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pulmonary fibrosis randomized controlled trial

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https://www.readbyqxmd.com/read/28430622/serious-adverse-events-of-cell-therapy-for-respiratory-diseases-a-systematic-review-and-meta-analysis
#1
REVIEW
Runzhen Zhao, Zhenlei Su, Jing Wu, Hong-Long Ji
BACKGROUND: Cell therapy holds the most promising for acute and chronic deleterious respiratory diseases. However, the safety and tolerance for lung disorders are controversy. METHODS: We undertook a systematic review and meta-analyses of all 23 clinical studies of cell therapy. The outcomes were odds ratio (OR), risk difference (RD), Peto OR, relative risk, and mean difference of serious adverse events. RESULTS: 342 systemic infusions and 57 bronchial instillations (204 recipients) of cells were analyzed for acute respiratory distress syndrome (ARDS), bronchopulmonary dysplasia, pulmonary arterial hypertension, silicosis, sarcoidosis, extensively drug-resistant tuberculosis, chronic obstructive pulmonary diseases (COPD), and idiopathic pulmonary fibrosis...
February 16, 2017: Oncotarget
https://www.readbyqxmd.com/read/28403943/plasma-metabolomics-in-adults-with-cystic-fibrosis-during-a-pulmonary-exacerbation-a-pilot-randomized-study-of-high-dose-vitamin-d3-administration
#2
RANDOMIZED CONTROLLED TRIAL
Jessica A Alvarez, Elizabeth Y Chong, Douglas I Walker, Joshua D Chandler, Ellen S Michalski, Ruth E Grossmann, Karan Uppal, Shuzhao Li, Jennifer K Frediani, Rabindra Tirouvanziam, ViLinh T Tran, Vin Tangpricha, Dean P Jones, Thomas R Ziegler
BACKGROUND: Cystic fibrosis (CF) is a chronic catabolic disease often requiring hospitalization for acute episodes of worsening pulmonary exacerbations. Limited data suggest that vitamin D may have beneficial clinical effects, but the impact of vitamin D on systemic metabolism in this setting is unknown. OBJECTIVE: We used high-resolution metabolomics (HRM) to assess the impact of baseline vitamin D status and high-dose vitamin D3 administration on systemic metabolism in adults with CF with an acute pulmonary exacerbation...
May 2017: Metabolism: Clinical and Experimental
https://www.readbyqxmd.com/read/28345369/pulmonary-fibrosis-part-ii-state-of-the-art-patient-management
#3
Keith C Meyer
While many pharmacologic therapies for the treatment of idiopathic pulmonary fibrosis (IPF) have been evaluated via randomized, placebo-controlled clinical trials (RCTs) conducted over the past two decades, most therapies have been shown to be ineffective or even potentially harmful. However, a number of recently completed RCTs have shown significant efficacy for pirfenidone and nintedanib for the treatment of IPF. Areas covered: This manuscript reviews recent advances in the management of IPF and other forms of fibrosing interstitial lung disease (ILD) with an emphasis on IPF...
May 2017: Expert Review of Respiratory Medicine
https://www.readbyqxmd.com/read/28319489/autogenic-drainage-in-children-with-cystic-fibrosis
#4
Lieselotte Corten, Brenda M Morrow
PURPOSE: Airway clearance is an essential part of the management of cystic fibrosis (CF) as it facilitates clearance of viscous pulmonary secretions. This review aimed to determine the effect of autogenic drainage (AD) and assisted autogenic drainage (AAD) compared with no, sham, or other types of airway clearance in children with CF. SUMMARY OF KEY POINTS: Two pediatric randomized cross-over trials were identified on the use of AD in children with CF; no studies were available on the use of AAD...
April 2017: Pediatric Physical Therapy
https://www.readbyqxmd.com/read/28287347/predicting-life-expectancy-for-pirfenidone-in-idiopathic-pulmonary-fibrosis
#5
Mark Fisher, Steven D Nathan, Christian Hill, Jade Marshall, Fred Dejonckheere, Per-Olof Thuresson, Toby M Maher
BACKGROUND: Conducting an adequately powered survival study in idiopathic pulmonary fibrosis (IPF) is challenging due to the rare nature of the disease and the need for extended follow-up. Consequently, registration trials of IPF treatments have not been designed to estimate long-term survival. OBJECTIVE: To predict life expectancy for patients with IPF receiving pirfenidone versus best supportive care (BSC) in a population that met the inclusion criteria of patients enrolled in the ASCEND and CAPACITY trials...
