sildenafil-citrate and Protein-Losing-Enteropathies

Protein-losing enteropathy (PLE) is a chronic condition involving multiple organ systems that may develop any time following Fontan completion. The pathogenesis of PLE is complex and multifactorial. Chronic venous hypertension, low cardiac output, and abnormal lymphatics may all play a role in the pathogenesis of PLE. Common signs and symptoms include chronic diarrhea, abdominal pain, and ascites. Diagnosis is based on the presence of signs and symptoms in addition to hypoalbuminemia and elevated stool alpha 1 antitrypsin. Early identification and a comprehensive approach to evaluation and treatment are important, as they may affect survival. The initial evaluation should include cardiac catheterization for hemodynamic assessment. Although an evidence base for treatment is lacking, various medical, interventional, and surgical approaches have been described with variable degrees of success. Commonly used therapies include nutritional support, diuretics, subcutaneous unfractionated heparin, budesonide, and sildenafil. Limited data exist for Fontan conversion or takedown. Assessment for heart transplantation should be considered. PLE mortality is high-approximately 50%-but may be mitigated by aggressive investigation and management. The evolving understanding of the role of lymphatics in the pathophysiology of PLE and the emerging role of interventional lymphatic procedures may further improve outcomes in this patient population.

Topics: Abdominal Pain; Academic Medical Centers; Ascites; Budesonide; Chronic Disease; Combined Modality Therapy; Diagnosis, Differential; Diarrhea; Diuretics; Female; Fontan Procedure; Heart Defects, Congenital; Heparin; Humans; Male; Prognosis; Protein-Losing Enteropathies; Rare Diseases; Risk Assessment; Sildenafil Citrate; Treatment Outcome

Sildenafil is increasingly being used in the management of pulmonary arterial hypertension in the newborn. Its role in patients with congenital cardiac disease is less well defined and as yet has only been reported sporadically.. Present our experience with sildenafil treatment in patients with a failing Fontan circulation.. Retrospective review of 13 symptomatic patients after Fontan palliation who received treatment with sildenafil between January, 2006 and July, 2008.. Three patients suffered from protein-losing enteropathy, four patients presented with bronchial casts, two had severe cyanosis after fenestrated Fontan procedure, two had prolonged chylous effusions, one had a previous failure of Fontan and take-down, and one patient had arrhythmias and end-stage cardiac failure requiring conversion to an extra-cardiac Fontan. Sildenafil was used in the dosage of 1-2 milligrams per kilogram 3-4 times per day. Protein-losing enteropathy and alpha-1-antitrypsin levels improved in all three patients on sildenafil treatment. One of these patients had a concomitant catheter creation of a fenestration, as did two patients presenting with bronchial casts and both patients with persistent chylous effusions. All four patients with bronchial casts and two patients with cyanosis improved significantly on sildenafil treatment. Chylous effusions decreased after sildenafil and stent enlargement of a fenestration. There were no significant side effects requiring sildenafil withdrawal over a treatment period ranging from 2 months to 2 years.. Sildenafil can be used safely and effectively in the treatment of patients with a failing Fontan circulation.

Topics: Child; Child, Preschool; Female; Follow-Up Studies; Fontan Procedure; Heart Failure; Humans; Hypertension, Pulmonary; Male; Piperazines; Postoperative Complications; Protein-Losing Enteropathies; Purines; Retrospective Studies; Shock; Sildenafil Citrate; Sulfones; Time Factors; Treatment Outcome; Vasodilator Agents

A 9.5-year-old girl after Fontan procedure for hypoplastic left heart syndrome had recurrent protein-losing enteropathy (PLE) develop 2 months after partial catheter closure of the Fontan fenestration. Despite satisfactory hemodynamic measurements under general anesthesia, we postulated that she suffered from increased pulmonary vascular reactivity and commenced her on Sildenafil treatment. After 6 weeks of oral Sildenafil treatment, her serum albumin and the fecal alpha-1-antitrypsin levels normalized, and her exercise tolerance had increased. There was also an improvement of the mesenteric arterial flow patterns on Doppler studies. Sildenafil should be considered in the treatment of PLE after the Fontan procedure.

Topics: Child; Female; Fontan Procedure; Humans; Hypoplastic Left Heart Syndrome; Piperazines; Protein-Losing Enteropathies; Purines; Sildenafil Citrate; Sulfones; Vasodilator Agents