Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults

Study Overview
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Brief Summary
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The aim of this project is to evaluate the psychological reshuffle induced by ORKAMBI. The particular focus of this study is the consequence of its introduction on anxiety, depression, quality of life and adherence to all cystic fibrosis (CF) treatment. To answer this question investigators will monitor the psychological function of CF adolescents and young adults treated with ORKAMBI and compare them to CF adolescents and young adults not treated with ORKAMBI.

Detailed Description
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Introduction of KALYDECO then ORKAMBI, as a personalized medicine in the therapeutic strategy of Cystic Fibrosis (CF) may deeply modify the prognosis of patients with CF. These approaches realize the dream of short-circuiting the molecular mechanism of the disease and therefore opens the perspective of an extended and functional survival. It suddenly offers the possibility of a completely new future to the patient and his family, and therefore must deeply impact on the psychological function of all involved. Moreover, those new therapies may also modify the adherence to CF basic treatment, as it brings a new feeling of good health and the hope of a longer life. For that reason there is a need to investigate modifications of the psychological state of mind induced by the treatment with ORKAMBI in CF adolescents and young adults. This project aims to understand the psychological impact and to study the consequences of the introduction of ORKAMBI on adherence to CF treatment, anxiety, depression and quality of life. To answer this question, investigators will evaluate the psychological function of adolescents and young adults, aged 12 to 20 years, with CF treated with ORKAMBI, and compare it to other adolescents and young CF adults not treated with ORKAMBI. This is a cohort prospective study, with a 1 year follow-up.

Official Title
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Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Behavioral: Questionnaires


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Treated group: Patients with proven CF (sweat-test > 60 mEq, 2 DF508 CF causing mutations, 12 to 20 years, and treated with ORKAMBI Control group: Patients not carrying 2 DF508 CF causing mutations, not treated with ORKAMBI, and not carrying the G551D, G178R, S549N, S549R, G551S, G1244E,S1251N, S1255P or G1349D mutation or treated with Kalydeco Exclusion Criteria: Transplanted patients Patients younger than 12 years Patients older than 20 years

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Treated group<br>Patients with proven CF (sweat-test > 60 mEq, 2 DF508 CF causing mutations, 12 to 20 years, and treated with ORKAMBI) | Behavioral: Questionnaires<br>* Answering 4 questionnaires : anxiety, depression, quality of life and adherence<br>|
| Control group<br>Patients not carrying 2 DF508 CF causing mutations, not treated with ORKAMBI, and not carrying the G551D, G178R, S549N, S549R, G551S, G1244E,S1251N, S1255P or G1349D mutation or treated with Kalydeco | Behavioral: Questionnaires<br>* Answering 4 questionnaires : anxiety, depression, quality of life and adherence<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Score of Generalized Anxiety Disorder-7 questionnaire (GAD-7) | to assess the impact on Anxiety | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Score of Patient Health Questionnaire-9 (PHQ-9 ) | To assess the impact on Depression | 24 months | 
| Scores of Cystic Fibrosis Questionnaire 14+ (CFQ 14+) | Disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being and symptoms (Quality of Life) | 24 months | 
| GIRERD Scale | The 6 items are assessed using a YES/NO answer .The scale was validated in patients with chronic disease to assess medication adherence | 24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cystic Fibrosis, Orkambi, Adherence, Adolescent, Anxiety, Depression, Psychology