HEART TO HEART
Clinical trial aims to help babies with rare congenital defect
The room started spinning. Christina DeShaw couldn't believe it was happening again. She and her husband l earned during a 20- week ultrasound that their son would likely be born with a rare heart defect that would require a series of open-heart surgeries — the same defect their daughter was born with several years before.
As she weighed their options after a period of denial , DeShaw knew t he one person she could trust to operate.
She reached out to Dr. Harold Burkhart, chief of pediatric card io thoracic surgery at The Children's Hospital. She couldn' t imagine handing her son over to anyone else. An Iowa native, De Shaw had never been to Oklahoma,
“I would have followed Dr. Burkhart to wherever. Another planet if that's what I had to do.” Christina DeShaw, of Clive, Iowa
but Burkhart had operated on her daughter, Ava, who also was born with hypoplastic left heart syndrome and was given a less than 40% chance of survival, when he worked as a cardiac surgeon at the Mayo Clinic.
“I would have followed Dr. Burkhart to wherever,” said DeShaw, of Clive, Iowa. “Another planet if that's what I had to do.”
Burkhart and The Children's Hospital are taking part in a clinical trial with the Mayo Clinic to help children with hypo plastic left he artsy nd rome(HL HS ), ar are heart defect present at birth.
In babies with HLHS, the left side of the heart is critically underdeveloped and can' t effectively pump blood to the body. Children with HLHS typically undergo three heart surgeries within their first few years of life to increase blood flow to the body and bypass the poorly-functioning left side of the heart.
The Children's Hospital is part of a nationwide consortium that was developed to give patients with hypoplastic left heart syndrome more options when it comes to participating in ground breaking clinical trials and other research.
Because HLHS is rare — it affects about 1,000 infants in the United States each year — no single hospital treats enough patients to advance research with the scope and pace provided by the consortium, officials said.
DeShaw's son, Miles Weitl, was among 1 0 babies who participated in a recently completed Phase 1 trial that focused on the feasibility of using stem cell treatments to strengthen the heart muscle of children with HL HS. Half of those patients were in Oklahoma. For the study, umbilical cord blood was collected at the time of birth. The cord blood cells were later injected into the heart muscle during the child' s second surgery.
Children with the syndrome
only have one pumping chamber that's essentially doing the work of two, Burkhart said. Sometimes they're born with a weak pumping chamber or their heart grows weak over time, Burkhart said.
Researchers hoped injecting the stem cells would promote the growth and strength of cells in the pumping chamber and stimulate the formation of new cells, possibly allowing some patients to avoid a heart transplant, Burkhart said.
A Phase 2 study is now underway that will focus on testing the ability of the stem cells to improve heart function.
“This is kind of a new era in cardiac surgery in children, and Oklahoma is at the lead of it,” Burkhart said. “I think that's a huge deal for Oklahoma, and the fact that we're able to collaborate with our colleagues at Mayo Clinic and there st of the groups in the consortium is a huge advantage to us and the kids here and the ability to offer advanced, state- of- the- art cardiac care to these kids.”
The Children's Hospital is the only pediatric heart surgical program in the state.
Burkhart was recruited to Oklahoma in 2014 to help revitalize the pediatric surgical program.
Before his arrival, most pediatric patients had to go out of state for heart surgery, and The Children's Hospital was doing less than 90 congenital heart surgery cases a year. The year he came to Oklahoma, the hospital had nearly 200 cases, and the number of cases has been growing ever since. Last year, the hospital had more than 500 pediatric heart surgery cases.
Carmen Taylor, a pediatric nurse practitioner who started her career at The Children's Hospital about 20 years ago, can remember when patients had to travel out of state for surgeries. For a while, t he hospital didn't have a pediatric cardiothoracic surgeon.
One boy with hypoplastic left heart syndrome whom she helped care for from birth traveled to California for surgery. His family worked to help raise money for an endowed chair position to fund a pediatric cardiac
surgeon for children with critical heart conditions, which allowed Burkhart to come to Oklahoma.
“This is the first time in a long time that we haven't had to turn somebody away because we didn't have the resources for them, which is amazing,” Taylor said.
Now not only can Oklahoma families stay in state, but families from other states, like DeShaw's, are traveling here for care.
DeShaw and her husband learned about the clinical trial when she was pregnant with their daughter Ava and Ava's twin brother, Aidan. The twins had their cord blood collected, but the study did not have U.S. Food and Drug Administration approval yet. Even if it had, Ava would not have qualified because of other complications she faced, DeShaw said.
Miles was the first person in Oklahoma to have the cells injected into his heart during his second surgery as part of the trial, DeShaw said.
“It's really important that we' re constantly trying to improve how we treat these kids and give them the best possible chance that we can,” DeShaw said.
Last month, Miles threw a tennis ball down the hallway at The Children's Hospital and smiled as Dany, a therapy dog, bounded after it.
The now 3- year- old boy, whose middle name is Harold after the doctor who performed his surgeries, was about to be discharged from the hospital after a long couple of weeks. He had recently undergone his third surgery.
A T-shirt that read “Chicks dig scars” covered the approximately 4-to-5-inch scar on his chest.
Now back home in Iowa, Miles recently started preschool. People would never guess he just had open heart surgery unless they saw his scars, his mom said.
“All these families that are impacted within this area, it makes me so happy to know that they can come to a hospital like this and their children can get amazing care, but that they're also part of this consortium so they're on the cutting edge,” DeShaw said.