Author Interviews, CHEST, Pulmonary Disease, Sleep Disorders / 12.12.2022

MedicalResearch.com Interview with: Pamela L. Lutsey, PhD, MPH Minneapolis, Minnesota Professor, Division of Epidemiology and Community Health School of Public Health University of Minnesota MedicalResearch.com: What is the background for this study? Response: Emerging evidence suggests that patients with OSA may be more susceptible to developing pneumonia. Mechanistically, OSA causes upper airway sensory dysfunction and excessive microaspirations, which can result in significant increases in bacterial organisms in the airway leading to upper airway and laryngeal inflammation. Systemically, healthy sleep is believed to play an important role in the body’s inflammation control and immune system regulation. Despite this evidence, few studies have prospectively evaluated whether individuals with OSA are at elevated risk of being hospitalized with pneumonia, respiratory infection, or any infection (more…)
Author Interviews, Pulmonary Disease, Surgical Research / 13.09.2022

MedicalResearch.com Interview with: Ms. Sara Buttery Research Physiotherapist at Royal Brompton & Harefield NHS Foundation Trust University of the West of England London, England, UK MedicalResearch.com: What is the background for this study? Would you describe the BVLR technique? Response: The CELEB trial is a multicentre randomised controlled trial that was carried out at five centres across the UK, with the objective of investigating if Lung volume reduction surgery (LVRS) is significantly superior to Bronchoscopic lung volume reduction (BLVR) in people who are suitable for both procedures. BLVR is a minimally invasive method of lung volume reduction whereby a fibreoptic camera is passed through the mouth, rather than by an incision in the chest wall as is the case with LVRS. The CELEB trial compared endobronchial valves (EBVs) as a type of BLVR, to LVRS. EBVs are designed to prevent airflow into the treated lobe, but allow air and mucus to exit. EBV treatment can be carried out under general anaesthetic or sedation. The primary outcome for the CELEB trial was the iBODE index score at 12 months post procedure, as a measure of disease severity. (more…)
Author Interviews, NEJM, Pulmonary Disease, Tobacco Research, University of Michigan / 04.09.2022

MedicalResearch.com Interview with: Meilan K Han MD, MS Henry Sewall Professor of Medicine Professor of Internal Medicine and Section Head Division of Pulmonary and Critical Care Medicine, Medical School University of Michigan MedicalResearch.com: What is the background for this study? What are the main findings? Response: In the NIH sponsored SPIROMICS study we demonstrated that symptomatic, tobacco exposed individuals have frequent exacerbations. Many of these individuals are treated with the same inhaled medications that have shown benefit in COPD, but we don’t have any evidence basis for this practice. (more…)
Author Interviews, Boehringer Ingelheim, NEJM, Pulmonary Disease / 15.05.2022

MedicalResearch.com Interview with: Professor Luca Richeldi MD PhD Chair and Head, Division of Pulmonary Medicine Gemelli University Hospital - IRCCS Catholic University of the Sacred Heart Rome MedicalResearch.com:  What is the background for this study?  Would you briefly explain the condition of Idiopathic Pulmonary Fibrosis? Response: As you may know, Idiopathic pulmonary fibrosis (IPF) is a progressive, irreversible lung disease with high mortality. IPF is one of the more common forms of progressive fibrosing interstitial lung diseases and its symptoms of IPF include breathlessness during activity, a dry and persistent cough, chest discomfort, fatigue and weakness. IPF is considered a “rare” disease, but it affects more than 3 million people worldwide. Currently, there are two approved antifibrotic drugs that slow, but do not stop, the progression of fibrosis. Therefore, there is a need for additional treatments that can be used alone or with existing antifibrotic therapies. Pre-clinical research indicated that phosphodiesterase 4 (PDE4) inhibition is associated with anti-inflammatory and antifibrotic effects that may be beneficial in patients with idiopathic pulmonary fibrosis. In this Phase 2, double-blind, placebo-controlled trial, we investigated the efficacy and safety of BI 1015550, an oral preferential inhibitor of the PDE4B subtype, in patients with IPF. Patients were randomly assigned in a 2:1 ratio to receive BI 1015550 at a dose of 18 mg twice daily or placebo. (more…)
Asthma, Author Interviews, NEJM, Pediatrics, Pulmonary Disease, Vanderbilt / 08.12.2021