March 2017: Journal of Managed Care & Specialty Pharmacy
https://www.readbyqxmd.com/read/28287346/systematic-review-and-network-meta-analysis-of-idiopathic-pulmonary-fibrosis-treatments
#6
Kelly Fleetwood, Rachael McCool, Julie Glanville, Susan C Edwards, Sandro Gsteiger, Monica Daigl, Mark Fisher
BACKGROUND: The antifibrotics pirfenidone and nintedanib are both approved for the treatment of idiopathic pulmonary fibrosis (IPF) by regulatory agencies and are recommended by health technology assessment bodies. Other treatments such as N-acetylcysteine are used in clinical practice but have not received regulatory approval. No head-to-head trials have been conducted to directly compare the efficacy of these therapies in IPF. OBJECTIVE: To compare the efficacy of treatments for IPF...
March 2017: Journal of Managed Care & Specialty Pharmacy
https://www.readbyqxmd.com/read/28266906/pirfenidone-for-the-treatment-of-idiopathic-pulmonary-fibrosis
#7
Peter M George, Athol U Wells
Idiopathic pulmonary fibrosis (IPF) is a diffuse parenchymal lung disease with no cure. Up until recently, no treatment had been proven to alter its natural history as judged by rate of lung function decline. In 2014 however, the emergence of two novel anti-fibrotic agents, Pirfenidone and Nintedanib revolutionized the management of this condition. Both have demonstrated the ability to deliver a major reduction in the rate of chronic IPF progression. Areas Covered: This review article focuses on Pirfenidone - a pyridone derivative initially designed as an analgesic and anti-pyretic agent...
May 2017: Expert Review of Clinical Pharmacology
https://www.readbyqxmd.com/read/28258928/inhaled-dry-powder-mannitol-in-children-with-cystic-fibrosis-a-randomised-efficacy-and-safety-trial
#8
K De Boeck, E Haarman, J Hull, L C Lands, A Moeller, A Munck, J Riethmüller, H Tiddens, S Volpi, J Leadbetter, B Charlton, A Malfroot
INTRODUCTION: Inhaled mannitol has beneficial effects on lung function, mucociliary clearance, quality of life and sputum properties. This trial examined the efficacy of inhaled mannitol in children with cystic fibrosis (CF). METHODS: The efficacy of inhaled mannitol in children with CF aged 6-17years was assessed in a phase 2, randomised, placebo-controlled crossover study. Subjects were randomly assigned to mannitol 400mg every 12h or matching placebo for 8weeks, followed by an 8week washout and an 8week period with the alternate treatment...
February 28, 2017: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/28137504/etiology-and-treatment-of-cough-in-idiopathic-pulmonary-fibrosis
#9
REVIEW
Christine L Vigeland, Andrew H Hughes, Maureen R Horton
Idiopathic pulmonary fibrosis (IPF) is a progressive disease of dysregulated wound healing leading to unremitting scarring and loss of lung function. The predominant symptoms are dyspnea on exertion and a persistent dry cough. For patients with IPF, cough is more than just bothersome; it has a significant negative impact on quality of life and is a marker of disease severity and progression. The etiology of cough in IPF is unclear but may be due to architectural distortion of the lungs, increased sensitivity of the cough reflex, airway inflammation, or changes in mucus production and clearance...
February 2017: Respiratory Medicine
https://www.readbyqxmd.com/read/28125158/chest-physiotherapy-can-affect-the-lung-clearance-index-in-cystic-fibrosis-patients
#10
Joerg Grosse-Onnebrink, Uwe Mellies, Margarete Olivier, Claudius Werner, Florian Stehling
OBJECTIVES: The lung clearance index (LCI) is determined by multiple-breath washout lung function (MBW). It is increasingly used as an endpoint in clinical trials. Chest physiotherapy (CP) is part of routine cystic fibrosis (CF) care. Whether the LCI is useful in detecting short-term treatment effects of CP has not been sufficiently investigated. We assessed the short-term influence of CP with highly standardized high-frequency chest wall oscillation (HFCWO) on the LCI in CF patients...
May 2017: Pediatric Pulmonology
https://www.readbyqxmd.com/read/28114922/a-prospective-pilot-study-of-home-monitoring-in-adults-with-cystic-fibrosis-home-cf-protocol-for-a-randomised-controlled-trial
#11
Jocelyn Choyce, Karen L Shaw, Alice J Sitch, Hema Mistry, Joanna L Whitehouse, Edward F Nash
BACKGROUND: Home monitoring has the potential to detect early pulmonary exacerbations in people with cystic fibrosis (CF), with consequent improvements in health outcomes and healthcare associated costs. This study aims to assess the effects of home monitoring on hospital admissions, quality of life, antibiotic requirements, exacerbation frequency, lung function, nutritional outcomes, anxiety, depression, costs and health outcomes, as well as the qualitative effects on the patient experience...
