract:
:Introduction: Cystic fibrosis (CF) is a complex, multi-system, genetic disease affecting over 70,000 people worldwide. The underlying defect is a mutation in the CFTR gene. Dysfunctional CFTR protein results in abnormal anion movement across epithelial membranes in affected organs. There has been a paradigm shift in CF treatment over the last decade with the advent of CFTR modulation, treatments which target this underlying genetic defect and have the potential to change the course of CF clinical disease.Areas covered: Available CFTR modulators in current clinical practice are reviewed in this article, with a direct comparison and summary of relevant pivotal clinical trials. The approval of ivacaftor and subsequent development of lumacaftor and tezacaftor dual combinations represents an exciting development in CF management in recent years.Expert opinion: Tezacaftor/ivacaftor (tez/iva) appears to have a more favorable adverse event and drug-drug interaction profile than lumacaftor/ivacaftor. Tez/iva has been approved, alongside Phe508del, for a large number of 'residual function' CFTR mutations, with some based on response to in vitro culture. Dual therapy with tez/iva has paved the way for triple CFTR modulation currently in clinical trials with an ultimate view to provide modulation therapy to the majority of CF genotypes in the future.
journal_name
Expert Rev Respir Medjournal_title
Expert review of respiratory medicineauthors
Paterson SL,Barry PJ,Horsley ARdoi
10.1080/17476348.2020.1682998subject
Has Abstractpub_date
2020-01-01 00:00:00pages
15-30issue
1eissn
1747-6348issn
1747-6356journal_volume
14pub_type
杂志文章,评审abstract::The historic perspective that used to define chronic obstructive pulmonary disease has changed. As reviewed in this article, it is based on a better understanding of the underlying inflammatory airflow obstruction and a multidimensional classification, which mostly targets a subgroup called 'frequent exacerbators'. Cl...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/17476348.2013.814392
更新日期:2013-08-01 00:00:00
abstract:INTRODUCTION:Pulmonary mold infections are caused by ubiquitous organisms found in soil, water, and decaying vegetation, including Aspergillus spp., the Mucormycetes, hyaline molds, and dematiaceous (black) molds. Areas covered: These infections are often a challenge to diagnose and even more difficult to treat. Recent...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1080/17476348.2017.1325322
更新日期:2017-06-01 00:00:00
abstract::Lung transplantation is the only effective treatment for many advanced lung diseases. However, long-term survival after transplantation remains relatively poor, thus limiting the application of lung transplantation to patients with end-stage disease only. Acute and chronic rejection is the main reason for allograft fa...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章
doi:10.1586/17476348.1.2.271
更新日期:2007-10-01 00:00:00
abstract::Cigarette smoking in asthma is frequently present and is associated with worsening of symptoms, accelerated lung-function decline, a higher frequency of hospital admissions, a higher degree of asthma severity, poorer asthma control and reduced responsiveness to corticosteroids. Furthermore, it is associated with reduc...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/ers.10.85
更新日期:2011-02-01 00:00:00
abstract::Erdosteine is a multimechanism, mucolytic agent that decreases the sputum viscoelastic properties and bacterial adhesion to the cell membrane, endowed with bronchial anti-inflammatory activity and a scavenging effect on free oxidant radicals. Erdosteine is a prodrug and metabolite I is the active metabolite of erdoste...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章
doi:10.1586/17476348.1.3.307
更新日期:2007-12-01 00:00:00
abstract::Refractory breathlessness is one of the most common and devastating symptoms of advanced cardiorespiratory disease, both malignant and nonmalignant. In spite of increased interest in research in the last 20 years, there have been few significant advances in the palliation of this distressing condition. The most succes...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章
doi:10.1586/17476348.3.1.21
更新日期:2009-02-01 00:00:00
abstract:INTRODUCTION:The use of beta-blockers in chronic obstructive pulmonary disease (COPD) is controversial, primarily due to concerns that they may worsen lung function and attenuate bronchodilator response. Areas covered: This review summarizes the reasons for and against the use of beta-blockers in COPD by evaluating the...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1080/17476348.2018.1419869
更新日期:2018-02-01 00:00:00
