Cystic fibrosis (CF) is a complex, chronic, multisystem disease for which there is currently no cure. Nonetheless, advances in management have led to dramatic improvements in patient survival. With this development, new issues have arisen for CF patients and their care providers, including an increased symptom burden and increased frequency of co-morbidities as patients reach older ages, leading to the need for a highly complicated and time-consuming regimen of treatments. Such high symptom and treatment burden often leads to non-adherence and low levels of competence with administration of therapy, both of which may have detrimental impacts on CF outcomes. Optimal management is also hindered by other patient-related factors, including inadequacies in disease education which may lead to issues with self-management. This is particularly important during the transition from parent-directed therapy to independent self-management that occurs during adolescence and early adulthood. Clinicians are also faced with a considerable challenge when selecting interventions for individual patients; although the paradigm of aggressive care necessitates a wide range of therapies, there is a limited evidence base with which to compare available therapeutic regimens. Novel pharmacological agents are being developed to target the underlying cause of CF, while non-pharmacological interventions aim to improve competence and maximize adherence and health outcomes. Comparative effectiveness research is needed to simplify management and facilitate the implementation of appropriate treatment strategies.
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