From almost 653 initially identified records 5 studies met inclusion criteria - 1 clinical trial, 2 observational studies and 2 case reports. Evidence suggests CFTRm may improve glucose tolerance and insulin secretion in some pediatric patients, particularly in those with preserved β-cell function or early-stage CFRD. However, results varied across studies with some showing no significant improvements in glycemic control.
Authors' conclusions
While early findings suggest CFTR modulators may offer metabolic benefits and potentially delay or reduce the need for insulin therapy in children CFRD, current evidence is limited. Larger, pediatric-focused clinical trials with standardized glycemic outcomes are essential to determine the long-term efficacy and safety of CFTRm in managing or preventing CFRD.