C61-08 Evaluating Incidence of Ocular Complications in People With Cystic Fibrosis on Highly Effective Modulator Therapy

Rationale Cystic fibrosis (CF) is an autosomal recessive disorder causing chronic, progressive lung disease secondary to mutations in the CFTR gene. This creates sticky, inspissated mucus in the airways and pancreatic exocrine glands leading to bronchiectasis/recurrent pneumonia and pancreatic insufficiency respectively. Additionally, CFTR is expressed in other tissues including skin, sinonasal tissue, and ocular tissue. Highly effective modulator therapy (HEMT) refers to a class of medications that improve the function of the abnormal CFTR protein in people with CF (pwCF). There have been increasing concerns for a potential association between HEMT and hordeolum formation. To explore this possible association, we constructed a survey distributed to national pediatric and adult CF centers to determine if these ocular complications in pwCF on HEMT have been observed at other centers. Methods A RedCap survey was developed to assess provider knowledge of ocular complications associated with HEMT and their incidence at each center. This study was approved through our institutional review board (IRB). Following approval, the survey was distributed via contact emails for adult and pediatric CF Center providers listed on myCFF.org. One provider from each center was instructed to fill out the survey once. Results were then analyzed using basic statistical methods. Results Forty-seven responses were collected. Twenty-five (53%) of the respondents were aware of a possible association of hordeola with HEMT. Twenty-two reported seeing recurrent/acute hordeola, 11 saw chronic styes, and 21 respondents did not observe any ocular complications. Collectively, there were 49 cases reported of acute/recurrent/chronic hordeola in pwCF on HEMT, most on elexacaftor/tezecaftor/ivacaftor (47 cases or 96%). The majority (32 cases or 65%) were in pwCF 18 years and younger, with most children being between 7-10 years of age (14 cases or 28.5%). Of the centers that saw ocular complications, 10 respondents reported using oral antibiotics, and four reported cases needing emergency room evaluation. Conclusion CFTR protein is expressed in ocular tissues, including the lacrimal tissue and the Meibomian glands. While a few cases of acute and chronic hordeola in pwCF on HEMT have been published, our survey identified 49 potential cases, highlighting the need for further investigation. It also revealed limited provider awareness of a potential link between HEMT and hordeola formation. Prospective observational studies tracking these complications may reveal risk factors such as age, sex, or genotype. Going forward, we advocate for screening and surveilling ocular complications in pwCF on HEMT.