Houston, U.S.A. – A tiny heart pump that maintains blood flow in babies and small children with serious heart failure proved effective and life-saving in a pioneering study involving 17 institutions led by Texas Children’s Hospital and Baylor College of Medicine (BCM). A report on this study appeared recently in the New England Journal of Medicine.

The study looked at the safety and probable benefit of the Berlin Heart EXCOR Pediatric Ventricular Assist Device (VAD), the only VAD available for babies and children. Patients who received the Berlin Heart lived longer on the device and were more likely to receive a transplant or recover heart function than children who were maintained on more traditional support using extracorporeal membrane oxygenation (ECMO), according to the study’s results.

“This study is unprecedented and represents broad collaboration among the top pediatric cardiac transplantation centers in North America, really the who’s who in the field,” said Dr. Charles Fraser Jr., surgeon-in-chief at Texas Children’s Hospital and professor of surgery and pediatrics at BCM. Fraser is also the corresponding author of the report and was national principal investigator of the study. “This study is now the gold standard for VAD therapies in children. Everything going forward will be compared to this.”

Before the Berlin Heart

Before the Berlin Heart, physicians used complicated medical therapies to treat children with heart failure, hoping to keep them alive until a suitable donor heart became available. Newborns and small children often died as they waited since no more than 70 or 80 small donor hearts become available each year. ECMO provided only short term support when the child’s heart failed completely. The Berlin Heart offers families a new ray of hope.

“With the Berlin Heart, we have a more effective therapy to offer patients earlier in the management of their heart failure,” said Fraser. “When we sit with parents, we have real data to offer so they can make an informed decision. This is a giant step forward.”

Children on the device can leave the intensive care unit and go to a regular hospital room where they receive rehabilitation and better nutrition while they wait for a transplant. While there are many such devices available for adults, development and approval of devices for children and especially for babies has lagged.

Prospective study

“This prospective trial is unprecedented because it was not a look back at how children on the device had fared but instead followed their clinical course from the device’s implantation. The results were compared to those of children who received ECMO, the only other method of treatment,” said Fraser, also chief of congenital heart surgery at Texas Children’s.

“We thought we knew a lot about this device and how it worked, but the FDA was insistent that we conduct a trial and they were right. We did not know as much as we should about how the device performs in practice.”

Higher survival rate

For example, he said the survival rate with the device is higher than anticipated and so was the stroke rate. While the stroke rate was of concern, residual effects from the stroke did not prevent most patients from receiving a transplant, the researchers note.

The Berlin Heart is not totally implanted inside the body. Physicians insert cannulas, or flexible tubes, in the heart and they extend through the skin and connect to a small pump located outside the body. That pump, along with its computerized drive unit, maintains blood flow.

Fraser and his colleagues in the United States and Canada compared the outcomes for 48 children (infants to 16 years) who received the device between 2007 and 2010 to matched patients in a national registry for those patients supported by ECMO. They divided the patients who received the German-manufactured heart assist device into two groups based on their body size.

Those in the cohort of smaller patients (average age 1 year) survived on the heart for a median time of 28 days compared to five days for the children on the ECMO device. The longest time a child in this cohort was on the Berlin Heart was 174 days compared to 21 days for the ECMO group. At 174 days, 88 percent of the children in the cohort had been successfully transplanted and 12 percent had died or failed weaning. In the comparison group, 25 percent of the children had died at 21 days and none were still on the ECMO device.

For children in the second cohort with a larger body size (average age 9), the median survival on the Berlin Heart was 43 days compared to five days for the ECMO group. The longest a child stayed on the assist device was 192 days; the longest a member of the matched group was on ECMO was 28 days. In the device group, 92 percent of the children who had received the VAD had been successfully transplanted or weaned off the device at 192 days and 8.3 percent had died. In contrast, 33 percent of the children in the ECMO group had died at 30 days and none were still on that device.

Some children did suffer serious side effects, including major bleeding, infection stroke and high blood pressure. These findings are as important as the survival statistics because they set the stage for future development and refinement of these devices, said Fraser.

