Fontan-Associated Protein-Losing Enteropathy and Plastic Bronchitis

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