title: Oral budesonide treatment for protein-losing enteropathy in Fontan-palliated patients.
abstract: 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.
authors.affiliation: Division of Pediatric Cardiology, CS Mott Children's Hospital, University of Michigan, 1500 E Medical Center Drive, L1242 Women's, SPC 5204, Ann Arbor, MI 48109, USA. kurts@med.umich.edu
authors.forename: Kurt R
authors.initials: KR
authors.lastname: Schumacher
authors.forename: Michael
authors.initials: M
authors.lastname: Cools
authors.forename: Bryan H
authors.initials: BH
authors.lastname: Goldstein
authors.forename: Viktoriya
authors.initials: V
authors.lastname: Ioffe-Dahan
authors.forename: Karen
authors.initials: K
authors.lastname: King
authors.forename: Diane
authors.initials: D
authors.lastname: Gaffney
authors.forename: Mark W
authors.initials: MW
authors.lastname: Russell
mesh_terms: D000284:Administration, Oral; D019819:Budesonide / Q000008:administration & dosage*; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D018729:Fontan Procedure / Q000009:adverse effects*; D005938:Glucocorticoids / Q000008:administration & dosage; D006330:Heart Defects, Congenital / Q000601:surgery*; D006801:Humans; D007223:Infant; D008297:Male; D011183:Postoperative Complications; D011504:Protein-Losing Enteropathies / Q000188:drug therapy* / Q000209:etiology; D012189:Retrospective Studies; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D005938:Glucocorticoids; D019819:Budesonide