Managing treatment complexity in cystic fibrosis: Challenges and Opportunities

Abstract

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.

Adherence Determinants in Cystic Fibrosis: Cluster Analysis of Parental Psychosocial, Religious, and/or Spiritual Factors

“…Parental cystic fibrosis (CF) treatment adherence is variable, which is partly due to the complex and time-consuming daily treatment regimen (1,2). For most patients, the current accepted CF treatment regimen consists of a minimum of airway clearance therapy (ACT), chronic use of oral and nebulized medications (NMs), and pancreatic enzyme replacement with intermittent need for additional oral, nebulized, and occasional intravenous medications during acute illness.…”

New and Emerging Treatments for Cystic Fibrosis

“…CF therapy is currently characterised by poly-pharmacy, but as our armamentarium increases, the ability to individualise, prioritise and ration therapy becomes more difficult. This results in increasing treatment complexity and burden for patients, which in turn risks failure of therapy due to non-adherence [92][93][94]. Effective strategies at improving adherence to maintenance medication remain as central a challenge in CF as development of novel medication, with reports indicating that even the most evidencebased treatments may not be taken as prescribed [95][96][97].…”

Lifestyle treatments in cystic fibrosis: The NHS should pay

“…This can take up a significant proportion of the two or more hours of daily treatment activity reported by patients. 3 An increase in the number of nebulised therapies and an increase in airway clearance time are not surprisingly associated with higher perceived treatment burden, which in turn has to be balanced against career and family responsibilities. 4 It is remarkable that 70.1% of patients over 16 years are now either in employment or studying, which itself is associated with a better quality of life and improved personal, social and financial well-being.…”