IPD2.10 Observational study of glucose tolerance abnormalities in patients with cystic fibrosis homozygous for Phe508del CFTR treated by lumacaftor-ivacaftor

Misgault, Bastien; Chatron, Eva; Durieu, I.; Mély, Laurent; Abély, M.; Bounyar, Leila; Porzio, Michele; Weiss, Laurence; Kessler, Romain; Kessler, L.

doi:10.1016/s1569-1993(18)30294-7

Cited by 2 publications

(3 citation statements)

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“…Another explanatory factor might be the recently available personalized medicine in terms of CFTR modifier therapy. Although research on the effect of CFTR modifiers on glucose metabolism are heterogenous and mostly limited to low sample sizes, some studies indicate an improved glucose tolerance in patients homozygous for F508del mutation under lumacaftor-ivacaftor use [14]. Moreover, favorable trends in the occurrence of CFRD and the prevalence of pseudomonas aeruginosa were observed in CF patients treated with ivacaftor over 5 years [27].…”

Section: Discussionmentioning

confidence: 99%

Glucose Tolerance in Patients with Cystic Fibrosis – Results from the German Cystic Fibrosis Registry

Prinz

Wosniok²,

Staab

et al. 2020

Klin Padiatr

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Background Oral glucose tolerance (OGT) deteriorates progressively in cystic fibrosis (CF). Clinical registries provide a unique basis to study real-world data. Patients & methods OGT tests (OGTTs) documented in the German CF-registry in 2016 were classified according WHO, modified by ADA: normal glucose tolerance (NGT), indeterminate glycaemia (INDET), impaired fasting glucose (IFG), impaired glucose tolerance (IGT), IFG+IGT, diabetes mellitus (DM). To study the association with lung function, multivariable regression adjusted for age, sex, and CFTR mutation was performed. Results Overall, OGTT screening was done in 35% of CF patients ≧10 years. Of the 996 patients (46.4% females; median age (IQR): 19 (14–27) years) with evaluable OGTTs, 56.2% had either NGT or INDET, whereas 34% had a pre-diabetic OGTT (IFG; IGT; IFG+IGT) and 9.8% a diabetic OGTT. 7 patients had glucose tolerance abnormalities <10 years. DM was more common in females or patients with F508del homozygote mutation, whereas IFG was more frequent in males (all p<0.05). Nearly 75% of patients after transplantation and about half with enteral/parental nutrition and/or steroid use had either a pre-diabetic or diabetic glucose tolerance. In the adjusted model, age (p<0.001) and OGTT category (p=0.013) had both a significant impact on %FEV1. Conclusion Our data of the German CF-registry highlights incidence of glucose tolerance abnormalities in second decade of life in CF patients. However, it also underlines the need for improvement of the documentation and/or performance of OGTT screening in real-world CF care.

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Section: Discussionmentioning

confidence: 99%

Glucose Tolerance in Patients with Cystic Fibrosis – Results from the German Cystic Fibrosis Registry

Prinz

Wosniok²,

Staab

et al. 2020

Klin Padiatr

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“…However, in a long-term 1-year study, glucose tolerance was found to be improved in patients homozygous for the F508del mutation. 58 Due to the ever-increasing life expectancy in patients with CF, CFRD is becoming more prevalent, adding a further burden to patients with CF and creating new challenges for treatment. Different treatment programmes have tried to address both restoration of CFTR and insulin levels with varying degrees of success.…”

Section: Implications For the Clinical Management Of Cfrdmentioning

confidence: 99%

“…However, in a long-term 1-year study, glucose tolerance was found to be improved in patients homozygous for the F508del mutation. 58…”

Section: Implications For the Clinical Management Of Cfrdmentioning

confidence: 99%

Cystic Fibrosis–Related Diabetes: Pathophysiology and Therapeutic Challenges

Kelsey

Koivula

McClenaghan

et al. 2019

Clin Med Insights Endocrinol Diabetes

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Cystic fibrosis–related diabetes (CFRD) is among the most common extrapulmonary co-morbidity associated with cystic fibrosis (CF), affecting an estimated 50% of adults with the condition. Cystic fibrosis is prevalent in 1 in every 2500 Caucasian live births and is caused by a mutation in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene. Mutated CFTR leads to dehydrated epithelial surfaces and a build-up of mucus in a variety of tissues including the lungs and pancreas. The leading cause of mortality in CF is repeated respiratory bacterial infections, which prompts a decline in lung function. Co-morbid diabetes promotes bacterial colonisation of the airways and exacerbates the deterioration in respiratory health. Cystic fibrosis–related diabetes is associated with a 6-fold higher mortality rate compared with those with CF alone. The management of CFRD adds a further burden for the patient and creates new therapeutic challenges for the clinical team. Several proposed hypotheses on how CFRD develops have emerged, including exocrine-driven fibrosis and destruction of the entire pancreas and contrasting theories on the direct or indirect impact of CFTR mutation on islet function. The current review outlines recent data on the impact of CFTR on endocrine pancreatic function and discusses the use of conventional diabetic therapies and new CFTR-correcting drugs on the treatment of CFRD.

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IPD2.10 Observational study of glucose tolerance abnormalities in patients with cystic fibrosis homozygous for Phe508del CFTR treated by lumacaftor-ivacaftor

Cited by 2 publications

References 0 publications

Glucose Tolerance in Patients with Cystic Fibrosis – Results from the German Cystic Fibrosis Registry

Glucose Tolerance in Patients with Cystic Fibrosis – Results from the German Cystic Fibrosis Registry

Cystic Fibrosis–Related Diabetes: Pathophysiology and Therapeutic Challenges

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