Sustained recovery of exocrine pancreatic function in a teenager with cystic fibrosis treated with ivacaftor

Smith, Haley; Rayment, Jonathan H.

doi:10.1002/ppul.24952

Cited by 12 publications

(10 citation statements)

References 6 publications

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“…Data have also begun to surface regarding the extrapulmonary effects of CFTR modulator therapy, specifically elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA, Trikafta® Vertex Pharmaceuticals Incorporated) 10,11 . Previous reports suggest that ivacaftor has the potential to restore pancreatic duct function, which could improve pancreatic enzyme secretion, and in turn, absorption 12–16 …”

Section: Introductionmentioning

confidence: 99%

“…10,11 Previous reports suggest that ivacaftor has the potential to restore pancreatic duct function, which could improve pancreatic enzyme secretion, and in turn, absorption. [12][13][14][15][16] The primary objective of this study was to determine if the addition of ELX/TEZ/IVA resulted in improved vitamin D absorption as measured by serum 25-hydroxyvitamin D concentration.…”

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confidence: 99%

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Impact of elexacaftor/tezacaftor/ivacaftor on vitamin D absorption in cystic fibrosis patients

Wright

Ketchen

Rasmussen

et al. 2021

Pediatric Pulmonology

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Background: Cystic fibrosis (CF) is a multisystem disorder that results in the buildup of mucus in various organs. Ninety percent of CF patients are classified as pancreatic insufficient, leading to malabsorption of nutrients and fat-soluble vitamins without the assistance of exogenous pancreatic enzymes. This study was designed to determine if serum 25-hydroxyvitamin D concentrations were impacted by initiation of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA). Methods: Serum 25-hydroxyvitamin D concentrations were measured before and 1 year post-ELX/TEZ/IVA initiation. A Wilcoxon signed-rank test was used to compare values.Results: Seventy-six patients were included in the final analysis. The average age of our population was 25.8 years (SD = 13.2 years) with a majority being male, homozygous F508del, pancreatic insufficient, and not modulator-naive. The median increase of serum vitamin D concentration after initiating ELX/TEZ/IVA was 5 ng/ml (interquartile range = −4, 13; p = .0035). Conclusions:We suggest that ELX/TEZ/IVA may improve fat-soluble vitamin absorption, specifically serum 25-hydroxyvitamin D. These results may lead to adjustments in vitamin supplementation in patients receiving cystic fibrosis transmembrane conductance regulator modulator therapy.

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Section: Introductionmentioning

confidence: 99%

mentioning

confidence: 99%

Impact of elexacaftor/tezacaftor/ivacaftor on vitamin D absorption in cystic fibrosis patients

Wright

Ketchen

Rasmussen

et al. 2021

Pediatric Pulmonology

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“…Those who achieved pancreatic sufficient levels were more likely to have had detectable FE at baseline (8 of 11 vs. 0 of 7, p < .01), less likely to have a second severe mutation (F508del or minimal function: 2 of 11 vs. 6 of 7, p = .01), and more likely to be younger upon starting iva (4 vs. 8.6 years, p < .001) 23 . Several case reports published in 2020 also note improvements in pancreatic exocrine function in older children following use of CFTR modulators 24–26 (Table 2). Adult studies have not yet shown improvements in pancreatic function on CFTR modulators, so it unlikely that adults can achieve pancreatic sufficiency if started on modulators later in life.…”

Section: Other Clinical Parametersmentioning

confidence: 96%

“…In the study of lum/iva performed in the Netherlands in PwCF over age 6 years with FEV1pp ≥ 90%, 5 likely to be younger upon starting iva (4 vs. 8.6 years, p < .001). 23 Several case reports published in 2020 also note improvements in pancreatic exocrine function in older children following use of CFTR modulators [24][25][26] (Table 2). Adult studies have not yet shown improvements in pancreatic function on CFTR modulators, so it unlikely that adults can achieve pancreatic sufficiency if started on modulators later in life.…”

Section: Growth and Nutritionmentioning

confidence: 99%

Review of CFTR modulators 2020

Goetz

Savant

2021

Pediatric Pulmonology

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Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are small molecules that directly impact the CFTR protein, improving the function of the CFTR chloride and bicarbonate channel. Beginning in 2012 with the Food and Drug Administration approval of the first CFTR modulator, ivacaftor, this class of medications has had largely positive effects on many outcomes in people with cystic fibrosis (PwCF), including lung function, growth, and other clinical parameters. There have been continued exciting developments in the current research on CFTR modulators, expanding beyond original studies. This first part of a three-part cystic fibrosis (CF) year in review 2020 will focus on research on CFTR modulators. In addition to reviewing new clinical insights, we describe work done on novel outcomes, adverse effects, issues related to cost, and next steps for clinical trials. The review focuses on articles from Pediatric Pulmonology published in 2020, but it includes articles from other journals that are of particular interest to clinicians. New developments in CF research continue to be brought forth to the CF community, deepening the understanding of this disease and improving clinical care.

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“…Ivacaftor also corrects intestinal pH and improves the nutritional status of patients with gating mutations [27]. Remarkably, ivacaftor reverses pancreatic insufficiency in young as well as older patients [45,46]. Many individuals with the G551D-CFTR mutation taking ivacaftor normalize their fecal elastase levels and decrease or suspend pancreatic enzyme replacement therapy.…”

Section: Cftr Modulatorsmentioning

confidence: 99%

New Therapies to Correct the Cystic Fibrosis Basic Defect

Bergeron

Cantin

2021

IJMS

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Rare diseases affect 400 million individuals worldwide and cause significant morbidity and mortality. Finding solutions for rare diseases can be very challenging for physicians and researchers. Cystic fibrosis (CF), a genetic, autosomal recessive, multisystemic, life-limiting disease does not escape this sad reality. Despite phenomenal progress in our understanding of this disease, treatment remains difficult. Until recently, therapies for CF individuals were focused on symptom management. The discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene and its product, a protein present at the apical surface of epithelial cells regulating ion transport, allowed the scientific community to learn about the basic defect in CF and to study potential therapies targeting the dysfunctional protein. In the past few years, promising therapies with the goal to restore CFTR function became available and changed the lives of several CF patients. These medications, called CFTR modulators, aim to correct, potentialize, stabilize or amplify CFTR function. Furthermore, research is ongoing to develop other targeted therapies that could be more efficient and benefit a larger proportion of the CF community. The purpose of this review is to summarize our current knowledge of CF genetics and therapies restoring CFTR function, particularly CFTR modulators and gene therapy.

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Sustained recovery of exocrine pancreatic function in a teenager with cystic fibrosis treated with ivacaftor

Cited by 12 publications

References 6 publications

Impact of elexacaftor/tezacaftor/ivacaftor on vitamin D absorption in cystic fibrosis patients

Impact of elexacaftor/tezacaftor/ivacaftor on vitamin D absorption in cystic fibrosis patients

Review of CFTR modulators 2020

New Therapies to Correct the Cystic Fibrosis Basic Defect

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