Protein-losing enteropathy (PLE) is a rare complication of
Fontan palliation associated with significant morbidity and mortality. It is characterized by the loss of
serum proteins into the intestinal lumen, and its pathophysiology likely involves enteral
inflammation.
Budesonide, an oral
steroid, is an attractive treatment option because of its potent enteral activity and minimal systemic side effects. A single-center, retrospective review of Fontan-palliated PLE patients treated with oral
budesonide for 6 months or longer was performed. The patient characteristics reviewed were demographics, anatomic diagnosis,
budesonide treatment (dose and duration), other medications and therapeutic interventions, hospitalizations,
serum albumin levels, medical complications, and patient status at the time of follow-up assessment. The study enrolled 10 patients representing 228 patient-months of on-
therapy follow-up evaluation.
Serum albumin levels increased after initiation of
budesonide for 90% of the patients, and clinical evidence of fluid overload improved for 60% of them. Symptomatic improvement was reported in 80% of the cases. During the treatment period, 50% of the patients met the primary end point of death or
cardiac transplantation. In this series of PLE patients, oral
budesonide therapy was associated with significant symptomatic improvement and sustained increases in
serum albumin. However,
budesonide therapy may not alter the long-term outcome for patients with advanced PLE.