1. To evaluate the impact of acute worsening events on daily lives of patients with asthma2. To identify and explain the acute worsening associated behaviours, including the underlying reasons for not consulting a healthcare professional.3. To…
ID
Source
Brief title
Condition
- Bronchial disorders (excl neoplasms)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Not applicable
Secondary outcome
Not applicable
Background summary
Most randomised controlled trials (RCTs) studying the efficacy of a new
treatment for asthma define the primary outcome as occurrence of a severe
exacerbation for which the patient gets systemic corticosteroids prescribed.
However, patient diaries applied in RCTs have shown that it happens frequently
that periods of worsening symptoms are not reported to healthcare professionals
and are therefore not recorded as exacerbations in healthcare systems. The
clinical importance and impact of these unreported acute worsening events
(AWEs) on patients with asthma is largely unknown. Interventions at the
intermediary stage of acute worsening may mitigate progression to severe
exacerbation that requires more intense treatment including hospitalisations
In a post-hoc analysis of RCTs conducted in patients with asthma, various
combinations of predefined threshold values for peak expiratory flow, reliever
medication use, and symptoms were tested to objectively define acute worsening
events. A composite endpoint (CompEx Asthma) was defined as the occurrence of
such an acute worsening event or a severe exacerbation. The performance of this
composite endpoint was tested in 7 trials and at the end of follow-up, the
proportion of patients experiencing a CompEx Asthma event increased almost
3-fold versus severe exacerbations alone. Furthermore, the study showed that
CompEx Asthma can be used as a novel endpoint in RCTs as it consistently
reflected the treatment effect on severe asthma exacerbations. Extending the
definition of an exacerbation to include AWEs will encompass differences in
help-seeking behaviour and increase generalisability across different
healthcare systems. In addition, it will increase event rates and reduce the
required sample size.
For this, it is important to increase knowledge of the clinical importance of
unreported AWE*s and to better understand why patients do not consult a
healthcare professional for worsening of their condition.
Study objective
1. To evaluate the impact of acute worsening events on daily lives of patients
with asthma
2. To identify and explain the acute worsening associated behaviours, including
the underlying reasons for not consulting a healthcare professional.
3. To collect patient-reported outcome (PRO) data for evaluation of
psychometric properties of the CompEx items.
Study design
A qualitative study will be conducted in five countries (The Netherlands,
Australia, Portugal, United Kingdom (UK), United States of America (US)).
Participants will be recruited through pharmacies and healthcare professionals.
After a baseline period of 1 week, they will be followed up for 12 weeks to
monitor the occurrence of an acute worsening event (AWE). A previously
established algorithm (CompEx Asthma) will be used to define these events based
on diary records. Participants who have an AWE will be selected for two
interviews. The initial interview will be held at 3±2 days after the occurrence
of an event. A second interview will be scheduled 4 weeks ± 4 days after the
first interview when most participants are expected to have recovered from the
event. In the case of an AWE, participants will be followed up until the date
of the second interview (for a maximum of 18 weeks), to capture the
participant's state during the second interview.
A topic list and an interview guide will be used as a framework for the
interviews. To avoid influencing the behaviour of participants, they will not
be informed that the AWE was a trigger for the first interview.
Study burden and risks
Participants will twice a day (morning and evening) fill in an online diary and
perform a peak flow measurement. This will take approximately 10 minutes every
time. The peak flow measurement is not painful, but does take effort. We
estimate that 18% of the participants will be invited for two online interviews
of 30 minutes.
There are no risks associated to taking part in this study.
There are no direct benefits for individual participants. However, if the study
indicates that the new clinical outcome based on a change is symptoms performs
equally well as the standard outcome, this could strongly reduce the burden for
future research participants as the novel outcome has a three times higher
incidence.
Francis Crick Avenue 1
Cambridge CB20AA
GB
Francis Crick Avenue 1
Cambridge CB20AA
GB
Listed location countries
Age
Inclusion criteria
>=18 years old
Documented diagnosis of asthma for >=12 months
Documented history of >=1 severe exacerbation in the last 12 months
Symptomatic (Asthma Control Questionnaire-6 score >= 1.5)
Use of inhaled corticosteroid at a stable dose for >=3 months prior to enrolment
BMI 18-32 kg/m2
Exclusion criteria
Recent severe asthma exacerbation (within 6 weeks)
Clinically important other pulmonary disease, including COPD
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL85661.056.23 |