Study: Fairness rules hurt some young heart patients

What started out as an attempt to create a more-fair priority list for heart transplants in children has put a small group of patients at a new disadvantage, according to a study by cardiologists at UPMC Children’s Hospital of Pittsburgh.

Nationwide changes in 2016 were made to give higher priority to the sickest patients with fewer treatment options, such as children born with congenital heart defects.

The idea was to sort the sickest patients in the most urgent category, with 1A status, so they had shorter times on the list waiting for a donated organ, according to senior author Dr. Brian Feingold, medical director of pediatric heart failure and heart transplantation at Children’s.

However, the Children’s Hospital study found a dramatic leap in the number of patients who received the priority but may not have needed it.

“The intent was to redo the rules in a way that they could target organs for kids who were the most sick, the least able to wait long times,” Dr. Feingold said.

Patients with congenital heart disease make up about half of the

waiting list, but they are less likely to benefit from advances over the past 25 years in VADs (ventricular assist devices, implanted to help pump the blood), he said. Instead, they are given highdose intravenous medicine to help the heart, which is considered a short-term solution, since patients’ health often declines over time. The new rules aimed to help them.

“The idea came from a good place,” Dr. Feingold said.

The other half of patients on the waiting list have some type of cardiomyopathy, where the heart muscle can’t squeeze hard enough to pump effectively. The most common is dilated cardiomyopathy. VADs can successfully help those young patients recover and get stronger, while they wait for a transplant, he said. Patients with VADs automatically get 1A status, since it’s considered a type of life support. Doctors can also apply for exceptions for other patients, to get them the mosturgent status.

“The reality is that people are bypassing the change by seeking exceptions,” Dr. Feingold said.

Based on data from the national database of transplant candidates, the study was published Monday in the American Journal of Transplantation.

Patients with congenital heart disease did not show any improvement in survival rates while on the list under the new rules. However, the study did find a large increase of exceptions granted to dilated cardiomyopathy patients, giving them 1A status. The study concluded the smaller portion of other types of cardiomyopathy patients who weren’t able to get exceptions may have had a disadvantage as a result.

“We saw a ton of these exceptions, for dilated cardiomyopathy, a 13-fold increase in exceptions,” he said.

But even those exceptions didn’t seem to boost survival rates of these patients, according to the study.

The study said the “disproportionate and inappropriate use” of status 1A exceptions for dilated cardiomyopathy candidates probably diminished the benefit of the policy change for congenital heart disease patients on the transplant list.

“The exceptions are shortcircuiting the intended effect of those rules,” Dr. Feingold said.

And an unintended effect occurred:

Children with the other types of cardiomyopathy — hypertrophic or restrictive — who did not get exceptions, were found to be dying on the waitlist at a rate of four to six times higher than before the new rules went into effect.

Those patients are a small portion overall, about 10%, of the total 400-450 children getting heart transplants in the U.S. each year, Dr. Feingold said.

“We saw they’re not doing as well with the criteria change,” he said. “If they didn’t have an exception, they were dying more often on the waiting list. In the dilated patients who didn’t have an exception, they didn’t have any more increased risk of death.”

Changes need to be made to correct the problems caused by the increased use of exceptions, Dr. Feingold said.

“Our interpretation is the dilated cardiomyopathy candidates didn’t need an exception,” he said, explaining that VADs can ease the time waiting for a transplant.

Doctors strive for a fair distribution of organs for transplants, he said.

“The reality is, because of the shortage of organs that exists, because there is a great need, we have to prioritize who gets an organ,” Dr. Feingold said.

The principle, he said, is to get organs to the patients who are the sickest; the challenge is setting priorities that make that happen.

Dr. Feingold said the study has already affected his group’s approach.

“It makes us think a little bit harder ...,” he said. “There is a responsibility for equity, to make sure the sickest patient has access to organs,” and that includes thinking twice about asking for exceptions for patients and considering the advantages of VADs.

A heart transplant four years ago when he was 4 gave Dean Uplinger, of Cranberry, a new chance at childhood. Until then, his activity had been severely limited because of the effects of Barth syndrome, a rare condition that’s mostly found in boys, which comes with dilated cardiomyopathy, along with weakness in muscles and a short stature.

A VAD gave Dean his chance to survive the wait for a transplant.

His crisis began in February 2016 when he was at Children’s for breathing treatments and he had to be put on a heart-lung bypass machine. Within days, his mother said, he was placed on the Berlin Heart EXCOR, the first VAD made for children. He was placed on the heart transplant list, waiting to be matched with an organ.

Even with the VAD, a child with dilated cardiomyopathy can have a severely limited life, according to Dean’s mom, Angie.

“It was awful,” she said. “It could only be unplugged for 30 minutes a day. He was physically attached to it.”

It was tough keeping a 4year-old occupied as they waited for a donated heart, Ms. Uplinger said.

Then, on April 30 of that year, the transplant was done. He stayed in the hospital for another 2½ weeks. He continues to need therapy for his gross motor skills, but his mother said he’s doing well.

“Now he’s on the least amount of medicine he’s ever been on,” she said.