MedicalResearch.com Interview with: Leonard B. Bacharier, MD Janie Robinson and John Moore Lee Chair in Pediatrics Professor of Pediatrics Director - Center for Pediatric Asthma Research Scientific Director - Center for Clinical and Translational Research Section Chief - Pediatric Allergy and Immunology Division of Allergy, Immunology and Pulmonary Medicine Monroe Carell Jr. Children’s Hospital at Vanderbilt University Medical Center  MedicalResearch.com: What is the background for this study?  Is Dupilumab used for other atopic conditions, ie eczema/atopic dermatitis?   Response: Many children with moderate-severe asthma continue to experience asthma exacerbations and poor asthma control despite use of controller therapies.  Dupilumab has been shown to reduce asthma exacerbations in adolescents and adults, as well as to improve atopic dermatitis in children and adults. (more…)
Author Interviews, Blood Pressure - Hypertension, Pulmonary Disease, University of Pittsburgh / 22.10.2021

MedicalResearch.com Interview with: Stephen Chan, M.D., Ph.D. Professor of Medicine Director of the Vascular Medicine Institute at Pitt and UPMC MedicalResearch.com: What is the background for this study? Response: Pulmonary hypertension (PH) is a type of high blood pressure that occurs in the vessels that transport blood from the heart to the lungs. As the disease progresses and the heart must strain harder against these high pressures, it can lead to heart failure, multi-organ dysfunction and death. PH affects people of all ages but hits young women more often than men. Pulmonary hypertension is an example of a rare disease where there is an unmet need for new treatments, given its devastating consequences. Repurposing drugs that are already in use for other purposes can dramatically cut down the time and cost of developing treatments for rare diseases like PH. But a pipeline to predict and test for drugs in this way for PH and other rare diseases has not been described. (more…)
Asthma, Author Interviews, Brigham & Women's - Harvard, Circadian Rhythm, Occupational Health, PNAS, Pulmonary Disease, Sleep Disorders / 11.09.2021

MedicalResearch.com Interview with: Frank Scheer, PhD, MSc Professor and Director of the Medical Chronobiology Program Division of Sleep and Circadian Disorders Brigham and Women’s Hospital Steve Shea, PhD Professor and Director Oregon Institute of Occupational Health Sciences MedicalResearch.com: What is the background for this study? Response: For hundreds of years, people have observed that asthma severity often worsens in the nighttime. As many as 75 percent of people with asthma—20 million people in the U.S.—report experiencing worsening asthma severity at night. One longstanding question has been to what degree the body’s internal circadian clock—as opposed to behaviors, such as sleep and physical activities—contributes to worsening of asthma severity. Our research used long term intensive monitoring throughout two circadian protocols in dim light and without time cues to carefully isolate the influence of the circadian system from the other factors that are behavioral and environmental, including sleep. (more…)
Author Interviews, COVID -19 Coronavirus, Critical Care - Intensive Care - ICUs, Lancet, Pulmonary Disease / 26.08.2021

MedicalResearch.com Interview with: Jie Li, PhD, RRT, RRT-ACCS, RRT-NPS, FAARC Department of Cardiopulmonary Sciences Division of Respiratory Care Rush University, Chicago MedicalResearch.com: What is the background for this study? Response: Prone positioning has been shown to improve oxygenation and reduce mortality in intubated patients with acute respiratory distress syndrome (ARDS), as placing patients on their stomachs can help open alveoli and reduce ventilation to perfusion mismatch. At early pandemic, clinicians tried prone positioning for non-intubated patients with COVID-19 and found improvement in oxygenation. However, the evidence for patient outcomes such as intubation or mortality is still lacking. Thus we organized this international, multicenter, randomized controlled meta-trial, with 41 hospitals in 6 countries participated. (more…)
Author Interviews, Gastrointestinal Disease, Imperial College, Probiotics, Pulmonary Disease, Weight Research / 16.05.2021

