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Cystic fibrosis carriers at increased risk of digestive symptoms

Researchers have found that carriers of the most common genetic variant that causes cystic fibrosis experience some symptoms similar to those of people with cystic fibrosis. These findings were enabled by large-scale genomic data made available just a few years ago. Yu-Chung (Jerry) Lin, BA, MSc, a graduate student at the University of Toronto, presented the research at the American Society of Human Genetics 2019 Annual Meeting in Houston, Texas. Cystic fibrosis is a recessive disease caused by variants of the CFTR gene, which means that affected individuals have two such variants. It affects many organs, and symptoms vary from patient to patient. They can include lung function decline, inte

First Patients Dosed in Trial Testing MS1819-SD Plus PERT for CF-related Severe EPI, AzurRX Says

AzurRx BioPharma has started dosing patients in a Phase 2 clinical trial assessing its investigational therapy MS1819-SD in combination with porcine enzyme replacement therapy (PERT) for people with cystic fibrosis (CF) who have severe exocrine pancreatic insufficiency (EPI). People with CF can develop EPI when mucus obstructs the ducts of the pancreas, which prevents digestive enzymes from getting into the gastrointestinal tract. Patients then can’t digest food properly, which leads to malnutrition. The current standard of care for EPI is PERT — digestive pig enzymes similar to the ones produced by the human pancreas. With PERT, the enzymes are taken by mouth in a capsule. However, around o

New Vertex cystic fibrosis drug approved, extending treatments to 90% of patients

Vertex Pharmaceuticals on Monday won approval for its fourth treatment for cystic fibrosis, a combination drug that extends a new form of therapy to 90% of CF patients. The drug will be marketed as Trikafta. The list price is $311,000 per year — the same as one of Vertex’s (VRTX) earlier CF treatments. In a statement, acting Food and Drug Administration Commissioner Ned Sharpless said the approval makes “a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy.” Cystic fibrosis is a progressive disease that damages the lungs and is caused by m

Prevalence and characterization of chronic rhinosinusitis in patients with non–cystic fibrosis bronc

In this retrospective cohort study, researchers identified the prevalence of chronic rhinosinusitis (CRS) among bronchiectasis patients affiliated with a US tertiary medical center. In addition, they determined which comorbid diseases were correlated with the presence of CRS in bronchiectasis patients. In 45% of patients with bronchiectasis, CRS was present. Majority of bronchiectasis patients, both with and without CRS, were women. Findings suggested an association of asthma, allergic rhinitis, gastroesophageal reflux disease, and antibody deficiency with the presence of CRS in bronchiectasis patients after controlling for demographic factors. Such results have medical implications for the

CF patients experience improved lung health with lumacaftor-ivacaftor but with caveats

In adolescent and adult patients with cystic fibrosis taking lumacaftor-ivacaftor (ORKAMBI), the combination drug appears to improve lung function and body weight and reduce the need for intravenous antibiotic treatment, according to a French study published online in the American Thoracic Society's American Journal of Respiratory and Critical Care Medicine. However, nearly one in five patients could not tolerate treatment, most often due to adverse breathing events, during one year of follow up. The rate of discontinuation was considerably higher than previously reported in clinical trials, the authors wrote. In "Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cy

Can Blood Biomarkers Predict Clinical Response to Azithromycin in Cystic Fibrosis Patients?

Although azithromycin can lower pulmonary exacerbation (PEx) risk in patients with cystic fibrosis (CF), it does not help all patients. This study assessed whether any blood protein markers could serve as indicators of clinical response to azithromycin treatment in young CF patients. The study included 188 serum and plasma protein samples from 40 CF patients who were randomized to receive azithromycin in the AZ0004 trial. The researchers applied novel proteomic technologies to the samples and analyzed early changes in blood protein levels from day zero to 28 of treatment in regard to changes in forced expiratory volume in one second (FEV1) percent predicted and weight by days 28 and 168 and

Viral Respiratory Tract Infections in Young CF Children Usually Mild, Study Found

