What’s Involved

Multiple Breath Washout (MBW)

MBW is a simple breathing test involving normal quiet breathing through a mouthpiece or face mask. It involves breathing in 100% oxygen for a few breaths and then measuring the gases that are breathed out to determine if there is any slowing down of airflow in the lungs. The test takes about 20-30 minutes overall as the procedure is repeated three times for accuracy. MBW will be performed at your child’s initial visit and annually thereafter for 5 years.

Ultra-low dose, spirometry-controlled CT scanning

A CT scan is a detailed three-dimensional image of the inside of the lungs and airways. It allows us to collect detailed information in relation to CF lung disease. CT scans are extremely helpful in CF but are associated with radiation, so they must be used carefully over a person’s lifetime. The type of CT needed in CF can be performed with substantially less radiation than a standard CT scan. Many centres around the world perform low dose CT scans every two years in people with CF as part of annual assessment. Our partners in the LungAnalysis Core in Erasmus, Rotterdam have been working with hospitals around the world to standardise CT scanners, advise on ultra-low dose protocols and analyse the results. LungAnalysis has worked with each centre in ENHANCE to standardise scanners and help keep doses down. For ENHANCE, we plan to perform CT scans at the age of 6 ,12 and 16 years at centres where routine scans are performed(in the initial and follow-on studies). In centres that perform scans every two years, as part of usual care, we will collect the images and use them for the study also.

Sweat Chloride

Sweat chloride is a painless procedure and performed on both arms. Two electrodes are placed on the arm with a gel disc containing pilocarpine (a medicine that makes the sweat glands produce sweat) for 5 minutes to stimulate skin. Then a small sweat collecting “watch” is placed on the arm to collect the sweat. This takes roughly around 45 minutes in total to complete (30-minute sweat collection time per arm). Sweat will be analysed on samples using a specialised machine at each centre. This information will then be input into the database by the CF researcher.

Oropharyngeal Swabs (OPS)

OPS swabs (throat swabs) are collected regularly in children with CF as part of their usual care. For ENHANCE study visits, we will organise for these to be performed with a dual-tip throat swab. One tip of the cough swab will be sent to the local hospital microbiology labs for processing as usual and the second tip will be stored at the central labs in Dublin (CHI) for future microbiology analysis.

Liver Ultrasound

As per routine clinical practice, most people with cystic fibrosis from 6 years of age onwards have annual liver ultrasound scans as part of annual assessment. An ultrasound is a painless procedure that takes less than 15 minutes to perform. The ultrasound scan will be performed at recruitment and annually thereafter for 5 years. We will link in with the radiology department in each hospital to collect the relevant information when routine scans are being performed, so repeat tests for ENHANCE are not necessary. For centres where annual ultrasound is not performed every year, we will ask that a scan is performed each year for ENHANCE.

Stool Collection

A stool sample will be collected from study participants at study visits at recruitment and annually for the duration of the study. Sample collection equipment will be provided in advance to participant’s parents, so that samples can be collected and refrigerated (if necessary) on the morning of a study visit.

Abdominal Symptom Score

Our collaborators in Brandenburg, Germany have developed a specific abdominal symptom questionnaire for people with CF. The CFAbd score is validated in children and adults with CF and scores correlate well with ultrasound findings and gut inflammation. The CFAbd score has been used in studies in leading international centres. This questionnaire will take 5-8 minutes to complete the first time and 3-5 minutes on subsequent occasions. Questionnaires will be delivered at centres by the local research coordinator at recruitment and annually thereafter for the duration of the study.

CFQ-R

Over the last few decades a growing recognition of the importance of patient reported health related quality of life indices has developed. These indices are now recognized by patient organisations, clinicians and regulators as a vital tool in the development and testing of new treatments in people with cystic fibrosis. The Cystic Fibrosis Questionnaire-Revised (CFQR) is a specifically designed quality of life questionnaire for people with CF. CFQ-R will be delivered to participant’s aged 6years and above and their parents annually for the duration of the study.

Mental health Questionnaires

Mental health will be assessed in ENHANCE participants aged 1month to adulthood. Although mental health is one of the top 3 research priorities of the CF community, major gaps remain regarding the study of mental health conditions in the CF population. ENHANCE will improve our knowledge within multiple mental health areas of focus. Mental health outcomes will be measured annually using the following questionnaires;

•The Paediatric Symptom Checklist (PSC): A brief and widely used parent-report questionnaire. The PSC measures cognitive, emotional and behavioural difficulties in children aged 1month-17 years.

•Patient Health Questionnaire – 9 and Generalized Anxiety Disorder -7: These brief questionnaires are used in both research and clinical practice and are recommended in international guidelines as measures of depression and anxiety symptoms in adolescents and adults with CF.  These questionnaires will be completed by children aged 12 years and over. 

•The PROMIS Paediatric Anxiety Scale and Depressed Mood Scale: Brief self-report questionnaires administered to children from the age of 8 years, providing discreet measures of depressed mood and anxiety for younger children.

•The PROMIS Paediatric Cognitive Function Scale: This is a brief self-report questionnaire of children’s experiences of attention, memory and comprehension, which is validated for use from the age of 8 years.

•Body image will be assessed using a three-item questionnaire designed for this study, completed by children from the age of 8 years.

Please note, if there are concerns about self-harm, suicidal ideation, clinically significant levels of anxiety or depression, or significant impact to quality of life following completion of the questionnaire, these concerns will be relayed to your child’s local PI, a CF nurse specialist and local CF psychologist.  Further assessment, psychological support and/or onward referral will be available to all participants if/as necessary.

Other clinical parameters in medical notes

Results from other clinical parameters as above will be collected from your child’s chart. Data collected will include the following:

•Measurement of FEV₁

•Liver disease parameters

•Nutritional indices such as height, weight and BMI

•Airway sample microbiology results