Treatment of pediatric autoimmune enteropathy with tacrolimus (FK506)

• Published in: Gastroenterology (New York, N.Y. 1943) Vol. 111; no. 1; pp. 237 - 243
• Main Authors: Bousvaros, A, Leichtner, AM, Book, L, Shigeoka, A, Bilodeau, J, Semeao, E, Ruchelli, E, Mulberg, AE
• Format: Journal Article
• Language: English
• Published: New York, NY Elsevier Inc 01-07-1996; Elsevier
• Subjects: Administration, Oral; Atrophy; Autoimmune Diseases - drug therapy; Autoimmune Diseases - pathology; Biological and medical sciences; Biopsy; Duodenum - pathology; Female; Humans; Immunomodulators; Immunosuppressive Agents - administration & dosage; Immunosuppressive Agents - therapeutic use; Infant; Intestinal Diseases - drug therapy; Intestinal Diseases - pathology; Male; Medical sciences; Pharmacology. Drug treatments; Remission Induction; Tacrolimus - administration & dosage; Tacrolimus - therapeutic use; Human; Autoimmunity; Case study; Chemotherapy; Treatment; Intestinal malabsorption; Digestive diseases; Intestinal disease; Immunosuppressive agent; Child
• ISSN: 0016-5085; 1528-0012
• DOI: 10.1053/gast.1996.v111.pm8698205

Autoimmune enteropathy is characterized by chronic secretory diarrhea, villous atrophy, associated autoantibodies, and a partial response to immunosuppression. Currently available therapy (including steroids and cyclosporine) has resulted in remission only in a subset of patients. We evaluated the effects of tacrolimus (FK506) in patients with autoimmune enteropathy refractory to steroids and cyclosporine. Three patients with diagnosed autoimmune enteropathy who continued to have intractable diarrhea despite treatment with steroids and/or cyclosporine were treated with oral tacrolimus. Despite documented histological villous atrophy and poor absorption of oral cyclosporine, therapeutic tacrolimus levels were easily achieved in all 3 patients. All patients showed clinical improvement as documented by decreased stool output and ability to be weaned off parenteral nutrition; response time ranged from 1 to 4 months after tacrolimus was begun. Histological improvement was noted in all patients, and the small bowel biopsy specimens of 2 of the 3 patients showed a return to normal. All patients have been followed up for at least 6 months and are in clinical remission; 1 has received a bone marrow transplant for underlying immunodeficiency. Tacrolimus is a useful drug in the treatment of autoimmune enteropathy, even in patients who have not responded to steroids or cyclosporine. No long-term follow-up of patients with autoimmune enteropathy treated with tacrolimus is currently available. (Gastroenterology 1996 Jul;111(1):237-43)