Viral respiratory tract infections in young children with cystic fibrosis tend to be mild and not require hospitalization, a study found. Infections caused by respiratory syncytial virus (RSV) were rare and did not lead to complications, suggesting that there is no need for preventive medication such as Synagis (palivizumab) in this young population. The study, “Viral respiratory tract infections in young children with cystic fibrosis: a prospective full-year seasonal study,” was published in Virology Journal. Bacteria are considered the leading cause of respiratory tract infections in young children with CF, but viral infections also are common. RSV can cause severe infection in some patien

Evaluating the Role of New Beta-Lactam Agents for Uncommon Pathogens

Stenotrophomonas maltophilia, Achromo-bacter xylosoxidans, and Burkholderia spp are relatively uncommon pathogens that are increasingly seen as causes of clinically significant infections, particularly respiratory tract infections among immunocompromised hosts and other vulnerable populations.1-4 Patients with cystic fibrosis (CF) are at an elevated risk for chronic infections and morbidity. According to the 2017 Cystic Fibrosis Patient Registry, which provides comprehensive data on nearly 30,000 patients with CF, S maltophilia was identified in 12.9% of patients, A xylosoxidans in 5.7%, and Burkholderia cepacia complex in a further 2.5%.5 Patients with cancer, specif­ically those with a h

Screening for cystic fibrosis-related diabetes and prediabetes: Evaluating 1,5-anhydroglucitol, fruc

Researchers examined whether alternate glycemic markers—hemoglobin A1c (HbA1c), 1,5-anhydroglucitol (1,5AG), fructosamine (FA), and glycated albumin (GA)—have utility as screening tests for cystic fibrosis (CF)-related diabetes (CFRD) and prediabetes (CFPD) in youth with CF as defined by the gold-standard oral glucose tolerance tests (OGTT) 2-hour glucose (2hG). Collection of youth 10 to 18 years with CF who had a 1,5AG, FA, GA, HbA1c, and 2-hour OGTT was done. They included 58 youth with CF (2hG < 140, n = 16; CFPD, n = 33; CFRD, n = 9; 41% male, mean ± SD age 14.2 ± 3.6 years, BMI z-score 0.0 ± 0.8, % predicted forced expiratory volume in 1 second 89.9 ± 15.1, % predicted forced vital capa

Hexoskin Smart Shirt Can Accurately Assess Respiratory Function, May Be Tool in CF, Study Shows

Using a simple shirt — a smart one — is closer to becoming a common method to measure lung function in patients with respiratory conditions, including chronic obstructive pulmonary disease (COPD) and cystic fibrosis. A new study conducted by researchers from Radboud University Medical Center and University of Twente, both in the Netherlands, has showed that the Hexoskin smart shirt — developed by Carré Technologies — can accurately assess lung function in healthy people. Supported by these positive results, the team plans to further explore the potential of this smart shirt to monitor lung function in people with lung diseases, like COPD. “Smart shirts are already available, but they tend to

5,000 Expected to Attend Cystic Fibrosis Conference, NACFC 2019, in Nashville

The Western Hemisphere’s largest gathering of experts in cystic fibrosis gets underway later this month in Nashville, Tennessee — site of the 33rd annual North American Cystic Fibrosis Conference (NACFC). The Oct. 31–Nov. 2 event, sponsored by the Cystic Fibrosis Foundation (CFF), will attract more than 5,000 delegates to discuss a disease that affects between 70,000 and 100,000 people worldwide, including 30,000 in the United States alone. That’s a far cry from 1987, when the first NACFC, in Toronto, had 300 participants. Cystic Fibrosis News Today will cover this year’s meeting onsite, as it did last year in Denver. All three plenaries and other selected sessions will also be streamed live

New Clinical Tool Predicts 1- and 2-Year Mortality in Cystic Fibrosis

Researchers in Canada have developed a novel clinical tool that uses patients’ overall health status and the risk for intermittent shock events to predict 1- and 2-year mortality in cystic fibrosis (CF), according study results published in European Respiratory Journal.1 Although survival in patients with CF has improved steadily during the past 3 decades, CF remains a life-shortening illness with half of all deaths occurring before the age of 35 years.2 Thus, there is a great need for developing an accurate prediction model for mortality to identify patients who would benefit from expedited referral to a lung transplant program. The researchers sought to develop a clinical tool for predicti

