Translational Research and Clinical Applications in the Management of Cystic Fibrosis

Cystic fibrosis (CF) is a life-shortening and recessive genetic disorder that affects approximately 1 in 2500 live births worldwide. The disorder affects the functions of multiple organs including the lungs, pancreas, liver, and reproductive system and requires adherence to a time-consuming and burdensome treatment regimen. This chapter reviews literature regarding adherence to this treatment regimen, the role of the family in treatment adherence, the impact of CF on family functioning and psychological health, the effect of infection control guidelines on CF patient social support and functioning, and the processes associated with the transition from pediatric to adult care. Empirically evaluated interventions targeting each of these challenges are discussed. Reviewed literature points to the importance of: 1) considering the changing needs of individuals with CF in light of developments in medical practices and policy, 2) the need for supportive interventions for families and individuals with CF to improve family functioning, 3) treatment adherence and mental health, and 4) the necessity of programs that facilitate the transition of treatment responsibility from parents to their children with CF.