MedicalResearch.com Interview with: Benjamin Mullish PhD NIHR Academic Clinical Lecturer Department of Metabolism Digestion and Reproduction Imperial College MedicalResearch.com: What is the background for this study? Which probiotic did you use and why?  Response: We recently reported the results of a randomized, double-blind placebo-controlled trial in which participants who were overweight or with obesity (aged between 30-65 years of age) were randomized to receive probiotics or placebo for six months.  The primary focus was on weight loss and metabolism.  The probiotic used was Lab4P, containing three different strains of Bifidobacteria and two of Lactobacilli, which have shown to be safe and efficacious for use in rodent models and earlier clinical studies. Of note, probiotics have also been shown to have other beneficial effects upon human health.  Previous studies have suggested that they may have a role in preventing upper respiratory tract infections (URTIs) in healthy people and children; however, this has not been explored in older people or overweight/ people with obesity, even though such groups have higher rates of URTIs. We looked back at our trial, and reviewed symptom diaries completed by participants daily during the study.  We were looking at recorded symptoms most consistent with upper respiratory tract symptoms (including cough, wheezing and headache), and explored if rates of these were different between those participants taking probiotics compared to placebo over the six month course of the study.   (more…)
Alzheimer's - Dementia, Author Interviews, Cognitive Issues, Obstructive Sleep Apnea, Pulmonary Disease, University of Michigan / 16.04.2021

MedicalResearch.com Interview with: Galit Levi Dunietz  MPH, PhD Assistant Professor

Tiffany Braley, MD, MS Associate Professor

University of Michigan, Medical School Department of Neurology Department of Nutritional Sciences Ann Arbor, MI 48109-5845 MedicalResearch.com: What is the background for this study? Response: Dementia is a public health crisis that affects more than 6 million Americans.  As no treatments to effectively reverse dementia are currently available, interest has shifted toward modifiable risk factors for dementia, which may offer a critical window for prevention or intervention. Recent research suggests that obstructive sleep apnea (OSA) is a common, yet undiagnosed, risk factor for cognition impairment in older adults. However, few studies have examined whether treatment of OSA with positive airway pressure (PAP) therapy could protect those with OSA against developing dementia, says principal investigator, Dr. Tiffany Braley, MD, MS, Associate Professor of Neurology from the University of Michigan. To address this gap, Dr. Braley and Dr. Galit Levi Dunietz, PhD, MPH, Assistant Professor and sleep epidemiologist, examined associations between PAP therapy use and 3-year incidence of Alzheimer’s disease, mild cognitive impairment (MCI), or other forms of dementia (DNOS, “dementia not otherwise specified”). (more…)
Author Interviews, COVID -19 Coronavirus, JAMA, Pediatrics, Pulmonary Disease / 04.03.2021

MedicalResearch.com Interview with: Dott.ssa Silvia Bloise MD Prof. Riccardo Lubrano MD PhD Pediatric and Neonatology Unit Maternal and Child Department Sapienza University of Rome Rome Italy MedicalResearch.com: What is the background for this study? Response: Pending new evidence, the universal facial masking, with other preventive measures remain the only strategies to limit the spread of SARS-CoV-2 infection. The use of face mask is particularly debated in the children, especially in younger children. Therefore, we wanted to test whether their in children was associated with episodes of desaturation or respiratory distress. (more…)
Author Interviews, NIH, Pulmonary Disease / 09.02.2021