Lower airway infection prevalence changing in children with cystic fibrosis

The prevalence of lower airway infections with Pseudomonas aeruginosa, Staphylococcus aureus and Haemophilus influenzae among young children with cystic fibrosis has decreased significantly during the past 2 decades, according to a study published in the American Journal of Respiratory and Critical Care Medicine. To analyze the prevalence of early lower airway infections among children with cystic fibrosis, researchers evaluated data for all after newborn screening who were participating in the Australian Respiratory Early Surveillance Team for Cystic Fibrosis (ARREST CF) cohort study. Infants underwent bronchoalveolar lavage at 3 to 6 months, 1 year and annually up to age 6 years. The study

AzurRx BioPharma Announces Promising Results from Trial Testing MS1819 for EPI in CF Patients

The investigational therapy MS1819 showed positive safety results in a Phase 2 clinical trial for the treatment of exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF), AzurRx BioPharma announced. In people with CF, EPI can occur when mucus builds up and blocks the ducts of the pancreas. This can prevent important enzymes from getting into the digestive tract, making it difficult for those affected to adequately absorb nutrients from their food. The current standard care for EPI in CF is porcine enzyme replacement therapies (PERT). As its name suggests, this involves taking supplemental versions of the missing pancreatic enzymes that are derived from pigs. Like PERT, M

Progress for Cystic Fibrosis [INFOGRAPHIC]

These figures were taken from the Cystic Fibrosis Foundation’s (CFF) 2017 Patient Registry Annual Data Report. The mortality rate in 2017 was 1.3 deaths per 100 individuals with CF in the Registry. There has been a steady decrease in the mortality rate over the last 30 years. This is encouraging, given that the median age of individuals in the Registry has increased from 11.7 years in 1987 to 19.3 years in 2017. Over the last ten years, there has been almost a doubling of the number of people with CF who are married or living together and the number of people with a college degree. The number of pregnancies among women with CF has increased steadily since the 1990s. Registry data show that 2

Translate Bio Prioritizes Development of CF Therapy MRT5005, Other Lung Disease Candidates

Translate Bio is going to focus on the development of potential therapies to treat pulmonary diseases, including its lead candidate, MRT5005, targeting the underlying cause of cystic fibrosis (CF), the company announced. MRT5005 is designed to restore the levels of working cystic fibrosis transmembrane conductance regulator (CFTR) protein and reverse the effects of any CF-related genetic mutation. The therapy is formulated to be directly delivered to airway lining cells via nebulization, and it contains the RNA sequence of functional CFTR gene, which will work as a template to produce the protein CF patients are lacking. MRT5005 was granted orphan drug designation in 2015 by the U.S. Food an

Prenatal Testing for Cystic Fibrosis Influences Pregnancy Outcomes, Review Finds

Prenatal genetic testing for cystic fibrosis (CF) significantly influences how women manage their pregnancies, with positive tests linked to high rates of pregnancy terminations, a systematic review study finds. The study, “Prenatal genetic testing for cystic fibrosis: a systematic review of clinical effectiveness and an ethics review,” was published in the journal Genetics in Medicine. Prenatal screening for mutations in the CFTR gene — the root of CF development — is performed when a fetus has a high risk for inheriting the disease. This is the case when both parents carry CF-causing genetic mutations, which represents a 25% risk of their descendants developing CF — a 625-times higher risk

Physical Activity Levels in CF Should Be Objectively Researched, Review Says

Adults with cystic fibrosis (CF) are less active than they should be, although they have comparable activity levels to people without the genetic disorder, according to a recent review. The review also showed that there are not enough studies that objectively assess physical activity levels in CF. Therefore, more research in this area is warranted, the investigators said. The study, “Physical activity and associations with clinical outcome measures in adults with cystic fibrosis; a systematic review” was published in the Journal of Cystic Fibrosis. Physical activity is known to benefit all people, but CF patients gain even more from being physically active because exercise can slow down the

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