Fontan-Associated Protein-Losing Enteropathy and Plastic Bronchitis
Kurt R. Schumacher, Kathleen A. Stringer, Janet E. Donohue, Sunkyung Yu,Ashley Shaver
DOI:
http://dx.doi.org/10.1016/j.jpeds.2014.12.068 Abstract
Objective
To characterize the medical history, disease progression, and treatment of current-era patients with the rare diseases Fontan-associated protein-losing enteropathy (PLE) and plastic bronchitis.
Study design
A novel survey that queried demographics, medical details, and treatment information was piloted and placed online via a Facebook portal, allowing social media to power the study. Participation regardless of PLE or plastic bronchitis diagnosis was allowed. Case control yses compared patients with PLE and plastic bronchitis with uncomplicated control patients receiving the Fontan procedure.
Results
The survey was completed by 671 subjects, including 76 with PLE, 46 with plastic bronchitis, and 7 with both. Median PLE diagnosis was 2.5years post-Fontan. Hospitalization for PLE occurred in 71% with 41% hospitalized ≥3 times. Therapy varied significantly. Patients with PLE more commonly had hypoplastic left ventricle (62% vs 44% control; OR 2.81, 95% CI 1.43-5.53), chylothorax (66% vs 41%; OR 2.96, CI 1.65-5.31), and cardiothoracic surgery in addition to staged palliation (17% vs 5%; OR 4.27, CI 1.63-11.20). Median plastic bronchitis diagnosis was 2years post-Fontan. Hospitalization for plastic bronchitis occurred in 91% with 61% hospitalized ≥3 times. Therapy was very diverse. Patients with plastic bronchitis more commonly had chylothorax at any surgery (72% vs 51%; OR 2.47, CI 1.20-5.08) and seasonal allergies (52% vs 36%; OR 1.98, CI 1.01-3.89).
Conclusions
Patient-specific factors are associated with diagnoses of PLE or plastic bronchitis. Treatment strategies are diverse without clear patterns. These results provide a foundation upon which to design future therapeutic studies and identify a clear need for forming consensus approaches to treatment.
