TOPIC 3: Definition of outcomes indicative of clinical benefits
Statement 4: The main outcomes to be evaluated in
order to assess the beneficial effects of mucoactive therapy in patients
with CF include pulmonary function, frequency of pulmonary exacerbations
and quality of life.The Italian experts in CF, in agreement with the most recent guidelines
on the management of CF patients, suggest to assess respiratory
function, frequency of pulmonary exacerbations and quality of life which
represent the main outcomes for establishing the clinical effectiveness
of mucoactive therapies (Figure 2 )22,23. In
final consensus meeting, there was unanimous response to this
statement.
The NICE guidelines indicate that inflammation markers, the need for
antibiotics for exacerbations and adverse events should also be
considered22.
Pulmonary exacerbations are critical events throughout the lifetime of
CF patients and may not be fully reversible24.
Frequent exacerbations are associated with accelerated decline in lung
function25. Poor lung function and pulmonary
exacerbations in the past 6 months have been related to poor health
related quality of life (HRQL)25-27. The Cystic
Fibrosis Questionnaire-Revised (CFQ-R) is a validated patient reported
outcome measure of HRQL specifically designed for patients with
CF28,29. This disease-specific instrument may be
utilized in clinical trials to assess the effects of new therapies, to
document the progression of disease, and to inform clinical
practice28.