{"id":12912,"date":"2018-11-27T18:48:31","date_gmt":"2018-11-27T18:48:31","guid":{"rendered":"https:\/\/www.tmc.edu\/news\/new-drug-helps-reverse-liver-damage-in-preemies\/"},"modified":"2019-08-16T15:04:39","modified_gmt":"2019-08-16T15:04:39","slug":"new-drug-helps-reverse-liver-damage-in-preemies","status":"publish","type":"post","link":"https:\/\/www.tmc.edu\/news\/2018\/11\/new-drug-helps-reverse-liver-damage-in-preemies\/","title":{"rendered":"New drug helps reverse liver damage in preemies"},"content":{"rendered":"
Twins Henry and Charlie Fitzpatrick are as physically\u00a0active as most 5-year-olds. They chase each other, play with their dog and dance to Michael Jackson videos.<\/p>\n
But their parents, David and Aly Fitzpatrick, weren\u2019t always sure the boys would grow to be the rambunctious children they are now. When David lifts up their shirts to tickle their bellies, the reason is evident: Both have several scars resembling railroad tracks\u2014reminders of the multiple surgeries needed to keep the brothers alive during their first months of life.<\/p>\n
The twins continue to grow and thrive, in part, because of Omegaven, an omega-3 fatty-acid-rich lipid solution made from fish oil. The potentially life-saving drug\u00a0is an important source of nutrition in infants suffering from a complication caused by long-term liver damage, known as parenteral nutrition-associated cholestasis or PNAC<\/a>.<\/p>\n Over the last decade, Texas Children\u2019s Hospital<\/a> has treated nearly 300 babies of PNAC with Omegaven while gaining recognition for its work and attracting patients from across Texas and beyond. Omegaven<\/a> was approved by the U.S. Food and Drug Administration on July 27, 2018 as a source of nutrition for cholestasis, a liver disease characterized by the stoppage or reduction of bile flow.<\/p>\n Nearly 20 percent of babies who spend time in a neonatal intensive care unit (NICU) develop intestinal failure and are dependent on artificial nutrition called total parenteral nutrition, or TPN, according to Muralidhar Premkumar, M.D., a neonatologist at Texas Children\u2019s\u00a0Hospital and assistant professor of pediatrics at Baylor College of Medicine<\/a>.<\/p>\n \u201cThese babies are born so early that their gut and intestines are immature,\u201d Premkumar said. \u201cMany of them develop necrotizing enterocolitis, an inflammation and infection in the intestines, and they can\u2019t tolerate feedings. So they spend a lot of time on artificial nutrition. The liver comes under stress from the gut imbalance, which leads to the cholestasis.\u201d<\/p>\n About half of TPN-dependent infants get better with time, but others like the Fitzpatrick twins develop severe liver damage that can result in death, Premkumar said. Treatment for those babies can include liver transplants, which also can have grave results.<\/p>\n \u201cThere is a 50 percent mortality rate with liver transplants, so the goal was to avoid this at all costs,\u201d said Amy Hair, M.D., program director of neonatal nutrition at Texas Children\u2019s and assistant professor\u00a0of pediatrics at Baylor College of Medicine. \u201cWe have a way, with this life-saving drug, to avoid this horrible road. We can give them Omegaven to save and protect the liver and we don\u2019t have to talk about a transplant.\u201d<\/p>\n Among the Texas Children\u2019s babies on Omegaven, 85 percent get better with time, and over the last five years not one has needed a liver transplant, Premkumar said.<\/p>\n The FDA approval follows 10 years of work by neonatologists and neonatal nutrition experts at Texas Children\u2019s, Boston Children\u2019s Hospital and the University of California, Los Angeles (UCLA) Mattel Children\u2019s Hospital. Team members collected data on patients, including Charlie and Henry Fitzpatrick, who were part of the compassionate use protocol of Omegaven.<\/p>\n Boston Children\u2019s began testing the drug under the leadership of Mark Puder, M.D., Ph.D., an associate in the hospital\u2019s department of surgery and professor of surgery at Harvard Medical School, who brought Omegaven to the United States. The drug, developed by German health care company Fresenius Kabi<\/a>, was already used by doctors across Europe.<\/p>\n Pruder and other researchers studied the use of fish oil-based emulsions on 10 patients receiving parenteral nutrition (PN) and published their results in the\u00a0February 2010 edition of the Journal of Pediatric Gastroenterology and Nutrition<\/em><\/a>.<\/p>\n At the time, researchers were concerned about the development of essential fatty acid deficiency (EFAD), which was why drugs like Omegaven were not widely used in clinical practice in the United States. The Pruder team found that none of the patients on Omegaven\u00a0and PN developed the deficiency. In fact, the bilirubin level\u2014a measurement used to determine jaundice, anemia and liver disease\u2014improved in nine of the 10 patients.<\/p>\n They concluded that \u201cwhen dosed appropriately, fish oil-based lipid emulsions contain sufficient amounts of essential fatty acids to prevent EFAD and sustain growth in patients completely dependent on PN.\u201d<\/p>\nOmegaven approved by FDA<\/h4>\n
Study of Omegaven<\/h4>\n