The cystic fibrosis (CF) care model to which clinicians and people with CF (pwCF) are accustomed has its roots in the era shortly after Dr. Dorothy Andersen's seminal post-mortem descriptions of the pancreatic manifestations of the disease [1] and Dr. Paul di Sant'Agnese's prescient observation of abnormally high sodium and chloride concentrations in the sweat of infants with CF who became markedly dehydrated during a heat wave that gripped New York City in 1948 [2]. In 1964, a visionary group of physicians in Cleveland, Ohio, led by Drs ..read more

Cystic fibrosis (CF) is an autosomal recessive disease that causes dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, resulting in impaired mucus hydration which leads to pulmonary infection and inflammation [1]. People with CF (PwCF) also experience comorbidities in addition to respiratory illnesses, including pancreatitis, CF-related diabetes, liver disease, and malnutrition. Lung transplantation is a therapeutic option for late-stage lung disease and accounts for the majority of transplants reported among PwCF in the United States [2 ..read more

The development of CF transmembrane regulator (CFTR) modulators has significantly advanced the understanding and treatment of CF pathophysiology [1]. In particular, the triple-combination therapy elexacaftor/tezacaftor/ivacaftor (ETI) has seen rapid integration into treatment for CF individuals with at least one F508del mutation [2] and has been shown to improve lung function measured by the forced expiratory volume in one second (FEV1%) derived from spirometry. However, with children having milder disease in the era of such highly effective modulators, FEV1% may be equivocal between healthy a ..read more

Costa and colleagues [1] recently published an analysis of the time from submission and approval for CF disease modulating drugs by the Federal Drug Agency (FDA) in the United States (US) and European Medicines Agency (EMA) in the European Union (EU) demonstrating an average delay of 103 days for submission and 267 days for approval between the EMA as compared to the FDA and the time for initial approval for a CFTR modulating drug was 167 days in the US and 346 days in the EU ..read more

People with CF (PWCF) with advanced cystic fibrosis lung disease (ACFLD) remain at risk for disease progression and potential lung transplantation (LT) even in the highly effective modulator therapy era. LT rejection has been attributed to chronic lung allograft dysfunction (CLAD), which has been conflated inaccurately with “gastroesophageal reflux disease” (GERD). As gastrointestinal (GI) interventions may slow lung function decline [1], the study of their application in advanced lung disease (ALD), ACFLD, and LT is an urgent priority ..read more

People with CF (pwCF) are living longer and having fewer CF clinic visits and hospitalizations [1,2]. An increasing percentage of patients are prescribed cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies, particularly elexacaftor-tezacaftor-ivacaftor (ETI), which has been associated with marked improvements in lung function and reductions in pulmonary exacerbations, culminating in an expected increase in survival. The overall symptom burden is less, and the quality of life has improved for many but not all pwCF, as some are not able to benefit from the modulators ..read more

“My health care team and I can be very honest with each other. I always feel like they really care about me and the outcome of whatever problem is at hand. It is hard trusting someone with your life expectancy. When new people are thrown at you it can be frustrating…” (Adult with cystic fibrosis (CF), age 25–34 ..read more

We have reached a point in our history of cystic fibrosis (CF) that we have long dreamed of – that the majority of those we serve, persons and families living with CF, feel better, suffer fewer complications of their condition, and can expect to live longer, much longer (Fig. 1). They can anticipate a healthier childhood and plan for aspects of adulthood (relationships, families, and meaningful careers) that previously felt out of reach. Historically, people with CF (pwCF) experienced significant reductions in life expectancy primarily due to progressive pulmonary disease [1,2 ..read more

Specialized CF care centers have long been staffed with interdisciplinary teams collaborating to provide comprehensive care for pwCF. This approach has improved health outcomes for complex chronic conditions (e.g., sickle cell disease, HIV, and cancer) through specialized and coordinated care [1,2], and has been transformative for CF, significantly increasing survival rates over time [3]. As pwCF manifest multi-system complications related to CF, including pulmonary and nutritional issues, the CF team members historically included a physician (typically a pulmonologist), program coordinator, n ..read more

One of today's greatest challenges in the field of Cystic Fibrosis (CF) is to treat all people with CF (pwCF) to the level of benefit reached by the highly efficient CFTR modulator therapies (HEMT ..read more