Adult Severe Asthma Patients Treated in France
Conditions
Keywords
Severe asthma, Adult, Respiratory disease
Brief summary
The French Society for Respiratory diseases (SPLF), through its Asthma and Allergy working group (G2A), wishes to set up a national cohort of severe asthma patients to describe this population and the use of step 4 and 5 treatment. This study also meets the demand for post - registration studies required by the Health Authorities for biotherapies and bronchial thermoplasty. Other European or international cohorts of asthmatic patients exist and RAMSES could contribute to data sharing initiatives.
Detailed description
Severe asthma is defined by poorly controlled disease despite treatment with high doses of inhaled corticosteroids, in combination with at least a long acting beta agonist, after optimal management for at least 6 months by an asthma specialist. The diagnosis of severe asthma can only be made after a rigorous evaluation of 6 to 12 months, including the review of diagnosis and treatment, by a pulmonologist, to distinguish difficult asthma from severe asthma. Severe asthma accounts for 3.5% to 5% of all asthma patients, a population estimated at 150 000 patients in France. This disease is responsible for significant health costs related to asthma treatments, comorbidities, hospitalizations and sick days. The management of severe asthma is complex, multidisciplinary and includes drug treatment, but also the management of side effects of treatment, including oral corticosteroid therapy, and comorbidities of asthma. Until 2018, omalizumab,systemic continuous oral corticosteroid therapy or inclusion in a therapeutic trial were the ony possibilities offered to severe asthmatic patients beyond the usually recommended and optimized management. In 2018, mepolizumab and reslizumab have been marketed in France, then benralizumab in 2019. They will probably be followed by dupilumab and bronchial thermoplasty. New initiatives to change the history of the disease are also proposed such as specific immunotherapy or long-term use of macrolides. Respiratory rehabilitation is also part of the treatments to be evaluated in some groups of patients. The description of the use of these treatments, as well as the evaluation of their effectiveness and tolerance in real life conditions is essential. Indeed, their use outside clinical trials raises a number of issues such as the optimal duration of treatment, the maintenance of efficacy over extended periods, the risk and the time to relapse when the treatment is stopped, the possibility to switch from one biotherapy to another in case of insufficient clinical response or to associate them which may prove to be an effective strategy in patients eligible for two biotherapies (allergic and eosinophilic patients). The cost of these treatments is high. The pharmaco-economic evaluation of severe asthma and its management is another important element. For example, no economic evaluation of the use of omalizumab is currently available in France. Lastly, the organization of a care network dedicated to severe asthma is being launched in France (severe asthma centers ), on the model of European countries, in order to improve the diagnosis and management of these patients and to reduce the burden of comorbidities of asthma and steroids side effects.It is therefore essential to set up a clinical research structure that will make it possible to obtain clinical data on this population of severe asthma patients and to evaluate all medical practices in real life conditions.
Interventions
None listed
Sponsors
Assistance Publique - Hôpitaux de Paris
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
\- Patient with severe asthma : Meeting the definition of ATS-ERS: which requires a combination of high dose CSI and B2LDA. Or receiving level 5 support (initiating treatment or already treated) : * Long-term oral corticosteroids (\> 6 months in the year prior to inclusion) * And / or monoclonal antibody * And / or bronchial thermoplasty * And / or other complex costs corresponding to the level 5 treatments * Consultant or hospitalized in one of the centers participating in RAMSES study
Exclusion criteria
* Patient does not accept the recording of his data in the cohort * Patient not benefiting from health insurance coverage * Patient under curatorship or tutorship
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of patients receiving asthma treatment | Previous treatment, collected at baseline | Severe asthma medication (high dose inhaled corticosteroids / B2LDA, Anti IgE antibody, Anti IL5 antibody, daily oral steroids…); Other asthma medications (long acting anticholinergics, montelukast, theophylline, inhaled corticoids); bronchial thermoplasty. |
| Dose of severe asthma treatments | At baseline | Severe asthma medication (high dose inhaled corticosteroids / B2LDA, Anti IgE antibody, Anti IL5 antibody, daily oral steroids…) |
| Duration of asthma treatments | Previous treatment, collected at baseline | Severe asthma medication (high dose inhaled corticosteroids / B2LDA, Anti IgE antibody, Anti IL5 antibody, daily oral steroids…); Other asthma medications (long acting anticholinergics, montelukast, theophylline, inhaled corticoids); bronchial thermoplasty. |
| Other medications | At baseline | Number of patients receiving antidiabetics, Number of patients receiving antidepressants, Number of patients receiving anti-osteoporotic drugs, Number of patients receiving cardiovascular drugs. |
| Asthma-related comorbidities | At baseline | Prevalence of asthma related comorbidities such as chronic rhinosinusitis, nasal polyps, gastroesophageal reflux, obesity, obstructive sleep apnea respiratory infections, … |
| Non-respiratory comorbidities | At baseline | Prevalence of non-respiratory comorbidities such as osteoporosis, diabetes , cardiovascular diseases... |
| Asthma control test (ACT) | 12 previous months, collected at baseline | Patient-reported asthma symptoms and control will be collected via the ACT questionnaire; a 5 items, self-administered survey that is designed to help the patient describe their asthma and how it affects their daily activities. ACT questionnaire is a 5-point scale (for symptoms and activities: 1=all the time to 5= not at all; for asthma control rating: 1=not controlled at all to 5=completely controlled). Asthma is controlled when the total score is \>20. |
| Asthma exacerbations | 12 previous months, collected at baseline | Number of systemic corticosteroids (SC) courses |
