Authors: Heather Bausell, RD, LDN (1), Katherine Kim, MS (1, 2),
Barbara Burton (1, 2)
Affiliations: (1) Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA
(2) Northwestern University Feinberg School of Medicine, Chicago, IL, USA
Email: firstname.lastname@example.org, Phone: 1-312-227-6122
Objective: Treatment of critically ill patients with long chain fatty acid oxidation disorders (FAOD) with intravenous (IV) sources of medium chain triglycerides (MCT).
Methods: Three critically ill patients with long chain FAODs were admitted in metabolic crisis unable to receive enteral nutrition. Patient A was a 19 month old female with a presumed long chain FAOD admitted in metabolic crisis with cardiomyopathy and ventricular tachycardia. The patient had an affected older female sibling that was deceased. The patient was later found to have TANGO2 gene variants. Patient B was a 10 month old female with long-chain 3-hydroxyacyl-CoA dehydrogenase
(LCHAD) deficiency admitted in metabolic crisis and heart failure secondary to dilated cardiomyopathy. Patients A and B had worsening cardiac function and were considered for cardiac transplantation. Patient C was an 18 year old male with trifunctional protein (TFP) deficiency, status post heart transplantation, admitted in metabolic crisis with severe rhabdomyolysis associated with intercurrent illness. Based on the critical nature of their condition and inability to tolerate enteral nutrition, all three patients received Lipofundin® MCT/LCT 20%, an IV source of MCT available in Europe. Lipofundin was obtained with approval of an emergency Investigational New Drug application (eIND) from the Food and Drug Administration (FDA) and Institutional Review Board
(IRB). Lipofundin® provided 50% MCT and 50% long chain triglycerides (LCT). During a subsequent admission, Patient C developed a bowel obstruction preventing enteral feeds. Smoflipid®, an IV source of MCT approved by the FDA in 2016, was administered to Patient C. Smoflipid® provided 30% MCT and 70%
LCT. Lipofundin® and Smoflipid® were administered with the goal of <10% of calories from LCT until enteral feeds were tolerated.
Results: Lipofundin® and Smoflipid® were well tolerated with no observed adverse reactions. Patients improved or remained metabolically stable while receiving these products. The initial shipments of Lipofundin® were held by United States Customs, delaying use for several weeks in critically ill patients. Smoflipid® was readily available and did not require eIND or IRB applications. The limitation of Smoflipid® was the 30% MCT content compared to 50% MCT content of Lipofundin®.
Conclusion: Use of Lipofundin® and Smoflipid® in long chain FAOD patients was well tolerated with no adverse effects.