Pediatric pulmonology · 2026
Exocrine Pancreatic Function in Pediatric Patients After Two Years of Triple CFTR Modulator Therapy: Is It Possible to Restore Pancreatic Function?
СтатьяНаблюдательное исследованиеМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Cárdenes C M López, Santamaría S Vicente, Romero R García, González A Tabares, Galindo C Gascón, Castro E Blitz, Quaresma J, Tirado A Morales, García M Garriga, Martínez J R Gutiérrez, Merino M Álvarez, Rubio E Torcuato, Pérez P Ortiz, Peña-Quintana L, Reyes-Domínguez A, Nicolás S Sierra San, Hurtado M Murray, Castillo R Del Brio, Mercedes M De Los Santos, Volpe C García, Beltrán M Álvarez, Cavia S Mesonero, Torne C Viñas, Jiménez D González, y Grupo de trabajo de FQ y páncreas de la SEGHNP
Аннотация
BACKGROUND: Improvement in exocrine pancreatic function in people with Cystic fibrosis (PwCF) after ivacaftor and lumacaftor/ivacaftor has been documented. Data on triple therapy with elexacaftor/tezacaftor/ivacaftor (ETI) are limited.
METHODS: In this multicenter, prospective, observational study, we reviewed changes in fecal elastase-1 (FE-1) in the pediatric population with exocrine pancreatic insufficiency (EPI) after 2 years of ETI. Patients were classified according to FE-1: mild-moderate EPI as 100-199 μg/g and severe EPI as <100 μg/g. Demographic, clinical, anthropometric, and body composition data were collected. Body composition was measured using bioelectrical impedance analysis.
RESULTS: Fifty-three children were recruited (58.5% males). The median age was 12 years [IQR: 9.0-14.5], and 56.6% were homozygous for F508del. Values of FE-1 below fifteen were designated with a 0. At baseline, the median FE-1 was 0.00 [IQR: 0.00-0.00]. We observed a slight yet statistically significant improvement in FE-1 values 2 years after ETI (Wilcoxon test, p < 0.05). We classified patients into three groups based on FE-1 values. At baseline, 98% were in the severe EPI group (FE-1 < 100 μg/g), the remaining 2% were in the mild-moderate group (FE-1 100-199 μg/g). Two years after triple therapy, six (11.4%) children showed a change in FE-1 to at least 100 μg/g from the baseline. Three (5.7%) had a change in FE-1 ≥ 200 μg/g from baseline, and the other three (5.7%) had a change in FE-1 between 100 and 199 μg/g. A significant reduction in daily lipase intake was observed (6346 vs. 5703 L/kg/day; p < 0.05). BMI Z-score improved over 2 years (-0.55 vs. -0.38; p < 0.05). Body composition data were available for 27 patients, showing increased fat mass and fat-free mass after ETI therapy. No differences in body composition were found when stratified by EPI groups.
CONCLUSION: CFTR modulators have improved pulmonary function and anthropometric measures in PwCF. Based on these findings, CFTR modulators may also enhance exocrine pancreatic function in pediatric patients.
METHODS: In this multicenter, prospective, observational study, we reviewed changes in fecal elastase-1 (FE-1) in the pediatric population with exocrine pancreatic insufficiency (EPI) after 2 years of ETI. Patients were classified according to FE-1: mild-moderate EPI as 100-199 μg/g and severe EPI as <100 μg/g. Demographic, clinical, anthropometric, and body composition data were collected. Body composition was measured using bioelectrical impedance analysis.
RESULTS: Fifty-three children were recruited (58.5% males). The median age was 12 years [IQR: 9.0-14.5], and 56.6% were homozygous for F508del. Values of FE-1 below fifteen were designated with a 0. At baseline, the median FE-1 was 0.00 [IQR: 0.00-0.00]. We observed a slight yet statistically significant improvement in FE-1 values 2 years after ETI (Wilcoxon test, p < 0.05). We classified patients into three groups based on FE-1 values. At baseline, 98% were in the severe EPI group (FE-1 < 100 μg/g), the remaining 2% were in the mild-moderate group (FE-1 100-199 μg/g). Two years after triple therapy, six (11.4%) children showed a change in FE-1 to at least 100 μg/g from the baseline. Three (5.7%) had a change in FE-1 ≥ 200 μg/g from baseline, and the other three (5.7%) had a change in FE-1 between 100 and 199 μg/g. A significant reduction in daily lipase intake was observed (6346 vs. 5703 L/kg/day; p < 0.05). BMI Z-score improved over 2 years (-0.55 vs. -0.38; p < 0.05). Body composition data were available for 27 patients, showing increased fat mass and fat-free mass after ETI therapy. No differences in body composition were found when stratified by EPI groups.
CONCLUSION: CFTR modulators have improved pulmonary function and anthropometric measures in PwCF. Based on these findings, CFTR modulators may also enhance exocrine pancreatic function in pediatric patients.