Tiny treatment trailblazers reunite
University Hospital trial participants celebrate medicine’s approval by FDA
From the moment she was born, Delia Anna Marie Castanon had to fight for her life.
Born at 34 weeks, the girl was immediately admitted to the neonatal intensive care unit at University Hospital, where she would remain for the next six months. She initially had trouble breathing and developed necrotizing enterocolitis, a condition largely seen in premature infants that kills part of the bowel. She underwent multiple surgeries, including one to insert a gastrostomy feeding tube.
When Delia grew jaundiced, a side effect that can develop in babies who receive nutrients intravenously over an extended period, her doctors enrolled her in a study the hospital was conducting for babies born with gastrointestinal complications. The hope was that giving them a fish oilbased supplement as part of their nutrition regimen would help reduce liver inflammation in the babies.
It worked.
“If it wasn’t for that, she probably wouldn’t have made it,” said her mother, Heather Gellhause.
Now 2, Delia was among several children and their families to return to University on Wednesday morning, as part of a reunion celebrating the recent federal approval of the treatment that had saved their lives. For the occasion, she wore a blue flower crown and pink glasses. During the speeches, she babbled happily and fiddled with one of the small stuffed bears given to the children, all wearing white shirts with icons that spelled out “I heart fish.”
Since 2011, University Hospital has enrolled 64 babies in a study of the fish oil treatment, now being produced under the name Omegaven. With previous treatments, about 31 percent of babies that developed liver disease from prolonged nutrition IV use died, said Dr. Cynthia Blanco, who led the NICU study at University and published its results last year in the Journal of Pediatric Gastroenterology and Nutrition.
Blanco, who has worked as a neonatologist at University since 2005, told the families that it was excruciating to watch so many infants succumb to their illnesses.
“Over time, we felt like we had no options and we had nothing else but wait and see,” Blanco said.
When doctors in Texas started exploring the fish oil treatment, which had been studied by a Boston surgeon, Blanco said it was unclear whether it would be effective, “but we knew that we had nothing else and we needed to try something.”
Initiating the study at University proved challenging. The supplement had been used in Europe, Canada and Australia, but it was not widely available in the United States. Pharmacists with University Health System were required to obtain a veterinary license in order to import the fish oil, paying for it out of pocket and administering it to pediatric patients at no charge because it was not yet federally approved.
The infants enrolled in the study were some of the sickest babies in the hospital’s NICU. They included babies with “short gut,” where nutrients cannot be sufficiently be absorbed because of a malformed or damaged small intestine, as well as those with gastroschisis, where an infant’s bowels form outside the body.
Under the fish oil regimen, survival rates increased to more than 90 percent. Worries about potential side effects dissipated, and the patients did not appear to have any long-term problems with their growth or development, Blanco said.
In July, the Food and Drug Administration approved Omegaven for children suffering from parenteral nutrition-associated cholestasis, which reduces liver bile’s flow to the small intestine. The approval relied in part on the data gathered from the children treated at University.
“This has been probably the most rewarding and most valuable thing I’ve done in medicine so far,” Blanco told the families, her voice wavering.
At 6 years old, Eluterio Mireles was one of the older children to attend the reunion at University. Born with just 10 centimeters of small intestine, he was transferred to University after stints at hospitals in Harlingen and McAllen so he could participate in the study. His mother, Laura Barrios, said at the gathering that she ran into the families of other children who had similar issues as her son.
“It’s changed many lives,” she said of the treatment.
Today, Delia still needs significant medical attention. She is under the care of a private-duty nurse, as well as 10 specialists who track her condition. She still uses the feeding tube and participates in therapies at home.
But, her mother said, she remains a fighter.