Keenan Griffin, 12, is a big fan of pizza. Adores it. So when he woke up one morning about three years ago with chest pains, his mom, Karen, assumed he had heartburn from the copious amounts of greasy cheese and acidic tomato sauce he ate the day before. When the pain didn’t subside, however, she brought him to an Urgent Care clinic. An electrocardiogram (EKG) to test his heart’s electrical activity and a short consultation found nothing abnormal, but as a precaution, Karen was told that Keenan needed to see a cardiologist the following week.
But over the weekend, Keenan didn’t get better, and by Monday, the typically athletic kid was pale and lethargic. “Bring him in,” advised his pediatrician, who took one look at the boy before putting him in an ambulance headed straight for the Emergency Department (ED) at Children’s Hospital Boston. Less than three hours later, with a failing heart, Keenan was on life support and fighting for his life.
The human heart functions like a pump, circulating blood to all the body’s cells. Divided into two halves, the right side takes blood from the body and pumps it into the lungs, while the left side takes blood from the lungs and pumps it out to the body. An average heart, about the size of a fist, beats 100,000 times a day. “If your heart’s not pumping properly, you won’t get enough oxygen and blood to the rest of your body and other organs begin to fail,” says Francis Fynn-Thompson, MD, a cardiac surgeon at Children’s who met Keenan soon after he was admitted.
Keenan’s heart was struggling to pump blood, and the heart muscle function was rapidly deteriorating. In order to save his life, doctors placed Keenan on extracorporeal membrane oxygenation (ECMO), an advanced technology that does the work for the heart and lungs in critically ill patients. But ECMO is only a short-term solution, as prolonged exposure to it can cause damage to a patient’s vital organs.
Cardiologists Elizabeth Blume, MD, and Christopher Almond, MD, MPH, determined that Keenan’s heart failure was caused by a viral infection and his prognosis was dire: he would need a new heart if he were to survive. But the question was, could he get one fast enough? Heart transplant candidates must sometimes wait months for an appropriate donor organ, and the doctors feared Keenan just didn’t have that much time. Karen watched her son, intubated and heavily sedated, and tried not to fixate on the gravity of the situation. “Listening to his doctors, it was clear that we didn’t have very many options,” she says.
In adults, there are a number of mechanical devices used to assist the failing heart and serve as a “bridge” until a donor heart is available. But no such pediatric device is available in the United States. However, there was one option that could potentially save Keenan. Almond explained to Karen that a German company had developed a pumping heart device for children, called the Berlin Heart. The Berlin Heart, once implanted in Keenan’s chest, would take over the role of his failing heart for much longer than ECMO, and hold him over until a donor heart was available. But there was a serious obstacle: The United States Food and Drug Administration (FDA) hadn’t yet approved the device, and in order to get the OK to use one in this country, Keenan’s doctors would need to work quickly with the FDA to get special permission.
The Berlin Heart is a mechanical pump that works by taking over the function of the heart. The first pediatric device of its kind, it’s available in a range of sizes, making it appropriate for kids of all ages. The device consists of thin tubes that are implanted within the heart and attached to a small pump that sits outside of the body.
Although it’s been approved for use in Europe since 1992, the FDA only allows its use on a case-by-case basis, through a “compassionate use” provision when there are no other treatment options. Almond and Fynn-Thompson took Karen aside to explain the process of applying for special permission from the FDA. Karen was gung-ho. “As soon as they told me about the Berlin Heart, I said, ‘Let’s try it,’” she says. The masses of paperwork were processed with haste and within 48 hours, the FDA accepted the request and the German company shipped a specially sized Berlin Heart to Children’s. After surviving a week on ECMO, Keenan underwent open-heart surgery with Fynn-Thompson, who implanted the pump. The next day, Keenan was able to sit up by himself. Over the next two months, while waiting for a heart, he lived in the hospital and became steadily healthier. Because he didn’t need to be intubated or sedated while on the Berlin Heart, as he was on ECMO, Keenan could walk around the hospital and breathe on his own, which made his lungs and the rest of his body stronger as he prepared for surgery. “By the time a heart became available for Keenan, he was in perfect shape for transplant,” says Fynn-Thompson. On May 30, 2007, Fynn-Thompson performed the six-hour-long surgery with success.
—Francis Fynn-Thompson, MD
According to the FDA, development of new pediatric medical devices lags about a decade behind those for adults. Because many diseases that afflict children are considered “orphan diseases” (meaning they affect fewer than 200,000 people) there’s limited financial incentive for companies to invest in and develop devices to treat these conditions.
Even after a company develops a new pediatric device, it faces unique challenges. Like all medical devices, the Berlin Heart couldn’t get FDA approval until it was studied in this country. But designing a clinical trial of the device has been tricky, as there was no similar technology to compare it to. And to get enough data, many hospitals needed to be involved. Luckily, a number of hospitals around the country were advocating for the approval of a pediatric pumping heart device—as quickly as possible. “Many of us came together and said, ‘Listen, we really need this,’” says Fynn-Thompson. “We felt strongly that we should be able to offer this device to our patients.”
Fynn-Thompson and Almond were major players in helping design a Berlin Heart clinical trial. In 2007, the FDA approved a multi-center trial, with Almond as the co-principal investigator. Fifteen centers are now participating, and each year, between 70 and 100 Berlin Hearts are implanted across the country. The study should end by 2011; if all goes well, the device will soon be approved for use.
Almond expects that with FDA approval, the device will make a major impact in the way hospitals care for children with advanced heart failure. “We think it will improve outcomes for children with end-stage heart disease,” he says.
On a recent spring afternoon, Keenan, now 12, munches on an afterschool snack and talks spiritedly about the beginning of baseball season. His first practice is next week and he’s confident he’ll get to play first base. When asked about school, he becomes pensive. He likes engineering and cars, he says, but definitely not medicine. He’s had enough of hospitals.
Luckily, Keenan has no long-term problems related to his time on ECMO. Like all heart transplant patients, Keenan must take immune-suppressing medications every day for the rest of his life so his body doesn’t reject the donor heart. But for Keenan, this barely qualifies as an impediment to his life. And since he’s switched schools, many of his classmates don’t even know he went through a heart transplant.
As a recipient of the life-saving device, Keenan is adamant that the Berlin Heart should be available to other kids. “Everyone who needs it should be able to have one,” he says firmly. “I don’t know where I’d be without it.”