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.