| Forced Expiratory Volume in 1 second (FEV1) | 12 previous months, collected at baseline | Mean FEV1, expressed in ml. |
| Number of patients with at least 1 sick leaves due to asthma | 12 previous months, collected at baseline | Patient-reported sick leaves. |
| Number of sick leaves due to asthma | 12 previous months, collected at baseline | Patient-reported sick leaves. |
| Number of patients with long term disease status for asthma | At baseline | — |
| Intensive care unit hospitalization due to asthma exacerbation | During life, collected at baseline | At least one intensive care unit hospitalization for asthma |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| EuroQual 5-Dimension Health Status Questionnaire (EQ-5D) index score and health state assessment | At 6 months | EQ-5D is a standardized instrument for measuring generic health status. The EQ-5D questionnaire has two components: health state description and evaluation. In the description part, health status is measured in terms of five dimensions (5D); mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Mobility dimension asks about the person's walking ability. Self-care dimension asks about the ability to wash or dress by oneself, and usual activities dimension measures performance in work, study, housework, family or leisure activities. Pain/discomfort dimension asks how much pain or discomfort the person has, and anxiety/depression dimension asks how anxious or depressed the person is. The respondents self-rate their level of severity for each dimension using three-level (EQ-5D-3L) scale. In the evaluation part, the respondents evaluate their overall health status using the visual analogue scale (EQ-VAS). |
| Hospital Anxiety and Depression scale | At 6 months | The HADS is a fourteen item scale that generates ordinal data. Seven of the items relate to anxiety and seven relate to depression. Each item on the questionnaire is scored from 0-3 and this means that a person can score between 0 and 21 for either anxiety or depression. |
| Long term adverse Events associated with corticosteroid therapy. | 60 months | Frequency of relevant medical events such as osteoporosis, diabetes, cardiovascular disease. |
| Asthma-related comorbidities | At 6 months | Prevalence of comorbidities such as rhinosinusitis, obesity, gastroesophageal reflux, sleep obstructive apnea syndrome, … |
| Death and cause of death | At 6 months | — |
| Post-procedural adverse Events associated with bronchial thermoplasty | Up to 24 hours following the procedure | Frequency of post-procedure medical events such as hemoptysis, respiratory symptoms, asthma, prolonged hospitalization |
| Long term adverse events associated with bronchial thermoplasty | 60 months | Frequency of long term medical events such as bronchial stenosis. |
| Events of special interest | From treatment initiation to 3 months following treatment initiation | Frequency of special interest events including new onset malignancy, severe infection, anaphylaxis. |
| Blood eosinophil count | At baseline | — |
| Total immunoglobulin E (IgE) | At baseline | — |
| Chest computed tomography scan | At baseline | Dates and description of major findings. |
| Forced Vital Capacity (FVC) | At baseline | — |
| Forced Expiratory Volume in 1 second (FEV1) | At 6 months | — |
| Number of unscheduled healthcare visits or hospitalization | At 6 months | Inpatient hospitalizations due to asthma including intensive care unit, inpatient hospitalization for other reasons, emergency department visits and unscheduled ambulatory visits |
| RV/TLC ratio | At baseline | — |
| Fractional exhaled nitric oxide (FENO) | At baseline | — |
| Costs of severe asthma management | Through study completion, an average of 5 years | Total amount of money, expressed in euros, reimbursed by Social Security ( for treatments, medical visits, radiological or laboratory examinations). |
| FEV1/FVC ratio | At baseline | — |
| Number of patients with unscheduled healthcare visits or hospitalization | At 6 months | Inpatient hospitalizations due to asthma including intensive care unit, inpatient hospitalization for other reasons, emergency department visits and unscheduled ambulatory visits |
| Daily dose of corticosteroids | At 6 months | Inhaled corticosteroids, oral corticosteroids (SC) in long term users |
| Number of patients with at least 1 other asthma medication | At 6 months | Long acting beta 2 agonists, long acting anticholinergics, montelukast, macrolides, Anti IgE monoclonal antibodies, theophylline. |
| Asthma exacerbations | 6 months | Systemic corticosteroids (SC) use or doubling the usual dose or at least 48 hours in SC long term users |
| Number of patients reporting suboptimal treatment adherence | Through study completion, an average of 5 years | Patient-reported adherence to asthma treatment. Suboptimal treatment is defined as less treatment received than expected. |
| Drug survival rate | At 6 month | Proportion of patients still exposed to treatment |
| Time to drug discontinuation | Reported at 6 month | Median time between the start of treatment and its definitive cessation |
| Asthma control test (ACT) | At 6 months | Patient-reported asthma symptoms and control will be collected via the ACT questionnaire; a 5 item, self-administered survey that is designed to help the patient describe their asthma and how it affects their daily activities. ACT questionnaire is a 5-point scale (for symptoms and activities: 1=all the time to 5= not at all; for asthma control rating: 1=not controlled at all to 5=completely controlled). |
| Number of patients with at least 1 sick leaves due to asthma | At 6 months | Patient-reported sick leaves. |
| Number of sick leaves due to asthma | At 6 months | Patient-reported sick leaves. |
| St. George's Respiratory Questionnaire (SGRQ) | At 6 months | The St. George's Respiratory Questionnaire (SGRQ) is a self-reporting questionnaire validated for evaluating quality of life in asthma. The total score is expressed as a percentage, from 0%, no impairment of life quality, to 100%, maximal impairment. |
| Asthma Quality of Life Questionnaire (AQLQ) overall score by Visit | At 6 months | The AQLQ is a 32-item disease-specific questionnaire that has been designed to measure the functional impairments that are most troublesome to adults with asthma. Patients are asked to recall their experiences during the previous 2 weeks and to score each item on a 7-point scale. The overall AQLQ score is the mean response to all 32 questions. |
Countries
France
Contacts
Primary ContactCamille TAILLE, MD,PHD
Backup ContactCandice ESTELLAT, Dr
Outcome results
None listed