Humanitarian Device Exemption approval

The Berlin Heart EXCOR Pediatric Ventricular Assist Device was approved by the U.S. Food and Drug Administration (FDA) for use in children in 2011. That allows the device to be used as a bridge to transplantation. The Berlin Heart has been used in approximately 1,000 children worldwide.

It assists the ventricles – the pumping chambers of the heart – in circulating blood throughout the body. In most children with heart failure, the left ventricle fails and the device is used to assist its activities. However, in a few instances, two devices are used to replace both chambers of the heart. This pump is available in a variety of sizes.

Without the assistance of the device, children awaiting a transplant would become increasingly sick as their heart failed. The device improves their circulation and keeps them alive until they receive a heart transplant. In some rare instances, patients recover enough to come off the device and maintain blood flow on their own.

Fraser first used pump seven years ago

Fraser first used the pump in an infant – tiny Brady Burch of Corpus Christi – seven years ago. Brady, now 7, went on to receive a transplant and enters first grade this fall. The success with Brady stimulated Fraser to push for a study of the device to determine its effectiveness.

Fraser said a major limitation of the study is that it is not truly randomized – a method by which patients are assigned an experimental treatment or currently accepted treatment by chance, as in the flip of the coin.

“There was no way we were going to randomize patients. Even using ECMO as a comparison was difficult,” Fraser said. They drew their comparison patients from the Extracorporeal Life Support Organization registry based in Ann Arbor, Michigan. They matched each patient who received the Berlin Heart to two closely comparable individuals in the registry. While using these historical control patients was difficult, it was the only ethical method of obtaining comparison, Fraser said. He believes the study will provide important information for the future.

“I don’t think we are far from a small, implantable VAD in children,” he said. “The implantable devices will be less cumbersome. There are approved, fully implantable devices already available for adults. This gives us the drive to push even further for more devices like this that can be life-saving options for these children who have no other treatment options.”

Texas Children’s Hospital implanted more Berlin Heart devices in the course of the study than any other center and has one of the most comprehensive pediatric ventricular assist device programs in the world.

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The Woodlands, Texas, U.S.A. – Berlin Heart announced recently that a study published in the current issue of the New England Journal of Medicine (NEJM) concludes that survival using the Company’s EXCOR Pediatric Ventricular Assist Device (VAD) is “significantly greater” than the standard-of-care (extracorporeal membrane oxygenation, or “ECMO”) as a “bridging therapy” for children in need of a heart transplant.

The principal author, Charles D. Fraser, Jr., M.D., Surgeon-In-Chief and Head of the Division of Congenital Heart Surgery at Texas Children’s Hospital, and Professor of Surgery and Pediatrics at Baylor College of Medicine served as the National Principal Investigator for the clinical trail.

The trial was a prospective, multi-center, single-arm cohort study. Children implanted with the EXCOR Pediatric VAD as a bridge to cardiac transplantation were compared to a historical control group of children supported by ECMO that were selected from the Extracorporeal Life Support Organization registry. Seventeen pediatric cardiac centers in the United States and Canada participated in the trial.

A total of 48 patients in need of support (ages 16 and under) were divided into two cohorts based on body size. Each cohort included 24 patients, Cohort 1 the “smaller” participants and Cohort 2, the “larger” participants. Among participants in Cohort 1, median time of support was 27 days in contrast to the matched ECMO control median support time of 5 days.  In Cohort 2, the median support time was 43 days whereas the ECMO control group was supported a median of 5 days. Overall, 88% of participants in Cohort 1 and 92% of participants in Cohort 2 survived to either heart transplantation or weaning with the use of the EXCOR Pediatric Ventricular Assist Device.

“The EXCOR Pediatric Ventricular Assist Device represents a major medical advance in the treatment of children who need a heart transplant,” said Dr. Fraser. “Most of these babies and children would not otherwise survive without the support of EXCOR while awaiting donor hearts. I know that I speak for all of the medical professionals who participated that we are tremendously gratified to have been a part of this ground-breaking study.”