January 23, 2017: BMC Pulmonary Medicine
https://www.readbyqxmd.com/read/28102546/ataluren-and-similar-compounds-specific-therapies-for-premature-termination-codon-class-i-mutations-for-cystic-fibrosis
#12
REVIEW
Aisha A Aslam, Colin Higgins, Ian P Sinha, Kevin W Southern
BACKGROUND: Cystic fibrosis is a common life-shortening genetic disorder in the Caucasian population (less common in other ethnic groups) caused by the mutation of a single gene that codes for the production of the cystic fibrosis transmembrane conductance regulator protein. This protein coordinates the transport of salt (and bicarbonate) across cell surfaces and the mutation most notably affects the airways. In the lungs of people with cystic fibrosis, defective protein results in a dehydrated surface liquid and compromised mucociliary clearance...
January 19, 2017: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/28011037/assessment-of-safety-and-efficacy-of-long-term-treatment-with-combination-lumacaftor-and-ivacaftor-therapy-in-patients-with-cystic-fibrosis-homozygous-for-the-f508del-cftr-mutation-progress-a-phase-3-extension-study
#13
Michael W Konstan, Edward F McKone, Richard B Moss, Gautham Marigowda, Simon Tian, David Waltz, Xiaohong Huang, Barry Lubarsky, Jaime Rubin, Stefanie J Millar, David J Pasta, Nicole Mayer-Hamblett, Christopher H Goss, Wayne Morgan, Gregory S Sawicki
BACKGROUND: The 24-week safety and efficacy of lumacaftor/ivacaftor combination therapy was shown in two randomised controlled trials (RCTs)-TRAFFIC and TRANSPORT-in patients with cystic fibrosis who were aged 12 years or older and homozygous for the F508del-CFTR mutation. We aimed to assess the long-term safety and efficacy of extended lumacaftor/ivacaftor therapy in this group of patients in PROGRESS, the long-term extension of TRAFFIC and TRANSPORT. METHODS: PROGRESS was a phase 3, parallel-group, multicentre, 96-week study of patients who completed TRAFFIC or TRANSPORT in 191 sites in 15 countries...
February 2017: Lancet Respiratory Medicine
https://www.readbyqxmd.com/read/28000919/antibiotic-treatment-for-nontuberculous-mycobacteria-lung-infection-in-people-with-cystic-fibrosis
#14
REVIEW
Valerie Waters, Felix Ratjen
BACKGROUND: Nontuberculous mycobacteria are mycobacteria, other than those in the Mycobacterium tuberculosis complex, and are commonly found in the environment. Nontuberculous mycobacteria species (most commonly Mycobacterium avium complex and Mycobacterium abscessus) are isolated from the respiratory tract of approximately 5% to 40% of individuals with cystic fibrosis; they can cause lung disease in people with cystic fibrosis leading to more a rapid decline in lung function and even death in certain circumstances...
December 19, 2016: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/27939076/efficacy-of-simtuzumab-versus-placebo-in-patients-with-idiopathic-pulmonary-fibrosis-a-randomised-double-blind-controlled-phase-2-trial
#15
Ganesh Raghu, Kevin K Brown, Harold R Collard, Vincent Cottin, Kevin F Gibson, Robert J Kaner, David J Lederer, Fernando J Martinez, Paul W Noble, Jin Woo Song, Athol U Wells, Timothy P M Whelan, Wim Wuyts, Emmanuel Moreau, Scott D Patterson, Victoria Smith, Selina Bayly, Jason W Chien, Qi Gong, Jenny J Zhang, Thomas G O'Riordan
BACKGROUND: Lysyl oxidase-like 2 (LOXL2) catalyses collagen cross-linking and is implicated in the pathogenesis of idiopathic pulmonary fibrosis (IPF). The aim of this study was to investigate the efficacy and safety of simtuzumab, a monoclonal antibody against LOXL2, in patients with IPF. METHODS: In this randomised, double-blind, phase 2 trial, we recruited patients aged 45-85 years with definite IPF diagnosed prior to 3 years of screening from 183 hospitals and respiratory clinics in 14 countries...
