The endocrine pancreas is involved early in cystic fibrosis, with multiple negative consequences caused by the deficient secretion of insulin, a hormone important for keeping blood sugar levels under control. The causes of this deficiency are not yet well understood and the effectiveness of new modulatory therapies is still unclear.
Thanks to FFC Ricerca grants, from 2013 to 2023 (FFC#21/2013, FFC#20/2016 e FFC#24/2019) researchers have characterized insulin secretory defects and glucose tolerance in 600 patients with CF, mostly prior to the advent of modulators, with the intention to follow them prospectively.
Now the aims are i) to assess the long-term consequences of insulin secretion deficiencies (which include diabetes, poorer respiratory function, nutritional status and cardiovascular risk); ii) to evaluate if insulin secretory defects independently predict future outcomes and iii) if modulators ameliorated such effects.
This is an observational retrospective study in the 2013-23 cohort. The results might confirm that insulin secretion defects are important for future health in people with cystic fibrosis, but that modulator therapies (administered until now after insulin deficiencies have arisen) cannot completely cure them. Then, it will be important to evaluate the effects of modulators at the earliest possible age, and also to increase attention and care for the metabolic health of older patients.