Pancreas · 2025
Gastrointestinal Burden in Patients With Pancreatic Insufficient Cystic Fibrosis Before and After Elexacaftor/Tezacaftor/Ivacaftor Use.
СтатьяМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
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Abi-Aad Sasha-Jane, Trocchia Carolena, Mosha Maua, Mark Jacob A, Leung Daniel H, Khalaf Racha
Аннотация
BACKGROUND: The advent of highly effective modulator therapies (HEMT), namely elexacaftor, tezacaftor, and ivacaftor (ETI), has resulted in substantial improvements in lung function, growth, and quality of life for people with cystic fibrosis (PwCF). However, the understanding of the impact of ETI on gastrointestinal (GI) disease burden is evolving. This study aims to describe and compare the prevalence of GI manifestations, prescribed GI medications, and GI procedures between 2 time periods, pre-ETI and post-ETI approval, in children with CF (CwCF) and adolescents with CF.
METHODS: This is a retrospective cohort study utilizing TriNetX, a multicenter database. The study includes patients between 6 to 21 years old with ICD-10 diagnostic codes for CF and a prescription for pancreatic enzyme replacement therapy (PERT) to match disease severity. We included 4 years before, and 4 years after the release of the ETI combination therapy.
RESULTS: When comparing CwCF taking PERT, on or off ETI, the prevalence of diagnostic codes for chronic pancreatitis, constipation, unspecified noninfective gastroenteritis, and colitis significantly decreased after ETI use (all P < 0.0001). The prevalence of other luminal disorders, liver disorders, and acute pancreatitis did not differ between the 2 groups. The prevalence of prescribed medications, including mucolytics, vitamins, PPI, and antidiarrheal, was similar for both groups except for a reduction in prescribed laxatives ( P -value = 0.0001). The prevalence of GI procedures was also similar in both groups.
CONCLUSIONS: The reduction in constipation and noninfective gastroenteritis and colitis is important as GI symptoms are linked to the quality of life of CwCF. There remains a great clinical need to evaluate the effects of ETI on GI disorders, especially as the age of initial use of this therapy decreases and the duration of use increases.
METHODS: This is a retrospective cohort study utilizing TriNetX, a multicenter database. The study includes patients between 6 to 21 years old with ICD-10 diagnostic codes for CF and a prescription for pancreatic enzyme replacement therapy (PERT) to match disease severity. We included 4 years before, and 4 years after the release of the ETI combination therapy.
RESULTS: When comparing CwCF taking PERT, on or off ETI, the prevalence of diagnostic codes for chronic pancreatitis, constipation, unspecified noninfective gastroenteritis, and colitis significantly decreased after ETI use (all P < 0.0001). The prevalence of other luminal disorders, liver disorders, and acute pancreatitis did not differ between the 2 groups. The prevalence of prescribed medications, including mucolytics, vitamins, PPI, and antidiarrheal, was similar for both groups except for a reduction in prescribed laxatives ( P -value = 0.0001). The prevalence of GI procedures was also similar in both groups.
CONCLUSIONS: The reduction in constipation and noninfective gastroenteritis and colitis is important as GI symptoms are linked to the quality of life of CwCF. There remains a great clinical need to evaluate the effects of ETI on GI disorders, especially as the age of initial use of this therapy decreases and the duration of use increases.