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The development of CFTR (cystic fibrosis conductance transmembrane regulator) modulators for people with cystic fibrosis (pwCF) and eligible for these treatments is a true therapeutic revolution. The major beneficial effect of CFTR modulators (CFTRm) on pulmonary function and the reduction of pulmonary exacerbations should have a considerable impact on the quality of life and patient's life expectancy. Data on the impact of CFTRm on glucose tolerance abnormalities are still very fragmentary. The investigators can think that their use, earlier and earlier in the history of the disease, will transform the evolutionary trajectories of patients on the respiratory, nutritional and metabolic levels.
Diabetes represents a major challenge in the management of pwCF because it is a factor in morbidity and mortality at all stages of the disease, from children to patients with terminal respiratory failure requiring lung transplantation. Early abnormalities in glucose tolerance observed in childhood, before the stage of diabetes, are also associated with poor pulmonary and nutritional outcomes. Experimental data suggest a positive effect of CFTRm on insulin secretion. However, investigators do not currently know the impact of CFTRm in patients with very early glucose disorders or at the stage of diabetes treated with insulin. Recently continuous glucose measurement (CGM) devices represent very effective tools for assessing abnormalities in glucose tolerance before the stage of diabetes and for monitoring patients treated with insulin.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Adult patients with cystic fibrosis |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| elexacaftor-tezacaftor-ivacaftor treatment | Drug | Effect of one year elexacaftor-tezacaftor-ivacaftor treatment on glucose tolerance abnormalities in adult patients with cystic fibrosis |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluate time in range of blood glucose variation >140 mg/dl during 2 weeks in a cohort of adult patients treated for one year with elexacaftor-tezacaftor-ivacaftor | time in range of blood glucose variation >140 mg/dl one year before and one year after the begining of elexacaftor-tezacaftor-ivacaftor treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluate other CGM parameters : time in range of blood glucose >180mg/dl ; >140mg/dl ; <70mg/dl ; 70-180 mg/dl ; 70-140mg/dl | One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment | |
| Marker of metabolic status: HbA1c (mmol/l) |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with cystic fibrosis aged over 18, followed in cystic fibrosis ambulatory care services of 18 french centers
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hopitaux Universitaires de Strasbourg | Strasbourg | 67000 | France |
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment |
| Marker of metabolic status: HbA1c (%) | One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment |
| Marker of metabolic status: C-Peptid (µg/L) | One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment |
| Marker of metabolic status: C-Peptid (nmol/L) | One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment |
| Markers of nutritional status: Evaluation of weight monitoring (kg) | One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment |
| Markers of nutritional status: Body Mass Index (BMI) | One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment |
| Diabetes treatment (assess insulin needs) : mean glucose value per day (mg/dl) | One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment |
| Assessment of respiratory function parameters : number of IV antibiotics | One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment |
| Number patients with severe hypoglycaemia and serious adverse events | One year before and one year after the start of elexacaftor-tezacaftor-ivacaftor treatment |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |