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New Treatment At UCLA Provides Hope For Children Afflicted With Severe Heart Failure

Date:
November 14, 2000
Source:
University Of California, Los Angeles
Summary:
For 7-year-old Kyle Schepens - near death due to a virulent form of heart disease - a new and surprisingly hopeful treatment allowed him to celebrate his eighth birthday. The UCLA findings emerge from a small study of five children who suffered from acute myocarditis - an infection of the heart that causes the muscular wall of the heart to become so inflamed that it can no longer pump blood.

For 7-year-old Kyle Schepens - near death due to a virulent form of heart disease - a new and surprisingly hopeful treatment allowed him to celebrate his eighth birthday.

The UCLA findings emerge from a small study of five children who suffered from acute myocarditis - an infection of the heart that causes the muscular wall of the heart to become so inflamed that it can no longer pump blood. The infection invades a child's heart quickly and can be fatal without immediate medical treatment. Epidemiologists believe myocarditis is caused by a viral infection and affects 1 percent of U.S. children per year.

Pediatric cardiologists at the Mattel Children's Hospital at UCLA report in the November issue of the journal of Heart and Lung Transplantation that by using OKT3, an immunosuppresive treatment used to prepare patients for organ transplant, the need for heart transplantation in children with acute myocarditis can be avoided.

The five children were close to death, but the UCLA doctors found that after the children were given OKT3, all five experienced an amazing recovery. The researchers say these are the first documented cases that using OKT3 in a modified way - and under different circumstances - works in seriously ill children.

In all five cases, OKT3 reversed the heart damage. To date, the doctors have used OKT3 in a total of eight children.

"Our findings show that acute viral myocarditis triggers an autoimmune phenomenon that may be suppressed by aggressive OKT3 immunosuppressive therapy," said UCLA pediatric cardiologist Dr. Juan Alejos, one of the developers of this protocol. "In the children we treated, the results showed a dramatic improvement in heart function by inhibition or reversal of the immune response."

The five patients, aged 15 months to 16 years, were treated at the Mattel Children's Hospital at UCLA for severe congestive heart failure, pulmonary edema and left ventricle heart problems. These children all required aggressive treatment, and four out of the five children experienced life-threatening arrhythmias. Temporary mechanical assist devices were required to keep four out of the five children alive.

All of the children received aggressive immunosuppressive therapy based on OKT3 in conjunction with corticosteroids, IVIG, cyclosporin and azathioprine. Within two weeks of treatment, all five patients demonstrated normalization of their left heart ventricle. Long-term immune suppression was employed in surviving patients for six months.

In Kyle's case, the Schepens family was camping in Yosemite when he got sick. According to his mother, Linda Schepens, he was happy, healthy and playing one day and acutely ill the next day.

"Kyle experienced flu-like symptoms while hiking," Schepens said. "By morning, his temperature had soared to 103 degrees and he became delirious. Three hours later, he was on full life-support, suffering from acute myocarditis."

"When one contracts a viral syndrome, the body forms antibodies to fight off the infection," said pediatric cardiologist Dr. Barbara George. "This immunologic response is manifested by fever, general malaise and muscle aches. As the heart is also a muscle, it too can be affected by this immunologic response, resulting in profound hemodynamic instability. No one knows why one person is affected and another is not. Some investigators suggest that a genetic predisposition may play a part."

A sudden onset of severe cardiac symptoms is a hallmark of the disease. Children suffering from acute myocarditis can deteriorate within a day - experiencing warning signs such as difficulty breathing, chest pain, rhythm problems and exercise intolerance. The body sends off cells from the blood stream into the heart muscle to fight off the virus and that damages the heart muscle.

"The problem with these children is that the body becomes so revved up and immunologically active fighting off the virus that it becomes a problem to give a heart transplant to these children which, previous to OKT3, was their only chance for survival," Alejos said. "If you transplant such a patient, some statistics say that the mortality rate can be as much as 50 percent because the body's immune system is so revved up that it will just reject the heart."

Acute myocarditis can be confirmed by a biopsy, taking a sample of heart tissue to determine the severity of the disease. A biopsy was performed on Kyle by Dr. John Caton, a pediatric cardiologist of Valley Children's Hospital in Madera, Calif., verifying that Kyle did indeed suffer from acute myocarditis.

"Kyle was so critically ill that I didn't think he would survive transport. I felt his chances for survival were less than 10 percent," Caton said. "His heart function was very poor and he was having life-threatening arrhythmias. There wasn't much hope for him at that point."

Caton had the option of transporting him to closer hospitals that had mechanical heart-assist devices that might keep Kyle alive, but he sent Kyle to UCLA because he knew they were using OKT3 with promising results.

"Kyle barely survived the transport," George said. "He was so ill when he arrived that we considered placing him on a mechanical assist device to support his heart. However, after one dose of OKT3, he improved dramatically. After one week, his cardiac function had returned to normal. He had a truly remarkable recovery."

"The doctors started the drug that very morning and within 24 hours we had every hope that he would make it," Kyle's mother said. "Kyle is now 100 percent completely recovered. He plays soccer and water skis. And we believe that Kyle's dramatic recovery was made possible by the use of OKT3."

UCLA cardiologists indicated that none of the children had a recurrence of heart failure or a progression to dilated cardiomyopathy - an enlarged heart - three years after their acute heart problems.

"It appears that OKT3 helps cool down the immune process by shutting down the entire immune system," said Alejos. "We're treating these children like transplant patients. The idea is this - if we can stop the body from being so reactive to the virus, we can minimize the damage to the heart muscle."

In addition to Alejos and George, other members of the research team included Dr. Joseph Ahdoot, Dr. Alvaro Galindo and Caron Burch from UCLA's division of pediatric cardiology; and Hillel Laks, Dr. Daniel Marelli and Dr. Ali Sadeghi from UCLA's division of cardiothoracic surgery.

Mattel Children's Hospital at UCLA is a "hospital within a hospital" located at UCLA Medical Center in Westwood. The hospital offers a full spectrum of primary and specialized medical care for infants, children and adolescents. Its mission is to provide state-of-the-art treatment for children in a compassionate atmosphere, as well as to conduct research that improves the understanding and treatment of pediatric diseases.


Story Source:

The above story is based on materials provided by University Of California, Los Angeles. Note: Materials may be edited for content and length.


Cite This Page:

University Of California, Los Angeles. "New Treatment At UCLA Provides Hope For Children Afflicted With Severe Heart Failure." ScienceDaily. ScienceDaily, 14 November 2000. <www.sciencedaily.com/releases/2000/11/001110065650.htm>.
University Of California, Los Angeles. (2000, November 14). New Treatment At UCLA Provides Hope For Children Afflicted With Severe Heart Failure. ScienceDaily. Retrieved October 22, 2014 from www.sciencedaily.com/releases/2000/11/001110065650.htm
University Of California, Los Angeles. "New Treatment At UCLA Provides Hope For Children Afflicted With Severe Heart Failure." ScienceDaily. www.sciencedaily.com/releases/2000/11/001110065650.htm (accessed October 22, 2014).

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