Ventricular assist devices make headway in pediatric population
When she first presented, the physicians attempted to medically manage Kyah, but due to the progressive HF, she was not able to eat. Also, she was poorly perfused and had constant vomiting and wrenching, the latter of which was “most distressing for the family,” VanderPluym adds. Their child was actively dying, and they were unable to provide her with any nutrition.”
VanderPluym first encountered Kyah in the intensive care unit (ICU), where the clinical care team had decided medical management was no longer viable, and they were concerned that she would go into cardiac arrest with intubation. Thus, Kyah remained extubated, simply receiving high-dose inotropes.
“In the past, we have been forced to allow these types of pediatric patients to die, because we have been limited by device options,” VanderPluym says. In the past, medical device makers have struggled to miniaturize ventricular assist devices to be appropriately implanted in children. In addition, research and development funding traditionally goes toward the adult population, because there is a larger patient population. “Due to Kyah’s size and adolescent age, we thought it appropriate to use a device that was indicated for adults.”
Since the HeartWare ventricular assist system was not FDA-indicated for use in pediatric or adult patients, VanderPluym and her colleagues advocated for and gained a compassionate-use provision from the FDA and Boston Children’s Institutional Review Board to use the device as an exception to save Kyah’s life. Incidentally, the FDA approved the HeartWare pump as a bridge-to-transplant device in adults just nine days after Kyah’s implant was approved. If the device is currently utilized in pediatric patients, it’s either an off-label or a compassionate-use provision.
VanderPluym met Kyah on Friday, Nov. 9, and by Monday morning, “we had received FDA approval, we had mobilized the surgical and nursing teams and the company’s representatives arrived to train our ICU and surgical teams and to bring the device.” By 10 a.m., Kyah was in the operating room and by 2 p.m., the procedure was successfully completed and she was resting in her room. Over the next 24 hours, the staff at Boston Children’s received extensive training about how to monitor and manage patients with these devices.
After she received the device, Kyah’s quality of life improved tremendously, as it transformed her ability to live as a normal teenager. She was even the first child in North America with this device to return to school on a full-time basis as an eighth-grader at Dennis-Yarmouth High School in Cape Cod, Mass.
In assessing her as a candidate for transplantation, the physicians also had to take into consideration Kyah’s concomitant lung disease, called bronchiolitis obliterans organizing pneumonia, which causes inflammation of the bronchioles. “Prior to the device implantation, we weren’t sure if her symptoms were related to her lung disease or her heart failure, which prohibited us from knowing if she would be a good candidate for a heart transplant,” says VanderPluym. “However, her improved hemodynamics and lung functionality with the pump demonstrated that transplant might be effective for her.” Within two weeks of receiving the device, she was put on a donor list.
Over Easter weekend, the care team at Boston Children’s informed Kyah’s family that a heart donation had become available. Her overall operative course with the transplantation, while a complicated procedure, went fairly routinely, and the teenager is back home again.
“With the miniaturization and general improvement of ventricular assist technologies, the reasons for implantation and the patient outcomes in the pediatric population are starting to mirror the adult population,” VanderPluym concludes. “As the technologies continue to advance and pediatric cardiologists get more experience with the devices, our safety threshold will continue to be lowered for implanting these devices in children who have no other clinical option.”