Advances in Treating Children with PAH
The Pediatric Pulmonary Hypertension Comprehensive Care Center at NYP/Columbia is making great strides in the treatment of children with this relatively rare diagnosis. In 2014, the hospital received accreditation as a Center of Comprehensive Care by the Pulmonary Hypertension Association. Established over 25 years ago, Columbia’s program continues to be one of the largest in the world, drawing patients from across the United States and from other countries.
Our experts treat patients with idiopathic (primary) pulmonary hypertension and pulmonary hypertension associated with a variety of conditions, including congenital heart disease, genetic (familial) conditions, scleroderma, lupus, clotting disorders, pulmonary hypertension in newborns, chronic lung disease, HIV, sickle cell disease, and toxins.
“In collaboration with the PPHNet,(Pediatric Pulmonary Hypertension Network) we have developed a large pediatric patient PH registry. We are also working on developing new guidelines for the treatment of PAH,” says the Center’s Medical Director, Erika Berman Rosenzweig, MD, who is Co–Chair of the Pediatric Task Force to develop guidelines at the next World Symposium on PH in Nice, France.
Children with PAH have narrow stiff arteries leading to the lungs, making the heart work harder to pump the blood in this direction. Their hearts tend to enlarge over time, becoming less flexible, and less able to pump blood to the lungs and the rest of the body. This leads the patients to feel out of breath. Without treatment, the average length of survival for children can be 10 months. PAH is often mistaken for asthma or a seizure disorder with a significant delay in diagnosis. That’s why Dr. Berman Rosenzweig and her colleagues are spearheading a campaign for early diagnosis. “Our clinical experience shows that early intervention leads to better outcomes, she says.
“Our team has also made recent advances managing PAH patients with ECMO (extracorporeal membrane oxygenation) and is known for taking caring of children undergoing high risk cardiac surgeries, as well as the atrial septostomy procedure for end-stage PAH.”
The PH Center has always been on the forefront of clinical trials of novel drugs for adult PAH, which has has expanded the treatment options from 1 to 14 FDA drugs. Although none are currently approved for children, investigators are currently enrolling patients in a trial of two new medications that have been FDA approved for PAH in adults.
The PH Center is also at the forefront in identifying inherited forms of PAH including the identification of the very first gene associated with PAH. “We now know that kids have different genetic underpinnings that may also lead them to develop this disease much earlier.” Since the first gene for PAH discovered at Columbia, several other mutations have since been identified by Wendy Cheung, MD, PhD and collaborators.
“Despite the poor prognosis if left untreated, the good news is that many of our patients are doing very well and surviving ten and twenty years from the time of diagnosis,” says Dr. Berman Rosenzweig. “While we don’t have a cure yet, the survival rate has improved greatly with better treatments. We’ve been caring for children who have grown up with us and are now going to college, when just 2 decades ago they would have only had 10 months to live. Some may be on medication via a continuous infusion pump, but they are otherwise mobile and able to live a very full life.”