Pediatric Pulmonary Hypertension Registry Receives NIH Funding

The National Heart, Lung and Blood Institute has awarded a grant to two investigators from the Children’s Hospital of Philadelphia (CHOP) for their work creating an informatics registry for pediatric patients who suffer from pulmonary hypertension (PH). The institute from the National Institutes of Health (NIH) recognized nine pediatric centers, which are members of the Pediatric Pulmonary Hypertension Network (PPHNet).

PPHNet was designed to address a major unmet need regarding PH in the pediatric field. It offers a shared platform with standardized data in order to enable researchers to evaluate specific medical outcomes from patients, including therapy response in pediatric patients. An individual pediatric PH center working on its own would hardly be able to collect such an amount of information.

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PH is already a rare condition, but the number of cases of pediatric PH is even lower than in the adult patient population. However, the number of hospitalizations due to the disease is increasing. Therefore, physicians face great difficulty in treating pediatric PH patients, since there is no cure for the disease or approved therapies for pediatric purposes, and there is little information on the effects of the disease and its treatments in children.

“The concept of pooling and precisely phenotyping children is going to make a huge difference,” stated the director of the PH program at The Children’s Hospital of Philadelphia, Brian Hanna, MDCM, PhD, in a press release from the hospital. “A key thing is that we eventually will be able to make diagnoses and conduct research protocols all the same way. We’ll be able to advance the science faster and understand it better, so that we’ll be to answer questions about pediatric pulmonary hypertension.”

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In addition, the researchers expect to increase knowledge on the causes, clinical course and diagnosis of the disease by improving the efficiency and accuracy of the registry data collection and storage. Pediatric treatment is currently most of the time based on adult treatment, but there is no evidence that supports the idea that treatment for a child or an adult that both suffer from PH with the same genetic base would result in the same clinical outcomes.

“We still have more questions than answers,” explained the assistant professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania, Rachel Hopper, MD, who is also an attending cardiologist in PH at the CHOP Cardiac Center. “Who gets pulmonary hypertension? Why do they get pulmonary hypertension? Why do some children with hypertension improve over time, while others will end up needing a lung transplant? We need to collaborate as practitioners to pool enough data to answer those questions.”

PH is a lung disease that affects the pulmonary arteries by an increase in blood pressure, making it harder for the right side of the heart to function, which may lead to heart failure. The causes of the disease are often unknown, but may also be related to congenital heart disease, chronic lung disease of prematurity, or genetic disorders.

“Without being melodramatic, pulmonary hypertension is a massive problem in the U.S.,” added Hanna, who currently follows over 650 children and adolescents with PH and is also a clinical professor of pediatrics for the Perelman School of Medicine at the University of Pennsylvania. “It consumes a huge amount of resources, not just hospital beds. We consult with virtually every service in the hospital — catheterizations, echocardiograms, laboratories, pharmacy, and others.”

The development of neonatal and pediatric PAH is known to be related to the impaired or incomplete transition and development of pulmonary circulation during the transformation from fetal to postnatal life. However, most recently, researchers suggested faulty angiogenesis (formation of blood vessels) as a cause of PAH in newborns and children, which leads to reduced vascular density.