Intestinal Hypoganglionosis Leading to Intestinal Failure and the Compassionate Use of Omegaven™.
10.5223/pghn.2017.20.1.55
- Author:
Racha KHALAF
1
;
Sara KARJOO
;
Paul DANIELSON
;
Michael WILSEY
;
Fauzia SHAKEEL
Author Information
1. Department of Medical Education, Johns Hopkins All Children's Hospital, St. Petersburg, FL, USA. rkhalaf1@jhmi.edu
- Publication Type:Case Report
- Keywords:
Hirschsprung disease;
Omega-3 fatty acids;
Liver diseases;
Total parenteral nutrition
- MeSH:
Alanine Transaminase;
Alkaline Phosphatase;
Aspartate Aminotransferases;
Bilirubin;
Cholestasis;
Compassionate Use Trials*;
Diagnosis;
Empathy*;
Emulsions;
Fatty Acids, Omega-3;
Hirschsprung Disease;
Humans;
Infant, Newborn;
Liver;
Liver Diseases;
Parenteral Nutrition, Total;
United States Food and Drug Administration
- From:Pediatric Gastroenterology, Hepatology & Nutrition
2017;20(1):55-60
- CountryRepublic of Korea
- Language:English
-
Abstract:
Intestinal hypoganglionosis is a rare innervation disorder that provides numerous nutritional, medical and surgical challenges. In this case report, we present a case of a newborn with intestinal hypoganglionosis leading to intestinal failure and intestinal failure-associated liver disease who responded to Omegaven™, a fat emulsion comprised of omega-3 fatty acids. Omegaven™ has been shown to be beneficial in the management of cholestatic liver injury. Clinical success with Omegaven™ was seen in this patient with a clear decrease in aspartate aminotransferase, alanine aminotransferase, alkaline phosphatase and complete resolution of cholestasis with a direct bilirubin of zero within two weeks of initiation of Omegaven™. No current guidelines for the diagnosis and management of hypoganglionosis are available. We recommend a multidisciplinary approach and the use of novel therapies such as fat emulsions composed of omega-3 fatty acids for improved patient outcomes. Appropriate compassionate use protocols should be obtained from the Food and Drug Administration prior to initiation of Omegaven™.
