Early and sustained improvements of lung clearance index from two to sixteen weeks of elexacaftor/tezacaftor/ivacaftor therapy in patients with cystic fibrosis-a real world study.

Since the introduction of CFTR modulator therapies, longitudinal real-life data of lung clearance index (LCI) during treatment is scarce. In this single-centre, post-approval setting, we report data of 51 patients with different stages of lung disease, age 2-52 years with repeated measurements of forced expiratory volume as a percentage of the predicted value (ppFEV₁) and LCI after 2, 4, and 16 weeks of CFTR modulator treatment and at baseline. In 25 patients during elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) treatment, significant improvements of LCI (median -1.

Malignancies in patients with cystic fibrosis: a case series.

Background: Previous reports have shown an increased number of colorectal cancers in patients with cystic fibrosis. We assessed the database of our cystic fibrosis center to identify patients with all kinds of cancer retrospectively. All patients visiting the Cystic Fibrosis Centre Innsbruck between 1995 and 2019 were included.

Arm muscle area for the longitudinal assessment of nutritional status in paediatric patients with cystic fibrosis - A single centre experience.

Background: In cystic fibrosis, adequate nutrition contributes to good long-term prognosis. A body mass index (BMI) at or above the 50th percentile for age and sex in all children has been recommended. As researchers have described a depletion of fat-free mass despite normal BMI, longitudinal studies using more sensitive nutritional parameters are warranted.

Reference Values Matter: Fewer Patients With Malnutrition Using American Compared to More Recent German Growth Charts.

Reference values are important for patient care as well as for comparisons between different centers or countries. We investigated two anthropometric reference datasets, the US Centers for Disease Control (CDC) growth charts and the German Health Interview and Examination Survey for Children and Adolescents Study (KiGGS) percentiles, which were established in Germany between 2003 and 2006. A smaller proportion of children with cystic fibrosis had decreased z scores <-2 with CDC (5.

Lung clearance index to detect the efficacy of Aztreonam lysine inhalation in patients with cystic fibrosis and near normal spirometry - A single-centre feasibility study.

Comparing the efficacy of inhaled antibiotics can be difficult in small groups of patients with cystic fibrosis and mild lung disease. In a feasibility study we compared Aztreonam lysine for inhalation solution (AZLI; Cayston®) to standard inhaled antibiotic therapy in patients with cystic fibrosis and near normal spirometry. To detect treatment responses we used both lung clearance index (LCI) and forced expiratory volume in one second (FEV1).

Fecal Calprotectin in Cystic Fibrosis and Its Relation to Disease Parameters: A Longitudinal Analysis for 12 Years.

Objectives: Fecal calprotectin (FC) is a marker of inflammation in the intestinal tract. We assessed FC levels longitudinally in patients with cystic fibrosis (CF) and evaluated the relation between FC results and relevant markers of disease.

Methods: Calprotectin was measured in fecal samples starting in 2003 and values were stored in the center's patient database.

Long-term improvement of lung clearance index in patients with mild cystic fibrosis lung disease: Does hypertonic saline play a role?

To assess whether long-term inhalation with hypertonic saline is able to halt the progression of mild CF lung disease, we analysed longitudinal data of lung clearance index (LCI) and spirometry. A total of 34 patients with mild lung disease (FEV1 ≥ 70% of predicted) had at least one LCI result before and ≥2 LCI measurements after start of hypertonic saline (HS) therapy. After a mean follow-up of 39.

Evaluating patient experience in a cystic fibrosis centre using a disease-specific patient satisfaction questionnaire.

Unlabelled: Medical care for persons with chronic diseases like cystic fibrosis (CF) is provided by multi-professional teams. We assessed the patients' perspective of care by reporting the results of two consecutive patient satisfaction surveys performed within a 2-year interval at our CF centre. The newly developed, disease-specific questionnaire for parents and adults had 104 items with up to 6 response categories each.

Cystic fibrosis (CF) care through the patients' eyes - a nationwide survey on experience and satisfaction with services using a disease-specific questionnaire.

Unlabelled: The patients' perspective is an important aspect of quality management. A newly developed disease-specific questionnaire was used to assess the patients' experiences with care provided in specialised cystic fibrosis (CF) care centres.

