Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial.

The cystic fibrosis (CF) modulator drug, elexacaftor/tezacaftor/ivacaftor (ETI), proved highly effective in controlled clinical trials for individuals with at least one F508del allele, which occurs in at least 85% of people with CF. PROMISE is a postapproval study to understand the broad effects of ETI through 30 months' clinical use in a more diverse U.S.

Testing the effects of combining azithromycin with inhaled tobramycin for in cystic fibrosis: a randomised, controlled clinical trial.

Rationale: Inhaled tobramycin and oral azithromycin are common chronic therapies in people with cystic fibrosis and airway infection. Some studies have shown that azithromycin can reduce the ability of tobramycin to kill . This trial was done to test the effects of combining azithromycin with inhaled tobramycin on clinical and microbiological outcomes in people already using inhaled tobramycin.

Pediatric Primary Care Relationships and Non-Urgent Emergency Department Use in Children.

Objective: Non-urgent emergency department (ED) use contributes to healthcare costs and disrupts continuity of care. Factors influencing patient/guardian decision-making in non-urgent situations are poorly understood. We sought to determine the association of patient/guardian - primary care provider (PCP) relationship with non-urgent ED usage and explore related factors.

PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy.

Highly effective CFTR modulator drug therapy is increasingly available to those with cystic fibrosis. Multiple observational research studies are now being conducted to better understand the impacts of this important therapeutic milestone on long-term outcomes, patient care needs, and future research priorities. PROMISE is a large, multi-disciplinary academic study focused on the broad impacts of starting elexacaftor/tezacaftor/ivacaftor in the US population age 6 years and older.