MedicalResearch.com Interview with: Stavros Garantziotis MD Division of Intramural Research National Institute of Environmental Health Sciences Research Triangle Park, NC MedicalResearch.com: What is the background for this study? How does hyaluronan differ from other medications for COPD? Is it used for other medical conditions? Response: Hyaluronan is a natural sugar found in the human body, including in the lung. We have found that when the lungs are exposed to pollution, this sugar breaks down, and the breakdown fragments cause inflammation in the lung. We also found that if we give back the natural form of hyaluronan, it protects the lung from inflammation. Patients suffering from COPD also have a lot of hyaluronan breakdown in their lungs. We therefore reasoned, that giving them back the natural form of hyaluronan, as an inhalation treatment, would help them reduce inflammation. We tested this, as a first step, in the treatment acute inflammation of the lungs in COPD patients who are suffering an exacerbation of their disease. Hyaluronan is different from existing medications in that it is a natural product of the body. It is used in Europe for conditions like cystic fibrosis, and after sinus surgery to humidify the airways. Because it is given by inhalation, it acts locally in the lungs.  (more…)
Author Interviews, COVID -19 Coronavirus, Critical Care - Intensive Care - ICUs, Pulmonary Disease / 15.01.2021

MedicalResearch.com Interview with: Dr Andrew Conway Morris Wellcome Trust Clinical Research Career Development Fellow University of Cambridge Hon Consultant in Intensive Care Medicine Addenbrookes Hospital, Cambridge MedicalResearch.com: What is the background for this study? Response: Patients with COVID-19 frequently need to come to the intensive care unit (ICU), where we use mechanical ventilation to support their lungs as they get over the intense inflammation caused by the virus.  During the first wave of the virus we noted that a lot of our patients appeared to be developing secondary infections (infections they didn’t have when they came into the ICU). We therefore rolled out a rapid diagnostic test for these secondary bacterial infections that we had developed previously, and this study reports the use of this diagnostic and also describes the types of bacteria seen.  To see if the increase in secondary infections was due to COVID specifically, we compared them to patients who were managed in the same ICU but who did not have COVID. (more…)
Author Interviews, COVID -19 Coronavirus, Pulmonary Disease / 12.01.2021

MedicalResearch.com Interview with: Dr. Liam Townsend, PhD Department of Infectious Diseases St. James's Hospital and Department of Clinical Medicine Trinity Translational Medicine Institute Trinity College, Dublin, Ireland  MedicalResearch.com: What is the background for this study? Response: Much is known about the clinical characteristics and pathological features of acute SARS-CoV-2 infection, but there is relatively little known about post-COVID recovery. This has come under increasing scrutiny in light of reports that patients suffer persistent symptoms beyond resolution of initial infection, known as long COVID. We set out to assess patients in our post-COVID clinic for ongoing ill-health, with particular focus on fatigue and breathlessness. Given that COVID-19 primarily affects the respiratory system, we also evaluated respiratory recovery. Patients underwent chest radiography and six-minute-walk testing, as well as routine blood tests including inflammatory markers and D-dimers. We included both patients who were admitted during their acute infection as well as those managed in the community in order to capture the full spectrum of disease. (more…)
Aging, Author Interviews, COVID -19 Coronavirus, Pulmonary Disease / 12.01.2021

MedicalResearch.com Interview with: Dr. Maria A. Blasco, PhD Director of the Spanish National Cancer Research Centre Head of the Telomeres and Telomerase Group – CNIO  MedicalResearch.com: What is the background for this study? Response: In my group we have previously described that telomere dysfunction in alveolar type II (ATII) cells in the lung is sufficient to induce pulmonary fibrosis in mice, thus demonstrating that these cells, which have a role in lung regeneration, are at the origin of the disease (Povedano et al., Cell Reports, 2015). Indeed, we further demonstrated that telomere elongation in these cells by using a gene therapy strategy based on telomerase activation, was sufficient to stop the progression of pulmonary fibrosis induced by short telomeres in mice (Povedano, eLife, 2018). (more…)
Author Interviews, JAMA, Pulmonary Disease / 10.12.2020