Methods: 90 CF centres in Germany were invited to participate.

Patient experience in cystic fibrosis care: Development of a disease-specific questionnaire.

Objectives: The aim of this study was to develop valid and reliable disease-specific questionnaires for adult patients with cystic fibrosis and for parents of minors with cystic fibrosis for assessing patient experience with cystic fibrosis care.

Methods: The pilot versions of the questionnaires were developed based on a literature review, interviews with health professionals and focus groups. A postal survey with two reminders was conducted in 56 German cystic fibrosis centres recruiting 2874 participants.

Tracking Lung Clearance Index and chest CT in mild cystic fibrosis lung disease over a period of three years.

Introduction: Lung disease remains the main cause of morbidity and mortality in patients with Cystic Fibrosis (CF). To detect lung disease before clinical symptoms become apparent, sensitive tools are essential. Spirometry is used for monitoring, but the FEV1 remains frequently normal throughout childhood.

Sensitivity of Lung Clearance Index and chest computed tomography in early CF lung disease.

It is widely accepted that CF lung disease starts before clinical symptoms become apparent or spirometry deteriorates. Computed chest tomography (CT) is the reference method for identifying structural changes in CF; however, radiation exposure limits its use as a monitoring tool. It has been suggested that the Lung Clearance Index (LCI) measured by Multiple Breath Washout (MBW) for assessing ventilation inhomogeneity is a more sensitive surrogate marker than spirometry allowing non-invasive monitoring of CF lung disease.

Add-on salmeterol compared to double dose fluticasone in pediatric asthma: a double-blind, randomized trial (VIAPAED).

Rationale: In asthmatic children whose symptoms are uncontrolled on standard doses of inhaled corticosteroids (ICS), guidelines recommend to either increase the ICS dose or to add further controller medication, e.g. a long acting ss2-agonist (LABA).

Once-weekly azithromycin in cystic fibrosis with chronic Pseudomonas aeruginosa infection.

Background: Data on the effects of long-term treatment with azithromycin (AZM) on inflammatory markers in cystic fibrosis patients chronically infected with Pseudomonas aeruginosa are scarce. So far there is no pharmacokinetic and clinical data on once-weekly dosage of AZM in CF patients.

Methods: In a randomised double-blind, placebo-controlled trial, patients received AZM or placebo 1 per week for 8 weeks (AZM dosage--20-29 kg: 500 mg, 30-39 kg: 750 mg, 40-49 kg: 1000 mg and > or = 50 kg: 1250 mg) after a course of intravenous antipseudomonal antibiotics.

Parental knowledge and behaviour to prevent environmental P. aeruginosa acquisition in their children with CF.

Background: Chronic infection with Pseudomonas aeruginosa (PA) is associated with an accelerated decline of lung function in patients with cystic fibrosis (CF). Precautions to avoid contact with PA from the environment have been recommended by caregivers and are being carried out by many families with CF children. The present study was conducted to portray the spectrum of hygienic measures and to evaluate the restrictions and impact caused by these measures.

Different opinions of physicians on the importance of measures to prevent acquisition of Pseudomonas aeruginosa from the environment.

Background: Since chronic infection with mucoid Pseudomonas aeruginosa (PA) is associated with deteriorating lung function, many parents of young children with cystic fibrosis (CF) fear the first PA positive throat swab as a milestone in the progression of the disease. To reduce the risk of PA acquisition from the environment, they perform preventive measures at home or outdoors.

Methods: In an attempt to evaluate the attitude of CF physicians towards these measures and the respective consulting practice, we mailed a questionnaire to all 65 certified paediatric CF centres in Germany.

Parental fears of Pseudomonas infection and measures to prevent its acquisition.

Background And Aim Of The Study: Chronic infection with Pseudomonas aeruginosa (PA) is associated with accelerated worsening of lung disease in patients with cystic fibrosis (CF). Fears of PA are widespread among parents of CF children, and many parents take precautions at home to prevent acquisition of the bacterium from the environment. The present study was undertaken to describe the type and intensity of these activities.