Elise Pele had been in labor for hours awaiting the arrival of her baby girl, Tatiana, on the evening of Aug. 29. Elise remembers wanting desperately to hear her baby cry – a sign that everything was ok. But that cry never came. She saw Tatiana for only a few seconds before nurses rushed her to the neonatal intensive care unit (NICU) at a local hospital.
“I instantly knew something wasn’t right and I was terrified,” Elise said. “The doctors told me my baby wasn’t breathing right and had to be transferred to Seattle Children’s.”
Tatiana experienced meconium aspiration syndrome where she inhaled a mixture of meconium (stool) and amniotic fluid during labor due to stress, which obstructed and irritated her airways, leading her down a path where doctors thought she would likely die.
Little did her parents know that a medication first developed in the late 1970s, first tried in a premature infant in 1989, (and featured in the 1989 movie “The Abyss”) would save their tiny baby’s life.
“One of the sickest babies I’ve ever seen”
When Tatiana arrived at Seattle Children’s Level IV NICU, which cares for the sickest babies in the region, doctors immediately put her on Extracorporeal Membrane Oxygenation (ECMO), a life support machine that functions as an artificial lung or heart when the organs have failed and need time to heal. Seattle Children’s is an international center of excellence in pediatric extracorporeal life support and is one of the top 10 programs in the nation.
“Tatiana was one of the sickest babies I’ve ever seen. We didn’t even know if she would survive the transport,” said Dr. Kendra Smith, neonatologist and medical director of Seattle Children’s Infant Transport Team. “An oxygenation index of 15 to 20 is a very sick patient who isn’t oxygenating well; Tatiana’s index was 100.”
For weeks, Smith and other doctors tried every possible treatment to no avail. Tatiana’s lungs would not open up to accept air and they were unable to oxygenate her blood. She also had a pulmonary hemorrhage, or bleeding in her lungs, which further complicated her recovery.
“Most babies with a meconium aspiration can come off ECMO after five to seven days, but Tatiana had been on ECMO for 21 days and nothing was working,” Smith said. “Her likelihood of dying was extremely high if we didn’t find a dramatic solution.”
Pele and her husband, Bruce, recognized that Tatiana was gravely ill and began preparing for the possibility that their daughter may not make it.
“We decided that if it was her time, then we should let her go peacefully because she’d already been through so much,” Pele said. “It was just heartbreaking to think that the first time I may get to hold her was when she was taking her last breaths.”
While Tatiana’s outlook was poor, Smith and other doctors on her medical team were determined to try everything they could to save her life.
“Tatiana would look up at you with her big beautiful brown eyes as though she were saying ‘just keep trying,’” Smith said. “She was there, she was responsive, she was saying you can make a difference in my life, and that’s what we were going to do. I just kept telling the family that even though things looked bleak, I hadn’t given up hope and to picture her someday in a little purple dress running through a field of flowers.”
An answer is found in “The Abyss”-like fluid
In searching for an answer, Smith and Dr. Craig Jackson, medical director of Seattle Children’s NICU who also cared for Tatiana, recalled research work they had done in the 1990s with a drug called perflubron. Perflubron is a clear liquid that is poured into the lungs, helping keep the air sacs (alveoli) open and allowing gas exchange to occur. The movie “The Abyss” featured a similar fluid that allowed one of the main characters to dive to great depths in the ocean.
Both Smith and Jackson were involved in several studies on perflubron, first with animals and then with premature and term babies and babies with congenital diaphragmatic hernias. The drug showed great promise in opening collapsed regions of the lungs, keeping them open and allowing gas exchange to occur. The drug also has anti-inflammatory properties that help the lungs to heal, preventing further injury.
But the drug was never FDA approved because its manufacturer withdrew funding for additional clinical research, which meant use of the drug would require special FDA authorization.
Smith approached Tatiana’s parents with the idea.
“They told us about liquid ventilation and even though it wasn’t FDA approved, it gave us hope,” Elise Pele said. “We told Dr. Smith to do anything to try to save her life.”
Smith called the FDA and within five minutes had approval to use the drug.
New life for baby and resurrecting a long forgotten drug
For five days, perflubron was poured into Tatiana’s lungs every two hours. At first, it didn’t seem as though her gas exchange was improving. But then, progress.
“We decided to let the liquid evaporate to see if it was working. Over the next two days, she made a dramatic change,” Smith said. “The fluid had helped heal her lungs and open up her alveoli, allowing her lungs to oxygenate her blood. We were able to take her off ECMO and wean her off her ventilator. It was clear she was going to survive.”
For the family, Tatiana’s dramatic improvement was an enormous relief.
“We went from thinking there was no hope to her suddenly being well enough to come off life support,” said Elise Pele, who was able to take Tatiana home for the first time this week. “We are so thrilled and thankful.”
Jackson and Smith were also very relieved the fluid “worked its magic.”
“It was very exciting and gratifying,” Jackson said. “Researching perflubron was a big chapter in our lives and now, 20 years later, we resurrected the idea and it saved a baby’s life.”
Inspired by Tatiana’s case, Smith and Jackson are determined to help make this drug more available in the neonatal community. Based on previous research, it could be used in treatment for a variety of neonatal lung diseases, preventing babies from needing life support, or possibly dying when all else fails. But in order to unlock this promise and potential use, more research needs to be done to prove its efficacy and safety.
“Neonatologists have known that perflubron holds great potential in pediatrics, and possibly in adult medicine as well, but there is not enough funding for the type of research that could give us insight into its true potential and bring the drug to market,” Jackson said.
Jackson and Smith are working with other investigators around the country to create a national clinical trial to rescue high-risk patients like Tatiana. If successful, this may lead to FDA approval and wider use around the country for infants and children with severe respiratory failure.
The doctors said that Tatiana is a testament to the profound impact perflubron can have.
“You can’t deny when you look at Tatiana, when you see her parents hold her, that it was worth the fight and pushing the boundaries to save her life, even though the situation looked grim,” Smith said. “Given the length of time she was on ECMO, most medical professionals at other centers would have probably given up. I’m so thrilled we went the extra mile at Seattle Children’s to get this drug, to give her a chance at life.”
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If you are interested in supporting liquid ventilation research, please contact Christina Lui in Seattle Children’s Hospital and Research Foundation at firstname.lastname@example.org or 206-987-6812.