Cystic Fibrosis
Conditions
Keywords
Elexacaftor-Tezacaftor-Ivacaftor, Cystic Fibrosis, Adapted Physical Activity, Body composition, Dietetic
Brief summary
Cystic fibrosis is an autosomal recessive inherited disease linked to various mutations in the gene coding for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, with respiratory and digestive disorders conditioning the prognosis. Digestive damage may be responsible for malnutrition of multifactorial origin (insufficient energy intake, increased energy losses, increased basal metabolic rate), and studies show a correlation between reduced lean body mass and respiratory function. In 2019, the French National Authority for Health (HAS) redefined undernutrition by including quantified reduction in muscle mass and/or function as a phenotypic diagnostic criterion. Elexacaftor-Tezacaftor-Ivacaftor, an innovative therapy (authorization in 2021) for this population, aims to restore the function of CFTR protein. Significant improvements in lung function and weight gain were observed from the first weeks of treatment. These improvements have also led to the emergence of lesser-known nutritional problems in these patients, such as overweight and the development of metabolic complications. Nonetheless, new management options in terms of dietary adjustments and adapted physical activity for these patients are possible, given the development of their abilities. Adapted Physical Activity (APA) helps to improve general muscular function by strengthening respiratory and skeletal muscles, improving aerobic capacity, and aiding bronchial drainage through muscle strengthening and endurance work. Maintaining or even increasing muscle mass depends not only on appropriate food intake and optimal dietary management, but also on regular physical activity, as recommended by the HAS. Our hypothesis is therefore that a structured dietetic/adapted physical activity program (DIAPASOM program) can increase the percentage of lean body mass at 12 months in adult cystic fibrosis patients treated with Elexacaftor-Tezacaftor-Ivacaftor.
Detailed description
Cystic fibrosis is an autosomal recessive inherited disease linked to various mutations in the gene coding for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, with respiratory and digestive disorders conditioning the prognosis. Digestive damage may be responsible for malnutrition of multifactorial origin (insufficient energy intake, increased energy losses, increased basal metabolic rate), and studies show a correlation between reduced lean body mass and respiratory function. In 2019, the French National Authority for Health (HAS) redefined undernutrition by including quantified reduction in muscle mass and/or function as a phenotypic diagnostic criterion. Elexacaftor-Tezacaftor-Ivacaftor, an innovative therapy (authorization in 2021) for this population, aims to restore the function of CFTR protein. Significant improvements in lung function and weight gain were observed from the first weeks of treatment. These improvements have also led to the emergence of lesser-known nutritional problems in these patients, such as overweight and the development of metabolic complications. Nonetheless, new management options in terms of dietary adjustments and adapted physical activity for these patients are possible, given the development of their abilities. Adapted Physical Activity (APA) helps to improve general muscular function by strengthening respiratory and skeletal muscles, improving aerobic capacity, and aiding bronchial drainage through muscle strengthening and endurance work. Maintaining or even increasing muscle mass depends not only on appropriate food intake and optimal dietary management, but also on regular physical activity, as recommended by the HAS. Our hypothesis is therefore that a structured dietetic/adapted physical activity program (DIAPASOM program) can increase the percentage of lean body mass at 12 months in adult cystic fibrosis patients treated with Elexacaftor-Tezacaftor-Ivacaftor.
Interventions
OTHERDIAPASOM program
A program of adapted physical activity carried out remotely by a specialist instructor, combined with personalized dietetic care by a dietician for one year.
Sponsors
Fondation Ildys
University Hospital, Tours
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subject aged 18 or over * Suffering from cystic fibrosis * Treated with Elexacaftor-Tezacaftor-Ivacaftor for at least 6 months * Affiliated to a social security scheme * with a signed Informed Consent form.
Exclusion criteria
* Pregnant and breast-feeding women * Subject under legal protection, guardianship or curatorship * Subject whose physical activity is not medically authorised or whose physical and motor capacities do not allow them to take part in physical activity. * Subject who is unable to comply with the requirements of the DIAPASOM program * Difficulty in understanding the self-questionnaires * Wearing a pacemaker or metal prosthesis * Fluid retention
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evolution of percentage of patients lean mass as a percentage of body mass | From randomization, up to 12 months | Impedancemetry |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Weight evolution | From randomization, up to 12 months | Weight measurement |
| Body Mass Index (BMI) evolution | From randomization, up to 12 months | Weight and height measurement |
| Cardio-respiratory endurance | From randomization, up to 12 months | 6-Minute Walk Test (6MWT) |
| Bilateral Handgrip strength | From randomization, up to 12 months | Handgrip Test |
| Lower limb muscle power | From randomization, up to 12 months | 30-second Sit-to-Stand test |
| Upper limb muscle power | From randomization, up to 12 months | Pump test |
| Static trunck extensors muscle endurance | From randomization, up to 12 months | Superman test |
| Evolution of percentage of patients fat mass as a percentage of body mass | From randomization, up to 12 months | impedancemetry |
| Balance between static muscular endurance of extensors and flexors | From randomization, up to 12 months | Calculated using the Shirado-Ito/Superman ratio |
| Posterior chain flexibility (Hamstring, hips and lower back) | From randomization, up to 12 months | Front trunk flexion test |
| Upper limb flexibility | From randomization, up to 12 months | scapulohumeral mobility test |
| Forced Expiratory Volume in 1 second (FEV1) | From randomization, up to 12 months | Spirometry |
| Physical activity volume and sedentary time | From randomization, up to 12 months | Physical activity and sedentary behavior self-questionnaire (ONAPS-PAQ) |
| Evolution of quality of life | From randomization, up to 12 months | Cystic Fibrosis Questionnaire-Revised (CFQR-14) |
| Program feedback questionnaire | 12 months after randomization | Self-questionnaire about how patients in the experimental group feel about the program |
| Static trunck flexors muscle endurance | From randomization, up to 12 months | Shirado-Ito test |
Countries
France
Contacts
Primary ContactAmelie GIBORY
Backup ContactArnaud DE LUCA, MD
Outcome results
None listed