CFTR potentiator therapy ameliorates impaired insulin secretion in CF patients with a gating mutation

Tsabari, Reuven; Elyashar, Hila Iron; Cymberknowh, Malena Cohen; Breuer, Oded; Armoni, S.; Livnat, Galit; Kerem, Eitan; Zangen, David

doi:10.1016/j.jcf.2015.10.012

Cited by 55 publications

(39 citation statements)

References 8 publications

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“…The second study reported lower glycemic variability upon GCM at 6 months post-treatment in nine CF subjects with early AGT [7] . Other small studies reported a positive effect of ivacaftor on glucose metabolism in CF patients with G551D or Delta F508/S549R mutations [8][9][10][11][12][13] .…”

Section: Discussionmentioning

confidence: 94%

Effect of one-year lumacaftor–ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients

Misgault

Chatron

Reynaud

et al. 2020

Journal of Cystic Fibrosis

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Section: Discussionmentioning

confidence: 94%

Effect of one-year lumacaftor–ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients

Misgault

Chatron

Reynaud

et al. 2020

Journal of Cystic Fibrosis

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“…There is some evidence to suggest that ivacaftor improves pancreatic endocrine function and sinus disease. [108][109][110][111][112][113] However, it is unclear whether CFTR modulator therapy will permit some chronic maintenance therapies to be safely discontinued to reduce treatment burden, which is currently overwhelming and untenable for many patients. 147 GLOSSARY Adeno-associated vector (AAV): A small virus that infects humans without causing disease.…”

Section: Guidelinesmentioning

confidence: 99%

New and emerging targeted therapies for cystic fibrosis

Quon

Rowe

2016

BMJ

119

122

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Cystic fibrosis (CF) is a monogenic autosomal recessive disorder that affects about 70,000 people worldwide. The clinical manifestations of the disease are caused by defects in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The discovery of the CFTR gene in 1989 has led to a sophisticated understanding of how thousands of mutations in the CFTR gene affect the structure and function of the CFTR protein. Much progress has been made over the past decade with the development of orally bioavailable small molecule drugs that target defective CFTR proteins caused by specific mutations. Furthermore, there is considerable optimism about the prospect of gene replacement or editing therapies to correct all mutations in cystic fibrosis. The recent approvals of ivacaftor and lumacaftor represent the genesis of a new era of precision medicine in the treatment of this condition. These drugs are having a positive impact on the lives of people with cystic fibrosis and are potentially disease modifying. This review provides an update on advances in our understanding of the structure and function of the CFTR, with a focus on state of the art targeted drugs that are in development.

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“…So far three small studies have reported improvement of insulin secretion and hyperglycemia after treatment with ivacaftor 17–19 . Anecdotal reports of hypoglycemia in people treated with CFTR modulators suggest the need for caution and perhaps increased blood glucose monitoring after beginning treatment with a CFTR modulator medication.…”

Section: Cf-related Diabetesmentioning

confidence: 99%

Endocrine Disorders in Cystic Fibrosis

Blackman

Tangpricha

2016

Pediatric Clinics of North America

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Summary Cystic fibrosis can cause multiple endocrine disorders including diabetes, bone disease, short stature, and male hypogonadism. The etiologies are multifactorial but may be directly related in some cases to CFTR dysfunction. Diabetes is the most common endocrine complication and can be expected to affect in almost all people with pancreatic insufficient CF but varies widely in its age of onset. Early identification and treatment of diabetes improves morbidity and mortality in CF. Short stature during adolescence can be transient if puberty is later than average, but it can also be pathologic. Inhaled and systemic steroids can slow growth and should be used at the minimum effective doses. Bone disease in CF is a frequent cause of fragility fractures and associates with worse lung disease. Bone mineral density and vitamin D levels should be monitored. Hypogonadism has been reported in 25% of men with CF. Endocrine complications can cause morbidity and mortality in CF and need to be appropriately detected and managed as part of the medical care of individuals with CF. These complications tend to occur more frequently in older individuals and may be expected to become more common as the CF population grows older.

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CFTR potentiator therapy ameliorates impaired insulin secretion in CF patients with a gating mutation

Abstract: The treatment with ivacaftor in patients with CF carrying gating mutation can ameliorate impaired insulin secretion. Further studies and larger cohorts are needed to evaluate the impact of ivacaftor on insulin secretion in patients with CF carrying gating or other mutations.

Cited by 55 publications

References 8 publications

Effect of one-year lumacaftor–ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients

Effect of one-year lumacaftor–ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients

New and emerging targeted therapies for cystic fibrosis

Endocrine Disorders in Cystic Fibrosis

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