Unmet needs in cystic fibrosis: the next steps in improving outcomes

Abstract

Cystic fibrosis (CF) outcomes and survival have improved over the last century primarily due to advancements in antibiotics, nutritional, and pulmonary therapies. Reviewed here are the significant unmet needs that exist for individuals with CF. Areas covered: With the recent development of medications that address the underlying defect in the CF protein, there is hope that there will be continued improvement in CF outcomes. However, there remains a need to prevent or stop progression of CF-related complications, as the CF protein is important to several body systems. As end stage lung disease is the primary cause of mortality in CF, a need exists for advancements in pulmonary therapies to reduce time burden, identification of best practices for the treatment of pulmonary exacerbations, further development of anti-infective and anti-inflammatory therapies, and appropriately timed referral for lung transplantation at end-stage lung disease. Extra-pulmonary complications are increasingly recognized and better understanding of such problems as CF related liver disease is needed. Expert commentary: While CFTR modulators are available for the majority of CF patients, there remains a need for effective therapies to address infection, inflammation, irreversible lung disease, and extrapulmonary complications of CF.

Keywords: Cystic fibrosis; cystic fibrosis-related liver disease; inflammation; ivacaftor; lumacaftor; lung transplant; pancreatic insufficiency; pulmonary exacerbations; tezacaftor; unmet needs.

Figure 1.

Classes of CFTR modulators. (Abbreviation: cystic fibrosis transmembrane conductance regulator (CFTR)). Reprinted from The Lancent Respiratory Medicine, Vol 1, Michael P Boyle and Kris De Boeck, A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect, 158–163., Copyright (2013), with permission from Elsevier.