In the past, the only treatment for children with potentially life-threatening pulmonary hypertension was a lung or a heart-lung transplant.
Today, there is new hope.
Sildenafil citrate – the active ingredient in the erectile dysfunction medication Viagra – is among the latest treatment options for the disease commonly found in children with congenital heart defects. The Food and Drug Administration approved the drug, marketed as Revatio, last year.
“Revatio has proven to be a very valuable tool for treating pulmonary hypertension,” said Scott Fletcher, M.D., director of the pulmonary hypertension clinic at Children’s Hospital. Dr. Fletcher also is associate professor of pediatrics in the Joint Division of Pediatric Cardiology/Congenital Heart Disease between Children’s, UNMC and Creighton University.
Pulmonary hypertension is a rare and progressive blood vessel disorder that results in high blood pressure in the lungs. The true incidence in children is unknown. Initial symptoms include difficulty breathing, dizziness and fatigue.
Pulmonary hypertension starts when the walls of the blood vessels that supply blood to the lungs are thicker than normal, making it more difficult for blood to get through to the lungs. As a result, the heart must pump harder to overcome the resistance. Over time, the heart’s right ventricle enlarges and thickens in response to the extra work. Left untreated, patients have an average survival time of less than three years from the time of diagnosis.
“Pulmonary hypertension is relatively rare in children. It can occur without an identifiable cause, can be familial, or associated with other medical problems, such as collagen vascular disease, portal hypertension, HIV infection and hemoglobinopathies,” Dr. Fletcher said. “In children, it is most frequently associated with structural heart disease or lung disease. It is becoming more frequently diagnosed in early childhood as we have babies who survive in neonatal intensive care units, that in the past have died.”
Pulmonary hypertension is one of the reasons for early surgical intervention in children with congenital heart defects. “If we don’t operate, pulmonary hypertension can progress to irreversible pulmonary obstructive disease,” Dr. Fletcher said. “My hope is to see less pulmonary hypertension in congenital heart defect patients as we operate earlier and earlier.”
Congenital heart defects are the No. 1 occurring birth defect and the No. 1 cause of birth-defect-related deaths in infants. At least eight of every 1,000 infants born each year have a heart defect. That translates to an estimated 250 infants born with congenital heart defects each year in Nebraska.
The best diagnostic test of pulmonary hypertension is echocardiography, Dr. Fletcher said.
Revatio and Tracleer (Bosentan) are the first oral agents for pulmonary hypertension that have shown clinical efficacy, Dr. Fletcher said. “These treatments are relatively new and offer hope for patients with the progressive disease,” he said. “It doesn’t cure, but it prevents the progression of a horrific disease.”
The treatment, however, is costly. Tracleer, an oral medication taken twice daily, costs $40,000 per year, Dr. Fletcher said. Revatio, an oral medication taken three times daily, costs $10,000. Alternatively, the use of Flolan, an IV-administered drug, is $90,000 per year, he said.