January 2017: Lancet Respiratory Medicine
https://www.readbyqxmd.com/read/27795804/hepatic-sarcoidosis-a-case-series
#16
Rym Ennaifer, Shema Ayadi, Hayfa Romdhane, Myriam Cheikh, Houda Ben Nejma, Wassila Bougassas, Najet Bel Hadj
Sarcoidosis is a systemic non caseous granulomas disease. Liver is a common location but usually asymptomatic. Evidence based guidelines for this location treatment is lacking and the effect of corticosteroids may be inadequate. The aim of our study was to describe the clinical, biochemical, radiological and therapeutic features of seven patients with systemic sarcoidosis and liver involvement. A retrospective and descriptive monocentric study, over 3 years, including seven patients with systemic sarcoidosis and liver involvement...
2016: Pan African Medical Journal
https://www.readbyqxmd.com/read/27764523/benzodiazepines-for-the-relief-of-breathlessness-in-advanced-malignant-and-non-malignant-diseases-in-adults
#17
REVIEW
Steffen T Simon, Irene J Higginson, Sara Booth, Richard Harding, Vera Weingärtner, Claudia Bausewein
BACKGROUND: This is an updated version of the original Cochrane review published in Issue 1, 2010, on 'Benzodiazepines for the relief of breathlessness in advanced malignant and non-malignant diseases in adults'. Breathlessness is one of the most common symptoms experienced in the advanced stages of malignant and non-malignant disease. Benzodiazepines are widely used for the relief of breathlessness in advanced diseases and are regularly recommended in the literature. At the time of the previously published Cochrane review, there was no evidence for a beneficial effect of benzodiazepines for the relief of breathlessness in people with advanced cancer and chronic obstructive pulmonary disease (COPD)...
October 20, 2016: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/27576473/probiotic-supplementation-in-children-with-cystic-fibrosis-a-systematic-review
#18
REVIEW
Anitha Ananthan, Haribalakrishna Balasubramanian, Shripada Rao, Sanjay Patole
UNLABELLED: Probiotics may benefit in cystic fibrosis (CF) as gut dysbiosis is associated with gastrointestinal symptoms and exacerbation of respiratory symptoms in CF. We conducted a systematic review of randomized controlled trials (RCTs) and non-RCTs of probiotic supplementation in children with CF, using the Cochrane methodology, preferred reporting items for systematic reviews (PRISMA) statement, and meta-analysis of observational studies in epidemiology (MOOSE) guidelines. Primary outcomes were pulmonary exacerbations, duration of hospitalization and antibiotics, and all-cause mortality...
October 2016: European Journal of Pediatrics
https://www.readbyqxmd.com/read/27450411/study-protocol-rationale-and-recruitment-in-a-european-multi-centre-randomized-controlled-trial-to-determine-the-efficacy-and-safety-of-azithromycin-maintenance-therapy-for-6%C3%A2-months-in-primary-ciliary-dyskinesia
#19
RANDOMIZED CONTROLLED TRIAL
Helene E Kobbernagel, Frederik F Buchvald, Eric G Haarman, Carmen Casaulta, Samuel A Collins, Claire Hogg, Claudia E Kuehni, Jane S Lucas, Heymut Omran, Alexandra L Quittner, Claudius Werner, Kim G Nielsen
BACKGROUND: Clinical management of primary ciliary dyskinesia (PCD) respiratory disease is currently based on improving mucociliary clearance and controlling respiratory infections, through the administration of antibiotics. Treatment practices in PCD are largely extrapolated from more common chronic respiratory disorders, particularly cystic fibrosis, but no randomized controlled trials (RCT) have ever evaluated efficacy and safety of any pharmacotherapeutics used in the treatment of PCD...
July 22, 2016: BMC Pulmonary Medicine
https://www.readbyqxmd.com/read/27415821/antibiotic-treatment-for-stenotrophomonas-maltophilia-in-people-with-cystic-fibrosis
#20
REVIEW
Reshma Amin, Valerie Waters
BACKGROUND: Stenotrophomonas maltophilia is one of the most common emerging multi-drug resistant organisms found in the lungs of people with cystic fibrosis and its prevalence is increasing. Chronic infection with Stenotrophomonas maltophilia has recently been shown to be an independent predictor of pulmonary exacerbation requiring hospitalization and antibiotics. However, the role of antibiotic treatment of Stenotrophomonas maltophilia infection in people with cystic fibrosis is still unclear...
2016: Cochrane Database of Systematic Reviews
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