MedicalResearch.com Interview with: Andrew M. Wilson, MD Clinical Senior Lecturer in Respiratory Health University of East Anglia Norwich and Honorary Consultant Physician in Respiratory Medicine Norfolk and Norwich University MedicalResearch.com: What is the background for this study? What are the main findings? Response: Idiopathic pulmonary fibrosis has a poor prognosis and limited treatment options. Clinical trial evidence suggested a survival benefit for people taking co-trimoxazole and microbiological data suggested that infection was implicated in prognosis. However this large multicentre study did not show that co-trimoxazole had an beneficial effect in terms of time to all-cause mortality, hospitalisation or lung transplant in people with moderate and severe idiopathic pulmonary fibrosis (more…)
AstraZeneca, Author Interviews, Pulmonary Disease / 22.08.2020

MedicalResearch.com Interview with: Frank Trudo, MD MBA Vice President, US Medical Affairs Respiratory & Immunology AstraZeneca  MedicalResearch.com: What is the background for this study? Response: ETHOS was a randomized, double-blinded, multi-center, parallel-group, 52-week trial to assess the efficacy and safety of PT010 in symptomatic patients with moderate to very severe COPD and a history of exacerbation(s) in the previous year. A subset of patients participated in the 4-hour pulmonary function test (PFT) sub-study, with the following primary endpoints: change from baseline in morning pre-dose trough FEV1 at Week 24 at (both doses of budesonide/glycopyrrolate/formoterol fumarate MDI versus glycopyrrolate/formoterol fumarate MDI), and FEV1 area under the curve from 0-4 hours at Week 24 (both doses of budesonide/glycopyrrolate/formoterol fumarate MDI vs budesonide/formoterol fumarate MDI).  (more…)
AstraZeneca, Author Interviews, NEJM, Pulmonary Disease / 29.06.2020

MedicalResearch.com Interview with: Frank Trudo, MD MBA Vice President, US Medical Affairs Respiratory & Immunology AstraZeneca MedicalResearch.com: What is the background for this study? Response: ETHOS is a randomized, double-blinded, multi-center, parallel-group, 52-week trial to assess the efficacy and safety of PT010 in symptomatic patients with moderate to very severe COPD and a history of exacerbation(s) in the previous year. Outcomes in the ETHOS trial included, as a primary endpoint, the rate of moderate or severe exacerbations. MedicalResearch.com: How does PT010 differ from other treatments for COPD?
  • Highly competitive: PT010’s Phase III clinical trial program demonstrates it has a highly competitive clinical profile in decreasing moderate or severe exacerbations. Severe exacerbations were defined as exacerbations leading to hospitalization or death.
  • All-cause mortality: In a secondary endpoint, PT010 showed a 46% reduction in the risk of all-cause mortality compared with glycopyrronium/formoterol fumarate. The data from ETHOS show that reducing risk of all-cause mortality is achievable for patients with this progressive disease and could transform treatment goals in COPD.
  • Two potential dose options: This is also the first time we have seen the benefit of closed triple-combination therapy at two ICS doses, which could transform care by allowing physicians to select the optimal dosing option for individual patients. 
(more…)
Author Interviews, COVID -19 Coronavirus, Infections, Pulmonary Disease / 08.05.2020

MedicalResearch.com Interview with: Jehan Alladina MD Massachusetts General Hospital Pulmonary and Critical Care Medicine Boston, Massachusetts MedicalResearch.com: What is the background for this study? Response: During the pandemic, clinicians around the world have shared anecdotal experiences to help inform care of patients with COVID-19. However, these anecdotes and observations, without careful analysis, can bias clinicians; many clinicians have even recommended experimental therapies based on this information alone. To that end, the goal of our study was to rigorously examine the respiratory failure experienced by critically ill patients with COVID-19 and understand their response to the standards of care for respiratory failure. (more…)
Author Interviews, CDC, NEJM, Pulmonary Disease / 23.04.2020

