Severe Complications in Pediatric Cystic Fibrosis: Overcoming the Challenges (case reports)

Abstract

Cystic fibrosis (CF), is an autosomal recessive disorder caused by mutations in the CFTR gene, leading to atypical ion transport across epithelial cells and resulting in thick mucus accumulation. This condition significantly affects multiple organ systems, particularly the respiratory and gastrointestinal tracts, leading to infections and progressive dysfunction. The introduction of CFTR modulator therapies has dramatically improved life expectancy, now reaching approximately 50 years in high-income countries. This paper presents two CF cases from the Emergency Clinical Children’s Hospital of Brașov, facing severe, life-threatening complications. One patient successfully overcame a complication related to CF, while the other experienced a significant COVID-19 complication, while presenting as a pulmonary exacerbation (PEx) of CF. The cases highlight the challenging clinical outcomes in CF and personalized approach. Intravenous antibiotic use, as piperacillin/tazobactam, one of the mainstay interventions for PEx due to its effectiveness against Pseudomonas aeruginosa, may rarely lead to severe adverse events, such as drug-induced thrombocytopenia. Furthermore, although respiratory viral infections may adversely affect pulmonary status in CF patients, emerging evidence suggests that SARS-CoV-2 infection did not severely impact this group. Paediatric inflammatory multisystem syndrome (PIMS) associated with SARS-CoV-2 infection has to be considered even in CF patients with scarce symptoms, underscoring the challenge of potential complications.