The combined use of patient-reported symptom data and objective lung function measures may help manage acute respiratory events in pediatric CF patients.
Recognizing that patient-reported outcomes (PROs) are important outcome measures in clinical practice and in research, investigators sought to assess the longitudinal variability of 2 measures of PROs — the Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory score and the Chronic Respiratory Infection Symptom Score (CRISS). Additionally, the researchers evaluated how well these PRO-based tools identified acute respiratory events among children with CF. Read more here.
Researchers Develop Cells that Represent CF Airways
Cystic fibrosis (CF) is caused by a mutation of the CFTR gene. While there are many hundreds of known mutations, not all of them are currently treatable which means a significant number of CF patients lack targeted therapies. In an effort to identify new treatments for these patients, researchers from Boston University School of Medicine (BUSM) set out to use blood cells from individuals with CF to make patient-specific induced pluripotent stem cells (iPSCs) and generate lung epithelial cells in the lab. These lung cells are functional and highly similar to the lung cells of the patient. Using these ‘lung cells in a dish,’ they have created a novel platform to discover effective drugs for those patients who currently don’t have any treatment options. Read more here.