abstract::The management of pulmonary hypertension has already incorporated the use of multiple therapies into routine practice. Available therapies act principally through vasodilatation of the pulmonary arterial circulation through well-delineated mechanisms, although right ventricular function determines function and prognos...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/ers.11.13
更新日期:2011-04-01 00:00:00
abstract::Premature infants are at substantial risk for a spectrum of morbidities that are gestational age dependent. Respiratory syncytial virus (RSV) infection is most common in the first two years of life with the highest burden in children aged <6 months. Preterm infants ≤35 weeks' gestation are handicapped by incomplete im...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/17476348.2015.1098536
更新日期:2015-01-01 00:00:00
abstract::Epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs) have significantly improved the survival of patients with advanced non-small cell lung cancer (NSCLC) harboring EGFR activating mutations. However, nearly all EGFR-mutant NSCLC tumors eventually acquire resistance to the currently used EGFR-TKIs a...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/17476348.2016.1164603
更新日期:2016-01-01 00:00:00
abstract:INTRODUCTION:Chronic obstructive pulmonary disease (COPD) patients experience the morning as the worst period of the day. Nevertheless, morning symptoms are not mentioned in COPD guidelines. Areas covered: Different topics on morning symptoms are covered in this review to underline their importance: occurrence, tools f...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1080/17476348.2017.1305894
更新日期:2017-04-01 00:00:00
abstract:INTRODUCTION:The treatment of lung cancer has radically changed over the last few years. The discovery of druggable oncogenic alterations and the introduction of immunotherapy have provided lung cancer patients with the possibility of more efficient and less toxic therapeutic alternatives than chemotherapy. In the case...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1080/17476348.2017.1326822
更新日期:2017-06-01 00:00:00
abstract:INTRODUCTION:Survival after lung transplantation lags behind outcomes of other solid organ transplants, and complications from lung transplant are the second most common cause of death in cystic fibrosis. Evolving surgical techniques, therapeutics, and perioperative management have improved short-term survival after lu...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1080/17476348.2018.1522254
更新日期:2018-11-01 00:00:00
abstract:INTRODUCTION:Lymphangioleiomyomatosis (LAM) is a destructive lung disease affecting primarily women. LAM is caused by inactivating mutations in the tuberous sclerosis complex (TSC) genes, resulting in hyperactivation of mechanistic/mammalian target of rapamycin complex 1 (mTORC1). Over the past five years, there have b...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1080/17476348.2018.1409622
更新日期:2018-02-01 00:00:00
abstract:INTRODUCTION:In sarcoidosis, muscle involvement is common, but mostly asymptomatic. Currently, little is known about respiratory muscle and diaphragm involvement and function in patients with sarcoidosis. Reduced inspiratory muscle strength and/or a reduced diaphragm function may contribute to exertional dyspnea, fatig...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1080/17476348.2018.1480940
更新日期:2018-07-01 00:00:00
abstract::Introduction: Idiopathic pulmonary fibrosis (IPF), the most common of the idiopathic interstitial pneumonias, is a disease with a poor prognosis, and a highly heterogeneous and unpredictable clinical course. While most patients experience a relatively slow clinical, functional and radiological deterioration, a signifi...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章
doi:10.1080/17476348.2020.1724096
更新日期:2020-04-01 00:00:00
abstract::Studies on cough have come a long way but many shortfalls still exist. These shortfalls can be attributed to: the lack of randomized controlled studies with a focus on cough; studies not using robust cough outcome measures, poor definition of target groups in studies and guidelines, the lack of safe and efficacious tr...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/ers.11.35
更新日期:2011-08-01 00:00:00
abstract::For the last 100 years, the tuberculin skin test (TST) has been the only diagnostic tool available for latent TB infection (LTBI) and no biomarker per se is available to diagnose the presence of LTBI. With the introduction of M. tuberculosis-specific IFN-gamma release assays (IGRAs), a new area of in vitro immunodiagn...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章
doi:10.1586/ers.09.31
更新日期:2009-08-01 00:00:00
abstract::Chronic respiratory disease is an increasingly prevalent cause of life-limiting illness. Care for patients with terminal respiratory diseases has historically focused on disease-modifying therapies, many of which have little impact on life expectancy. This exclusive focus can detract from attention to patient-physicia...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章