MedicalResearch.com Interview with: Angela K. Werner, PhD, MPH Environmental Public Health Tracking Program National Center for Environmental Health CDC MedicalResearch.com: What is the background for this study? Response: In August 2019, emergency department (ED) visits related to e-cigarette, or vaping, product-associated lung injury (EVALI) increased sharply, followed by a peak in September. This was followed by a gradual but persistent decline in the number of cases. As of February 18, 2020 (CDC’s latest and final published update), there were a total of 2,807 hospitalized EVALI cases or deaths reported to CDC from all 50 states, the District of Columbia, and two U.S. territories (Puerto Rico and U.S. Virgin Islands). Sixty-eight deaths were confirmed in 29 states and the District of Columbia. Although clinical presentations and outcomes of EVALI patients have been reported, data on patients who died are more limited. This article fills a gap by reporting on a national study assessing detailed demographic, substance use, and clinical characteristics of EVALI patients who have died, and comparing them to the characteristics of EVALI patients who survived. (more…)
Author Interviews, Cannabis, JAMA, Pulmonary Disease / 22.04.2020

MedicalResearch.com Interview with: Alex Hollingsworth PhD Assistant Professor O'Neill School of Public and Environmental Affairs Indiana University MedicalResearch.com: What is the background for this study? What are the main findings? Response: I've been working with Coady Wing and Ashley Bradford on a few different studies of the effects of recreational marijuana laws on drug and alcohol use. Soon after EVALI became a major issue, the prevailing theory from the CDC and others was that EVALI was caused by the use of vitamin E acetate in illegal THC vaping products. Our group read about this and we thought about some of the things that often happen in black markets for illegal drugs. For instance, during the alcohol prohibition era, bootleg alcohol producers often made and sold alcohol products that were not that safe to drink. In more recent years, there are cases where black market sellers of illegal drugs like heroin try to increase profit margins by adding other substances, which can be harmful. We thought that maybe something like that could be happening in EVALI. Perhaps people in states where recreational marijuana is legal tended to purchase marijuana products from the legal market and the legal market was not selling any marijuana vaping products that included vitamin E acetate. (more…)
Author Interviews, COVID -19 Coronavirus, Pulmonary Disease / 04.04.2020

MedicalResearch.com Interview with: Aurika Savickaite RN Adult Gerontology Acute Care Nurse Practitioner Bulletproof Coach University of Chicago Medicine MedicalResearch.com: Would you briefly explain what is meant by helmet-based ventilation? How does it work?   Response: For patients in respiratory failure, noninvasive positive pressure ventilation (NIPPV) is usually delivered through a nasal mask or facemask. Many patients develop pain, discomfort – even claustrophobia -- from using NIPPV systems.  The transparent helmet was developed to improve the tolerance of noninvasive ventilation. It allows the patient to see, read, speak and drink without interrupting noninvasive positive-pressure ventilation (NPPV). The helmet has a sealed connection and a soft collar that adheres to the neck which helps prevent the air leaks that are very common with nasal- or face masks.  High positive end-expiratory pressure (PEEP) is vital in treating patients in respiratory failure and thanks to helmets “none to minimum air leak” system, PEEP can be set high (up to 25). NIPPV via a nasal- or full-face mask typically begins to show air leaks when the required pressure exceeds 15-20cm H2O. (more…)
Author Interviews, Environmental Risks, Occupational Health, Pulmonary Disease / 24.10.2019

MedicalResearch.com Interview with: Orianne Dumas, PhD INSERM Aging and Chronic Diseases, Epidemiological and Public Health Approaches,Villejuif, University de Versailles St-Quentin-en-Yvelines, Montigny le Bretonneux France  MedicalResearch.com: What is the background for this study? Response: Exposure to cleaning products and disinfectants is common at work and at home and remains more frequent among women. Exposure levels are particularly high in the health care industry. The respiratory health risks associated with exposure to cleaning products and disinfectants are increasingly recognized. Although investigators have primarily focused on asthma, the irritant properties of many chemicals contained in disinfectants support the study of a broader range of respiratory effects, such as chronic obstructive pulmonary disease (COPD). (more…)
Author Interviews, Heart Disease, Pharmaceutical Companies, Pulmonary Disease / 21.10.2019

