Abstract
CFTR Modulator Therapy Reduces Acute Pancreatitis Episodes in Children with CFTR Variants: Report from INSPPIRE-2 Cohort
Pancreatology : official journal of the International Association of Pancreatology (IAP) ... [et al.], Vol.25(7), pp.1225-1226
11/2025
DOI: 10.1016/j.pan.2025.07.015
Abstract
Introduction: Cystic fibrosis transmembrane regulator (CFTR) modulator therapies have been shown to reduce acute pancreatitis (AP) episodes in people with pancreatic sufficient cystic fibrosis (CF). CFTR variants are present in 20-30% of children and adults with acute recurrent pancreatitis (ARP) or chronic pancreatitis (CP). The impact of CFTR modulators on AP episodes in CFTR variant carriers with ARP or CP is unknown. We hypothesize that highly effective modulator therapy (HEMT), elexacaftor/tezacaftor/ivacaftor (ETI) or ivacaftor alone, will decrease the frequency of AP episodes in children with ARP or CP who are CFTR variant carriers.
Methods: This was a multi-center retrospective cohort study of children with ARP or CP enrolled in the INternational Study Group of Pediatric Pancreatitis: In search for a cuRE-2 (INSPPIRE-2) cohort. Inclusion criteria were at least one CFTR variant, without a diagnosis of CF and HEMT initiation during the data collection period... The number of AP episodes before and while on HEMT were compared using incidence rate, accounting for different person-years of follow-up.
Results: Of the 1,025 INSPPIRE-2 patients, 11 subjects on HEMT were CFTR variant carriers; 73% male, 73% had ARP, and 27% had CP. 82% had one CF causing variant: 73% were heterozygous F508del, and 9% were heterozygous G551D. The remaining CFTR polymorphisms were variants of unknown significance or non-CF causing. 73% were on ETI and 27% on ivacaftor. There was significant (90%) reduction in AP incidence density pre-CFTR modulator vs on-CFTR modulator (1.17 vs. 0.18, p < 0.001).
Conclusions: HEMT significantly decreased the number of AP episodes in children with ARP or CP that were CFTR variant carriers., This observation was found with multiple types of CFTR variants. Given the high prevalence of CFTR variants in pediatric ARP or CP patients, this suggests the potential to use HEMT for reduction of AP episodes in this patient population. Future multicentered prospective studies on the efficacy of HEMT for the treatment of AP in patients with CFTR variants are warranted.
Details
- Title: Subtitle
- CFTR Modulator Therapy Reduces Acute Pancreatitis Episodes in Children with CFTR Variants: Report from INSPPIRE-2 Cohort
- Creators
- Michael WilschanskiGila GinzburgAnkur ChughMark JacobNicholas AntosTanja GonskaChee OoiGretchen CressMaisam Abu-El-HaijaReuven Zev CohenElissa DownsDouglas FishmanCheryl GariepyMathew GieferAmit GroverDouglas LinbadQuin LiuAsim MaqboolBrian McFerronMegha MehtaVeronique MorinvilleKenneth NgRobert Adam NoelEmily PeritoZachary SellersFuchenchu WangFeng TianYing YuanMark LoweAliye Uc
- Resource Type
- Abstract
- Publication Details
- Pancreatology : official journal of the International Association of Pancreatology (IAP) ... [et al.], Vol.25(7), pp.1225-1226
- DOI
- 10.1016/j.pan.2025.07.015
- ISSN
- 1424-3903
- Language
- English
- Date published
- 11/2025
- Academic Unit
- Stead Family Department of Pediatrics; Gastroenterology, Hepatology, Pancreatology, and Nutrition
- Record Identifier
- 9985024149202771
Metrics
1 Record Views