
Cystic Fibrosis Adherence Study
Cystic FibrosisThis study aims to assess the role of some factors which can influence the chest physiotherapy adherence in adult with cystic fibrosis. At first, we are going to try to consider the adherence of chest physiotherapy. Then, after dividing the patients into two groups - adherent or not adherent - we will attempt to identify the factors which can influence this adherence. It consists in a short questionnaire that the patient will fill during usual visits. The hypothesis is that the following factors can play a role in adherence of chest physiotherapy: Age Age of diagnostic Sex Fev1 Number of antibiotic course Anxiety/depression Work time Socio-professional category Individual situation Physiotherapy with liberal physiotherapist and transport time Sport practice Family support Feeling of work time burden Feeling of physiotherapy efficiency Feeling of physiotherapy burden

Evaluation of Sit-to-stand Test in Patients With Cystic Fibrosis and Matched Controls.
Cystic FibrosisIntroduction: In recent years, since the discovery of the cystic fibrosis (CF) transmembrane conductance regulator gene in human skeletal muscle, there appears to be growing interest in the measurement of muscle function in CF. One of the most used test in other chronic pulmonary diseases is the Sit-to-Stand test (STS) which consists of simply getting up from a chair. Although the main result of the STS test is the time developed during the task, the velocity and power generated during the task are considered very important variables to detect the functional decline. However, from our knowledge, no study has previously analyzed the differences in time, velocity and muscle power developed during the STS test in patients with CF and their respective healthy controls. Objectives: To compare the values gained from handgrip strength, walking speed and STS test (time, velocity, and muscle power) in a group of patients with CF and their respective healthy controls, and to analyze if these differences (if any) are associated with lung function in patients with CF. Methods: Cross-sectional study with a sample of 60 participants (30 patients diagnosed with CF and 30 healthy subjects) between 18-65 years old. The STS test will be measured through slow-motion video recording with a smartphone device (240 images per second) which will report the time, velocity and power generated during the test. Walking speed and handgrip strength will be also measured. Additionally, the relationship between the variables obtained during the test and the lung function of patients with CF will be analized.

Evaluation of Salt Status in Patients With Cystic Fibrosis
Salt; Deficit (or Low)Cystic FibrosisThe results of the annual check-up will be collected to evaluate the best urinary marker for fractional sodium excretion and salt status will be correlated to clinical outcome measures.

Pain in Patients With Cystic Fibrosis in Rouen University Hospital
PainCystic FibrosisObjectif Principal - To evaluate the intensity and frequency of pain episodes in patients with cystic fibrosis followed at the cystic fibrosis center of Rouen University Hospital, Normandy, France. Objectifs secondaires To assess the relationship between pain and disease severity. To assess the relationship between pain and the age of the patient. To describe the pain locations. To describe the use of pharmacological or nonpharmacological treatment. To evaluate the procedural pain.

A Study of Web and Tablet-based Interactive Audiometry in Adults With CF
Hearing LossSensorineural1 moreAdults with CF regularly receive anti-infective therapy that can result in impaired hearing. Recent studies have identified that up to 50% of adults with CF have hearing loss with resulting significant impact on quality of life. At present, however, there is no national recommendation to screen for hearing loss within adult CF. Formal hearing tests are costly, require specialist staff and equipment and require further outpatient visits resulting in significant impact on health, social and financial resource. We propose to analyse over a 12 month period at two adult cystic fibrosis centres in London whether outpatient tablet based audiometry testing and web-based hearing apps are able to accurately screen for ototoxicity in adults with CF, and whether these methods of screening are acceptable. The results of this study will aim to streamline audiometry screening to improve detection rates, reduce cost of healthcare, enable equity in service delivery, and minimize disruption to the patient's social and work-life using smart technology.

Qualitative Understanding of Experiences With the SIMPLIFY Trial
Cystic FibrosisDrug WithdrawalThis qualitative longitudinal study is designed to elicit and thematically analyze the perspectives of SIMPLIFY subjects about treatment withdrawal research and treatment burden in the context of triple combination CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator therapy (TCT). Cohorts will be determined based on sub-groups of the assigned treatment arms from the SIMPLIFY study. Participants will be asked to participate in two interviews; the first interview will be conducted at the completion of the SIMPLIFY study and the second interview will be conducted approximately 4 months after the first interview. Trained research staff will conduct the semi-structured interviews that will be audio-recorded and transcribed for thematic analysis. Each interview should last between 30-60 minutes.

Treatment With Elexacaftor/Tezacaftor/Ivacaftor, in Patients With Cystic Fibrosis and Caloric Intake...
Cystic FibrosisThe aim of the study is to describe the evolution of caloric intake in patients with cystic fibrosis with an indication to start treatment with Elexacaftor/Tezacaftor/Ivacaftor according to the Marketing Authorization, between the start of treatment and at 12 months.

Intestinal Fatty Acid Binding Protein in Very Low Birth Weight Infants With Meconium Obstruction...
Meconium Obstruction of PrematurityUrinary intestinal fatty acid binding protein (i-FABP), a marker of intestinal mucosal cell damage, has recently been proposed as a clinically useful measure in the early detection of necrotizing enterocolitis (NEC). However, there are no data on urinary i-FABP in meconium obstruction of prematurity (MOP). This study aimed to evaluate urinary i-FABP in MOP patient as a marker for early detection.

Expanded Access Program for Aztreonam Lysine for Inhalation in Canadian Patients With Cystic Fibrosis...
Cystic FibrosisPseudomonas AeruginosaThis program is to provide expanded access to aztreonam lysine for inhalation (AZLI) prior to its commercial availability to patients with cystic fibrosis (CF) and chronic P. aeruginosa airway infection who have limited treatment options and are at risk for disease progression.

Indirect Comparison of Tobramycin Solution for Inhalation Versus Aztreonam Lysine for Inhalation...
Cystic FibrosisMatching-adjusted indirect comparison of tobramycin solution (TIS) versus aztreonam lysine (AZLI) using TIS patient level clinical trial data and AZLI aggregated clinical trial data from published literature.