MedicalResearch.com Interview with: Olivier Sitbon, MD, PhD Université Paris–Sud  MedicalResearch.com: What is the background for this study? How does this treatment competition differ from other treatments for PAH?  Response:  Pulmonary arterial hypertension (PAH) is a complex, progressive and potentially fatal disease with no cure. Over the past decades, advances in understanding the pathophysiology of PAH have led to major prognostic improvement and developments of new treatment guidelines and therapies. Current treatment guidelines recommend initial combination therapy for these patients to target multiple PAH-associated pathways in parallel. OPTIMA was a prospective, multicenter, single-arm, open-label, Phase IV trial designed to evaluate the efficacy, safety and tolerability of initial oral combination therapy with macitentan and tadalafil in patients with newly diagnosed PAH. Treatment with macitentan 10 mg once-daily and tadalafil 20 mg once-daily was initiated on the same day. After 8±3 days, tadalafil dose was increased to 40 mg once-daily. Safety and tolerability findings were consistent with previous clinical trials that supported the approval and use of macitentan 10 mg once-daily. Efficacy outcomes were assessed at Week 16 and safety continued to be monitored until study closure. The results from the OPTIMA analysis suggest that initial treatment with macitentan in combination with tadalafil is associated with hemodynamic improvement in newly diagnosed patients with pulmonary arterial hypertension (more…)
Author Interviews, Pulmonary Disease / 13.10.2019

MedicalResearch.com Interview with: Pierre-Régis Burgel MD, PhD Professor of Respiratory Medicine French National Reference Center for Cystic Fibrosis (coordinator) Cochin Hospital and Paris Descartes University Paris, France MedicalResearch.com: What is the background for this study? How does lumacaftor-ivacaftor differ from other treatments for CF?  Response: Cystic fibrosis (CF) is a genetic disease caused by mutations in the gene encoding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which acts as a chloride and bicarbonate ion channel across many epithelia. Defective ion transport leads to multiple organ dysfunction, but airway involvement (related to mucus plugging and infection) and malnutrition are among the most important prognostic factors in patients with CF. Over the past decades, symptomatic treatment, including inhaled and systemic antibiotics, nutritional support, pancreatic enzyme replacement, and specialized center care organization have led to major prognostic improvement. More recently, mutation-specific small molecules targeting defective CFTR have been shown to partly restore ion transport in epithelia, which translated into clinical benefits. Phe508del is the most common CFTR mutation with approximately 70% of patients with cystic fibrosis carrying one Phe508del mutation and 40-50% of patients being homozygous for this mutation. Safety and efficacy of lumacaftor-ivacaftor, which partially restores CFTR function, have been reported in phase 3 clinical trials in patients 12 years of age or older who had CF and were homozygous for the Phe508del. Improvement in lung function, reduction in pulmonary exacerbations and a trend towards an increase in body mass index (BMI) led to its approval by the Food and Drug Administration in February 2015 and by the European Medicines Agency in November 2015. However, the magnitude of effect on percent predicted forced expiratory volume in 1 sec (ppFEV1), the small improvement in nutritional status and the limited use of concomitant treatment for reducing exacerbations have cast doubt on the clinical benefits associated with lumacaftor-ivacaftor, which has not been approved in several countries. The present study is a multicenter (n=47 centers) observational post-marketing study aimed at evaluating the effects of lumacaftor-ivacaftor treatment in a real-life setting in France. All patients who initiated lumacaftor-ivacaftor in 2016 in the French cystic fibrosis reference network, which comprises 47 pediatric and/or adult centers, was performed. Our goal was to examine its safety and effectiveness over the first year of treatment in a large, unselected, population of adolescents (≥12 years) and adults (≥18 years) with CF and Phe508del homozygous mutations.  (more…)
Author Interviews, Critical Care - Intensive Care - ICUs, End of Life Care, Primary Care, Pulmonary Disease, University of Pennsylvania / 07.10.2019