“Until the FDA approved EXCOR, options for mechanical circulatory support as a bridge to heart transplantation in children with severe heart failure were finite,” added Robert Kroslowitz, President and CEO of Berlin Heart’s North American operations. “More specifically, the effective period of ECMO support is typically limited to only 10 days before life-threatening complications ensue that often preclude transplantation.

The narrow duration of support afforded by ECMO is often inadequate considering current waiting times for a pediatric heart transplant (a median of 119 days for all infants in 2008). Consequently, less than 50% of the children supported on ECMO survive to undergo heart transplantation.”

“Publication of the EXCOR study results in one of the most esteemed peer-reviewed medical journals in the world offers strong testimony that the FDA’s recent approval of EXCOR represents a landmark event for children suffering from terminal heart failure,” said Kroslowitz. “Indeed, the worldwide medical community is now offered this lifesaving device to support desperate children who would not otherwise survive while waiting for a heart transplant.  No doubt, EXCOR represents a new era for treating children with heart disease.”

The Berlin Heart EXCOR Pediatric VAD is a mechanical cardiac support system for critically ill pediatric patients suffering from severe heart failure. The system is designed to support pediatric patients of all age groups, from newborns to teenagers, and is intended to bridge patients awaiting heart transplantation from days to many months, until a donor heart becomes available. The FDA granted “Humanitarian Device Exemption” (HDE) approval of Berlin Heart’s EXCOR Pediatric VAD in December 2011. The device is also approved for use in Europe and Canada.

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Few hospitals have as much experience with the Berlin Heart as DMC Children’s Hospital of Michigan. This is why the U.S. Food and Drug Administration (FDA) was eager to hear testimony from Henry L. Walters III, MD, chief of cardiovascular surgery at DMC Children’s Hospital of Michigan. Dr. Walters provided testimony about the device at a recent public hearing.

After reviewing the clinical trial data and hearing testimony from Dr. Walters and others, the device has now been approved. The Berlin Heart is the first ventricular assist device made specifically for pediatric patients. Ventricular assist devices are common in adults. But, until recently, they weren’t manufactured in pediatric sizes.

Ventricular assist devices are used when a patient needs a heart transplant, but can’t wait for a donor heart to become available. The Berlin Heart assists the failing heart until an appropriate donor heart can be found.

“A few times a year, we are faced with a situation where a child simply can’t wait for a heart transplant,” Dr. Walters said. “In these cases, the Berlin Heart works very well and the children generally stabilize and start to thrive and improve while they wait for a donor heart.”

Before the device was approved by the FDA, Dr. Walters and his colleagues used the Berlin Heart under the FDA’s “compassionate use” regulations. Since 2005, Dr. Walters and his colleagues have inserted 14 Berlin Hearts. Of the 14 patients, 10 survived to cardiac transplantation and nine of the patients are thriving today – a result directly attributable to the efficacy of the Berlin Heart. The nine survivors ranged in age from 6 months to 13 years.

Before availability of the Berlin Heart, Dr. Walters and his colleagues occasionally adapted an adult ventricular assist device for use in a child, but the results were not optimal and some patients were too small for a modified adult device.

“The Berlin Heart stands alone in filling a tremendous void in the field of pediatric cardiac failure. I can honestly say that of our nine survivors, eight were too small to have possibly accommodated any of the available adult ventricular assist devices. For these patients the Berlin Heart was truly the key to their survival,” adds Dr. Walters.

The pediatric heart transplant program at the DMC Children’s Hospital of Michigan is the largest in the state of Michigan and one of the largest in the Midwest. For 125 years the DMC Children’s Hospital of Michigan is the first and most experienced hospital in the state dedicated exclusively to the treatment of children.

A leader internationally in neurology and neurosurgery, cardiology, oncology, and diagnostic services, it is ranked one of America’s best hospitals for children. More Michigan pediatricians are trained at the Children’s Hospital of Michigan than in any other facility.

Children’s Hospital of Michigan is one of eight hospitals operated by the Detroit Medical Center (DMC). The DMC is proud to be the Official Healthcare Services Provider of the Detroit Tigers, Detroit Red Wings and Detroit Pistons.

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