Real Life Experience Survey of Dupilumab in the Netherlands
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 120
- 试验地点
- 1
- 主要终点
- Annual exacerbation rate
研究概览
简要总结
Rationale: Dupilumab has proven to be effective and safe in several large randomized controlled trials. However, study populations in RCT's represent only a small proportion of severe asthma patients treated in real life circumstances. Therefore, real-life studies provide data complementary to RCT's derived from a more diverse and heterogeneous group of patients. This study is unique in that it represents a large population of European patients that are not represented in the Global Registry.
Objective: To collect and analyse efficacy and safety data in severe asthma patients treated with dupilumab in a real-life setting.
Study design: Single center, retrospective cohort analysis of severe asthma patients treated with dupilumab, as decided by the treating physician.
Setting: Severe Asthma Expert Center Franciscus Gasthuis & Vlietland, Rotterdam Study population: All patients that started with dupilumab from jan 2019 (early access program) until June 2020 are eligible for the study. They should be 18 years or older, with severe T2 high asthma and treated with at least one dose of dupilumab.
Main study parameters/endpoints:
- Primary endpoint: To assess changes in annual exacerbation rate between baseline and after 12 months treatment in severe asthma patients treated with dupilumab.
- Secondary endpoints: To collect data on ACQ, OCS use, FEV1, treatment response rate, influence of co-morbidities and safety.
Methods:
Exacerbation history and patient characterization will be done at baseline. We aim to include patients in the analysis that had at least one administration of dupilumab (intention-to-treat). Treatment response evaluation is done at 12 months. Final evaluation of efficacy and safety is done at 12 months of treatment. We aim to enroll at least 120 patients in the study. Data collection will be done in Castor Electronic Data Capture. Of the 120 patients scheduled for inclusion, all have already started treatment and data will be collected retrospectively at the predefined time points. Patient data will be handled confidentially.
Nature and extent of the burden and risks associated with participation, benefit and group relatedness: Since this is a retrospective cohort analysis there are no extra visits, interventions or procedures needed. Therefore, there is no increased burden or risk for the patients involved.
详细描述
- INTRODUCTION AND RATIONALE
Background:
Severe asthma is a rare condition affecting approximately 5% of all asthma patients. Patients experience daily symptoms and often have frequent exacerbations despite the use of high dose ICS and LABA. Influencing factors such as co-morbidity and non-adherence to therapy are optimized. Nevertheless, they continue having uncontrolled asthma or can only be controlled with systemic corticosteroids.
Dupilumab is a monoclonal anti-IL-4Rα antibody developed for the treatment of severe asthma. It effectively targets both the IL-4 and IL-13 pathway, resulting in reduction of asthma exacerbations and improvement of asthma control in patients with T2-high asthma. T2-inflammation is defined by the presence of eosinophils in sputum or blood, clinically relevant allergic sensitization and/or increased fraction of exhaled nitric oxide (FeNO). This type of inflammation is present in 60-70% of severe asthma patients. Severe asthma is a heterogeneous group of asthma phenotypes which are not all represented well in previously performed clinical trials. Therefore, it is of importance to conduct real-life studies which include all kind of patients regardless of co-morbidity, environmental exposures, previous treatment with biologicals or ethnical background. These studies provide the necessary data that complement to those obtained by RCT's.
To date, little is known about the response rate, efficacy, and safety of dupilumab in daily practice. To our knowledge, this study will be the largest study to date to assess response rate, efficacy as well as safety in patients with severe T2-high asthma who are treated with at least one dose of dupilumab. The investigators expect 80% of the patients to respond to dupilumab. This is in line with the French cohort of dupilumab in real life published bij Dupin et al. Furthermore, half of the patients will be on chronic OCS treatment, these patients are expected to respond well, even so patients with nasal polyposis and atopic eczema. The investigators expect a reduction in mean ACQ of 0.8 point (MCID = -0,5) and in asthma-exacerbations of at least 70% compared to baseline (MCID = -30%). This would be in line with previous real life biological studies that the investigators performed in and the French cohort. As for post-FEV1 the investigators expect an increase of 10% predicted during the study period. The investigators expect 40% to be super-responders.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All patients that started with dupilumab from Jan 2019 (early access program) until June 2020 are eligible for the study. They should be 18 years or older, with severe T2 high asthma and treated with at least one dose of dupilumab. All included patients should provide written informed consent.
排除标准
- •Patients with COPD (Chronic Obstructive Pulmonary Disease) or EGPA (Eosinophilic Granulomatosis with PolyAngiitis) will be excluded.
- •Also, patients that had dupilumab prescribed for other reasons (nasal polyposis or atopic dermatitis) will be excluded as well.
研究组 & 干预措施
Severe T2 high asthma
Included patients will have severe type 2 high asthma, are eligible for dupilumab conform EU label, minimally treated with high dose ICS and LABA and have:
- blood eosinophils 150 - 1500/mm2 OR
- FeNO >20 ppb OR
- clinical significant inhalation allergy (+ sIgE positive) OR
- OCS dependency
干预措施: Dupilumab (Drug)
结局指标
主要结局
Annual exacerbation rate
时间窗: 12 months before and after start medication
To assess changes in annual exacerbation rate prior to and after 12 months treatment in severe asthma patients treated with dupilumab.
次要结局
- Change in lung function(12 months before and after start medication)
- Change in asthma control(12 months before and after start medication)
研究者
Gert-Jan Braunstahl
Principal Investigator
Franciscus Gasthuis