Lumacaftor/ivacaftor therapy fails to increase insulin secretion in F508del/F508del CF patients

Moheet, Amir; Beisang, Daniel; Zhang, Lin; Sagel, Scott D.; VanDalfsen, Jill M; Heltshe, S.; Frederick, Carla A.; Mann, Michelle; Antos, Nicholas; Billings, Joanne; Rowe, Steven M.; Moran, Antoinette

doi:10.1016/j.jcf.2020.09.001

Cited by 49 publications

(44 citation statements)

References 27 publications

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“…Part of the pathophysiology of IGT and CFRD involves decreased insulin secretion, insulin resistance and hepatic glucose control abnormalities, along with other mechanisms of insulin insufficiency such as destruction of the pancreas, islet cell inflammation, and oxidative stress 39 . In past studies of those with the G551D mutation, iva improved insulin secretion 38 . Therefore, studies have been undertaken to evaluate if lum/iva improves glucose tolerance in F508del homozygotes.…”

Section: Diabetesmentioning

confidence: 98%

“…The impact of CFTR modulators on the development or control of diabetes is a pertinent area of study as CF related diabetes (CFRD) affects up to 20% of adolescents and 50% of adults with CF 38 . Many pwCF who do not yet have CFRD have impaired glucose tolerance (IGT).…”

Section: Diabetesmentioning

confidence: 99%

“…A separate study evaluated glucose alterations in 39 subjects, aged 12-51 years, prior to and 3, 6, and 12 months after initiation of lum/iva 38 . At one year post treatment, the mean values comparing baseline, 3-, 6-, and 12-month values did not differ for any of the parameters, including fasting glucose levels (p=0.74), 2-hour glucose levels (p=0.26), glucose area under the curve (p=0.67), insulin area under the curve (p=0.82), and peak insulin levels (p=0.33).…”

Section: Diabetesmentioning

confidence: 99%

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Review of CFTR Modulators 2020

Goetz

Savant

2021

Preprint

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CFTR (cystic fibrosis transmembrane conductance regulator) modulators are small molecules that directly change the CFTR protein, improving function of the CFTR chloride channel. Beginning in 2012 with the FDA approval of the first CFTR modulator, ivacaftor, this class of medication has had largely positive effects on many outcomes in people with cystic fibrosis (pwCF), including lung function, quality of life, and growth. 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. Subsequent parts of the CF year in review will cover pulmonary and infectious inflammatory aspects, and the multisystem effects of CF in the 2020 literature. The review focuses on articles from Pediatric Pulmonology, 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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Section: Diabetesmentioning

confidence: 98%

Section: Diabetesmentioning

confidence: 99%

Section: Diabetesmentioning

confidence: 99%

See 1 more Smart Citation

Review of CFTR Modulators 2020

Goetz

Savant

2021

Preprint

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“…The impact of CFTR modulators on the development or control of diabetes is a pertinent area of study as CF-related diabetes (CFRD) affects up to 20% of adolescents and 50% of adults with CF. 27 Many PwCF who do not yet have CFRD have impaired glucose tolerance (IGT). Part of the pathophysiology of IGT and CFRD involves decreased insulin secretion, insulin resistance, and hepatic glucose control abnormalities, along with other mechanisms of insulin insufficiency such as destruction of the pancreas, islet cell inflammation, and oxidative stress.…”

Section: Diabetesmentioning

confidence: 99%

Review of CFTR modulators 2020

Goetz

Savant

2021

Pediatric Pulmonology

View full text Add to dashboard Cite

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.

show abstract

“…Several CFTR modulators and their combinations are under investigation for treatment of CF. There is hope that these new CFTR modulators and combinations of drugs, especially when given at earlier ages, will prevent and/or improve CF bone health and other endocrine-associated diseases such as CF-related diabetes and CF-associated hypogonadism ( 6 , 7 ). One study, the PROMISE study, will examine longitudinally the impact of triple combination therapy with elexacaftor/tezacaftor/ivacaftor in adults and children with CF on not only lung outcomes but also endocrine outcomes, including CF diabetes and CF bone disease ( 8 ).…”

mentioning

confidence: 99%

Correction of the Gene Defect in Cystic Fibrosis: Is It Too Late for Bone?

Tangpricha

2021

The Journal of Clinical Endocrinology &Amp; Metabolism

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Lumacaftor/ivacaftor therapy fails to increase insulin secretion in F508del/F508del CF patients

Cited by 49 publications

References 27 publications

Review of CFTR Modulators 2020

Review of CFTR Modulators 2020

Review of CFTR modulators 2020

Correction of the Gene Defect in Cystic Fibrosis: Is It Too Late for Bone?

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