Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data.

Clinical trials demonstrate the short-term efficacy of dual CFTR modulators, but long-term real-world data is limited. We aimed to investigate the effects of 24-month lumacaftor/ivacaftor (LUM/IVA) therapy in pediatric CF patients (pwCF). This observational study included pwCF homozygous for F508del mutation treated between 2021 and 2023.

Hungarian Linguistic, Cross-Cultural, and Age Adaptation of the Patient Satisfaction with Health Care in Inflammatory Bowel Disease Questionnaire (CACHE) and the Medication Adherence Report Scale (MARS).

Background: The TRANS-IBD study examines the superiority of joint transition visits, with drug adherence and patient satisfaction among the outcome measures. Our aim was a cross-cultural, age- and disease-specific adaptation of the 'Medication Adherence Rating Scale' (MARS) and 'Patient satisfaction with health care in inflammatory bowel disease questionnaire' (CACHE) questionnaires in patients with inflammatory bowel disease (IBD).

Methods: Linguistic and cultural adaptation using test and re-test procedures were performed.