SAN ANTONIO (Oct. 24, 2018) ― A San Antonio physician’s passion to save premature babies was instrumental in the U.S. Food and Drug Administration’s recent approval of Omegaven, a lifesaving fish oil treatment for babies with gastrointestinal complications.
The study, led by Cynthia Blanco, M.D., of UT Health San Antonio, was published in the May 2017 edition of the Journal of Pediatric Gastroenterology and Nutrition. The study followed the outcomes of babies treated with fish oil in the Neonatal Intensive Care Unit at University Hospital. The study data provided some of the evidence the FDA considered for its approval July 27 of Omegaven in the United States..
“Overall, since 2011, we have had more than 50 patients enrolled in our long-term study and their survival without liver transplant increased dramatically ― to better than 90 percent,” she said. Dr. Blanco and her team will now be continuing their efforts in writing the national guidelines for Omegaven in the U.S
Dr. Blanco is a professor of pediatrics and holds the Greehey Family Foundation Chair in Neonatology Research at UT Health San Antonio. She also is medical director of the Neonatal Nutrition & Bone Institute and the neonatal transport team at University Health System.
Fish oil treatment
Omegaven is a fish oil-based solution used to provide nutrition to critically ill patients. It has been used in Canada, Australia and Europe, but was not previously approved for widespread use in the U.S.
The FDA’s approval of Omegaven is for pediatric patients with parenteral nutrition-associated cholestasis (PNAC), a liver condition caused by a reduction in the flow of bile from the liver into the small intestine.
Risk of liver disease
“Prematurely born infants typically have many health risks. Because some of the critical development of the gastrointestinal system occurs in the last few weeks of a full-term pregnancy, some babies can have intestinal problems leading to short gut (a shortened intestine). This, in turn, requires prolonged IV nutrition, which significantly impairs liver function,” she explained. In addition, full-term babies born with intestinal disease or who develop intestinal problems can require prolonged IV nutrition, which put them at the same risk of liver disease as preterm infants.
“PNAC affects up to 60 percent of neonates and in those babies requiring long-term IV nutrition, up to 15 percent develop end-stage liver disease. Historically, 31 percent would have died from liver disease,” Dr. Blanco said.
New treatment in the U.S.
“Before the FDA approval of Omegaven, there were only two plant-based emulsions readily available for IV nutritional feeding in the U.S. and they are based on soybean oil. Although there could be multiple factors involved in the development of PNAC, soybean oil has been suspected of causing the complications,” Dr. Blanco said. Our study was the first in the U.S. to examine the acute and long-term outcomes of feeding preterm infants with the fish oil-based emulsion.”
Cutting through the red tape
Dr. Blanco said the European formulation was available to U.S. doctors only on a case-by-case basis but had to be applied for through a lengthy process. “I couldn’t continue to see these babies bleed to death without doing something about it,” she said. So, after initial reports of Omegaven’s success came from Boston, where it initially was being tested, Dr. Blanco applied for an investigational new drug (IND) license to have ready access to Omegaven for babies who needed it.
But it took some doing. University Health System’s pharmacy department had to obtain a special veterinary license to import the fish oil medication. And, because it was not FDA approved, University Health System did not charge patients for the medication. “I am so grateful to University Health System for covering the cost. This investment was the right thing to do for our babies and has helped paved the way to save the lives of countless babies here and across the nation in the future,” Blanco added.
Saving babies’ lives
In the study, Dr. Blanco and her research team found that the fish oil-based lipid emulsion, given intravenously to infants, was well-tolerated and safe, resolved intravenous nutrition-related liver disease and decreased the likelihood of liver transplant. Long-term growth of the babies was comparable to non-treated infants and neurodevelopment was not affected by treatment.
Thirteen preterm babies received Omegaven, ranging from 26 to 210 days, until their condition improved. “All of the babies’ intestinal problems improved so that they could be discharged, none required transplants and none died. And there were no significant side effects, such as high infection rates, increased bleeding or further liver injury from being on an IV for so long,” she said
For ethical reasons, no patients were placed in a comparison group with the soybean oil-based feeding treatment, as it had poor results. However, 400 charts from University Health System’s PREMIEre Clinic, a program for preterm babies staffed by UT Health San Antonio physicians, were reviewed and 119 cases were matched as controls to the study group.
In addition to her work to develop the Omegaven guidelines, the team also is researching the optimal dose of the fish oil medication to ensure the best nutritional outcome while preventing PNAC.
Other members of the study group with appointments to both UT Health San Antonio and University Health System include neonatology fellow Michael Sorrell, D.O.; Alvaro Moreira, M.D., M.S.; Rachel Jacob; Robin Tragus, RN, M.S.N.; Amy Quinn, M.D.; Donald McCurnin, M.D.; Alice Gong, M.D.; and Naveen Mittal, M.D.
Kay Green, an adjunct faculty member associated with University Hospital and the UT Austin College of Pharmacy; Laura Keller from the San Antonio Military Medical Center; and Abeer El Sakka, a neonatal research volunteer associated with Ain Shams University in Cairo, Egypt, also were part of the study.
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