doi:10.1586/ers.09.47
更新日期:2009-12-01 00:00:00
abstract:INTRODUCTION:Pleural disease encompasses a large range of conditions, is a common presentation to the acute medical take and often requires comprehensive investigation and treatment. Ultrasound is well recognised as a useful investigative tool in pleural disease especially in the field of pleural effusion, pleural thic...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1080/17476348.2017.1300531
更新日期:2017-04-01 00:00:00
abstract::Since chronic obstructive pulmonary disease (COPD) has a progressive and major impact on health management, many aspects of this disorder, including development of effective and reliable biomarkers to monitor disease progression, are under intensive investigation. A huge amount of data, accumulated over the years, hav...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/ers.13.4
更新日期:2013-04-01 00:00:00
abstract:BACKGROUND:Approximately 14% of UK hospital in-patients receive supplemental oxygen therapy, only 57% have valid prescriptions. Oxygen must be optimally prescribed to ensure maximal therapeutic response whilst minimizing adverse outcomes (including fatality). This study investigates prescription compliance. METHODS:Al...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章
doi:10.1080/17476348.2021.1826316
更新日期:2020-10-14 00:00:00
abstract::The need for novel and more effective approaches to tobacco control is unquestionable. The electronic cigarette is a battery-powered electronic nicotine delivery system that looks very similar to a conventional cigarette and is capable of emulating smoking, but without the combustion products accountable for smoking's...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/ers.11.92
更新日期:2012-02-01 00:00:00
abstract::In 2007 the long-term outcome of lung cancer is still disappointing, with a 5-year survival rate not exceeding 15%. However, small but significant improvements in survival times have been achieved in the last decade. This progress has been achieved through the improvement of surgical techniques and following the intro...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章
doi:10.1586/17476348.1.1.99
更新日期:2007-08-01 00:00:00
abstract::Long-term oxygen therapy (LTOT) has been shown to reduce pulmonary hypertension and improve survival in patients with chronic obstructive pulmonary disease and resting hypoxemia (reduced arterial partial pressure of oxygen ≤55 mmHg). However, the benefit of its use for chronic pulmonary diseases other than chronic obs...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/ers.12.69
更新日期:2012-12-01 00:00:00
abstract::In many countries numbers of adults with cystic fibrosis (CF) exceed that of children, with median survival predicted to surpass 50 years. Increasing longevity is, in part, due to intensive therapies including eradication of early infection and suppressive therapies and pulmonary exacerbations. Initial infections with...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/17476348.2016.1162715
更新日期:2016-01-01 00:00:00
abstract::Chronic obstructive pulmonary disease is an inflammatory disorder characterized by airflow limitation. Its cardinal symptom is dyspnea, which develops gradually with even low levels of exercise. Bronchodilators (BDs) are the most effective drugs for relieving dyspnea. Two main types of BD are currently available, β-mi...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/ers.13.18
更新日期:2013-04-01 00:00:00
abstract::Asthma is often associated with various comorbidities. The most frequently reported asthma comorbid conditions include rhinitis, sinusitis, gastroesophageal reflux disease, obstructive sleep apnea, hormonal disorders and psychopathologies. These conditions may, first: share a common pathophysiological mechanism with a...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1586/ers.11.34
更新日期:2011-06-01 00:00:00
abstract::Non-small-cell lung cancer is the most common cause of cancer-related death worldwide. Surgery remains the cornerstone of treatment for localized, resectable lung cancer, although advanced stages are associated with a high risk of developing distant metastases. Large randomized trials have demonstrated an improvement ...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章
doi:10.1586/17476348.2.5.655
更新日期:2008-10-01 00:00:00
abstract:INTRODUCTION:A growing number of immunocompromised (IC) patients with acute hypoxemic respiratory failure (ARF) is admitted to the intensive care unit (ICU) worldwide. Areas covered: This review provides an overview of the current knowledge of the ways to prevent intubation in IC patients with ARF. Expert commentary: S...
journal_title:Expert review of respiratory medicine
pub_type: 杂志文章,评审
doi:10.1080/17476348.2018.1511430
更新日期:2018-10-01 00:00:00