No abstract

The inclusion of health-related quality of life (HRQoL) as an outcome measure in cystic fibrosis (CF) clinical trials can supply important patient-reported information not captured by other endpoints. Both an appropriate HRQoL measure and sound methodology are required in order to draw valid inferences about treatments and HRQoL. This paper provides the current consensus of the HRQoL Outcomes Group. Particular consideration has been given to the appropriateness of measurement scales, the rationale for including specific domains as endpoints, the importance of considering baseline ceiling effects and the difficulties of data interpretation. Guidance is provided on HRQoL measurement in National and European CF clinical trials.

Palliative care is an approach that improves quality of life for patients and their families facing problems associated with a life-threatening illness. Care planning is particularly important in CF, where predicting a time of death is extremely difficult. The patient and family should receive realistic information about health status and further options of care. Particularly important is the explanation that treatment does not stop during the terminal phase of the disease, instead the primary aim is to alleviate unpleasant symptoms. More invasive end of life care is becoming the norm in patients awaiting lung transplantation. Terminal care should be organised in the place chosen by the patient and their family. Ideally terminal care should not end when the patient dies, instead psychological and spiritual support should continue to bereaved families.

Cystic fibrosis increases the risk of developing anxiety and depression in female patients and in mothers. Geographical differences were observed, with higher anxiety and depression in southern Italy for parents, but not for patients. Anxiety and depression levels also depend on clinical status. Pediatr Pulmonol. 2016;51:1311-1319. © 2016 Wiley Periodicals, Inc.

Caregiving burden is a relevant and frequent issue among parents of adolescent patients with cystic fibrosis. We showed that the humanistic and vocational impact of caring for young patients with the disease is striking and demands health-care and welfare supportive actions.