MedicalResearch.com Interview with: Gary Weissman, MD, MSHP Assistant Professor of Medicine Pulmonary, Allergy, and Critical Care Division Palliative and Advanced Illness Research (PAIR) Center University of Pennsylvania Perelman School of Medicine  MedicalResearch.com: What is the background for this study? Response: There are millions of hospitalizations every year in the United States (US) that include a stay in an intensive care unit (ICU). Such ICU stays put strain on health system resources, may be unwanted by patients, and are costly to society. As the population of the US gets older and more medically complex, some have argued that we need more ICU beds and a larger ICU workforce to keep pace. We hypothesized that some proportion of these ICU admissions could be prevented with early and appropriate outpatient care. Such a strategy would alleviate some of the strains and costs associated with ICU stays. If an appreciable proportion of ICU stays were preventable in this way, it would strengthen support for an alternative population-health based framework instead of further investments in the ICU delivery infrastructure.  (more…)
Author Interviews, Boehringer Ingelheim, Pulmonary Disease / 01.10.2019

MedicalResearch.com Interview with: Dr. Donald ZozDonald Zoz, M.D. Director, Clinical Development and Medical Affairs Respiratory Specialty Care Boehringer Ingelheim Pharmaceuticals, Inc. MedicalResearch.com: What is the background for this announcement? Would you briefly explain what is meant by systemic sclerosis and how it affects lung function?  Response: In September, the U.S. Food and Drug Administration (FDA) approved Ofev as the first and only therapy to slow the rate of decline in pulmonary function in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD). The FDA granted priority review and Fast-Track designation earlier this year. Interstitial lung disease (ILD) is characterized by thickening and scarring of lung tissue and is the leading cause of death among people with systemic sclerosis (SSc), also known as scleroderma. In fact, approximately 25 percent of SSc patients develop significant lung involvement within three years of diagnosis. Prior to this approval, there were no options for this patient population, making this an exciting announcement for the community.  (more…)
AstraZeneca, Author Interviews, Pulmonary Disease / 17.09.2019

MedicalResearch.com Interview from: AstraZeneca Spokesperson MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by COPD? How common is severe COPD? Response: COPD, or chronic obstructive pulmonary disease, is a progressive disease associated mainly with tobacco smoking, air pollution or occupational exposure, which can cause obstruction of airflow in the lungs making it hard to breathe. In the United States, COPD is the fourth leading cause of death. Its prevalence in adults 18 years of age and older is 6.5 percent. An estimated 16 million people are currently diagnosed with COPD, and millions more are believed to have it but do not know it. ETHOS is a randomized, double-blind, multi-center, parallel-group, 52-week trial to assess the efficacy and safety of PT010 in symptomatic patients with moderate to very severe COPD and a history of exacerbation(s) in the previous year. The trial compared two doses given twice daily of PT010 (320/14.4/9.6mcg and 160/14.4/9.6mcg) with glycopyrrolate/formoterol fumarate (14.4/9.6mcg) and PT009 (320/9.6mcg), all using AEROSPHERETM Delivery Technology in a pressurized metered-dose inhaler (pMDI). Outcomes in the ETHOS trial included, as a primary endpoint, the rate of moderate or severe exacerbations. The Phase III ETHOS trial builds on the Phase III KRONOS data which together show PT010’s ability to reduce exacerbation risk in a broad range of patients with COPD, irrespective of whether they have had an exacerbation in the previous twelve months. (more…)
Author Interviews, Flu - Influenza, Pulmonary Disease, Stem Cells, University of Pennsylvania / 22.08.2019

MedicalResearch.com Interview with: Andrew E. Vaughan, PhD Assistant Professor, Biomedical Sciences School of Veterinary Medicine University of Pennsylvania MedicalResearch.com: What is the background for this study? What are the main findings? Response: Severe respiratory infections, including influenza, can progress to acute respiratory distress syndrome (ARDS), wherein barrier function and gas exchange are compromised.  It’s a very life threatening scenario.  This is due in part to loss of alveolar type 2 (surfactant producing) and type 1 cells (gas exchanging).  Interestingly alveolar type 2 cells are also stem cells in the lung.  We wondered whether transplant of these cells might aid in recovery from severe influenza infection, and sure enough, it did! (more…)