Cystic Fibrosis Transmembrane Conductance Regulator Modulators Decrease Hospitalization Rates

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Investigators assessed rates of hospitalization for acute pancreatitis among patients with cystic fibrosis (CF) receiving CF transmembrane conductance regulator therapy.

For patients with cystic fibrosis (CF), using a CF transmembrane conductance regulator (CFTR) modulator was associated with reduced hospitalizations for acute pancreatitis (AP). These findings, from a retrospective analysis, were published in The American Journal of Gastroenterology.

Data were sourced from the MarketScan database, which includes >32 billion service records from inpatients and outpatients in the United States. Between 2012 and 2018, all patients (N=10,417) with CF were assessed for AP hospitalization on the basis of CFTR modulator use.

Patients did (n=1795) or did not (n=8622) use CFTR modulators. These cohorts were aged mean 20.7 (SD, 12.9) and 22.6 (SD, 16.4) years (P <.0001), 52.8% and 48.9% were boys or men (P =.003), and Charlson Comorbidity Index was 0.4 (SD, 0.7) and 0.7 (SD, 1.3), respectively.

Patients were stratified by pancreas-insufficient CF (PI-CF; n=6658) and pancreas-sufficient CF (PS-CF; n=3759) status, among whom 1516 and 279 used CFTR modulators, respectively. More patients with CFTR modulator use had PI-CF (P <.0001).

There were a total of 240 AP hospitalizations, 31 of which occurred among patients using CFTR modulators. Stratified by pancreas sufficiency, 1.4% of AP admissions occurred among the PS-CF and 5.3% of admissions among the PI-CF groups during CFTR modulator use.

AP hospitalizations occurred at rates of 1.76 and 10.20 per 1000 patient-years among the untreated PI-CF and PS-CF cohorts, respectively. The highest AP hospitalization rate occurred among patients with PS-CF who had discontinued CFTR modulator use (12.93 per 1000 patient-years). The lowest rates of AP hospitalization were observed among the patients with PI-CF who were currently using CFTR modulators (0.34 per 1000 patient-years).

This study may have been limited by defining pancreas sufficiency status by distribution of pancreas enzyme therapy rather than laboratory data.

This study found that patients with CF benefited from CFTR modulator use, regardless of established pancreas sufficiency. These data justify a cost-effectiveness analysis to better assess the feasibility of expanded CFTR modulator use for preventing AP hospitalization among patients with CF.


Ramsey ML, Gokun Y, Sobotka LA, et al. Cystic fibrosis transmembrane conductance regulator modulator use is associated with reduced pancreatitis hospitalizations in patients with cystic fibrosis. Am J Gastroenterol. 2021;116(12):2446–2454. doi:10.14309/ajg.0000000000001527