Pediatric Pulmonary Hypertension Studies

The Vanderbilt Pediatric Pulmonary Hypertension (PH) Program provides comprehensive care to diagnose and treat all forms of PH in children. Our multidisciplinary team of experts use traditional types of care, and we provide patients and their families opportunities to take part in clinical research.

While there is no cure for many forms of PH, new insights into the disease are helping children live better lives. Advances in the field through simple observation or more detailed research have led to a better understanding of PH and development of new therapies for its treatment.

By joining with you on your journey and learning from your observations, we at Children's Hospital can better serve you and other patients who may share your experience.

Vanderbilt has a long tradition of research into the causes, consequences and treatment of various forms of both pediatric and adult PH. We continue that close collaboration today through leading-edge research. We take part in both investigator-initiated and industry-sponsored research investigations. Our studies range from basic science investigations to clinical trials.

Your participation in research investigations at Vanderbilt is always an option as a part of your child's and family’s experience with PH. We will never require you to be involved in research to receive care at Children's Hospital.

Current clinical study opportunities include but are not limited to:

Human research studies

Investigation of Actigraphy, an Exercise Measurement Device, as a Novel, Well-defined, Reliable, Feasible, Easy to Use, and Non-Invasive Study Endpoint to Facilitate Pediatric Pulmonary Arterial Hypertension Trials and Drug Development

Sponsor: FDA
National PI: D. Dunbar Ivy, M.D.
Vanderbilt Site PI: Eric D. Austin, M.D. MSCI

Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry

Vanderbilt Site PI at Vanderbilt: Eric D. Austin, M.D., M.S.C.I.

PPHNet is a 10-center North American network of clinical specialists, researchers, and centers bringing a collaborative and multidisciplinary approach to improving care for children with pulmonary vascular disease. This study is a registry of subjects who agree to enroll in a longitudinal evaluation of pediatric patients with pulmonary hypertension over time.

Pediatric Pulmonary Hypertension Network (PPHNet) Biorepository Program

Sponsor: Cardiovascular Medical Research Network
National PIs: Eric D. Austin, M.D. M.S.C.I. and Allen Everett, M.D.

This study is a biorepository program and genomic study of pediatric pulmonary hypertension.

Pulmonary hypertension in children and adults

Sponsor: National Institutes of Health, NHLBI, and the Vanderbilt Institute for Clinical and Translational Research
Vanderbilt Site PI: Eric D. Austin, M.D. M.S.C.I.

The Pediatric and Adult Pulmonary Hypertension Programs at Vanderbilt collaborate on a longitudinal study of pulmonary hypertension subjects, and subjects at risk of developing pulmonary hypertension. Studies include clinical, genetic, genomic, and other studies in coordination with our collaborative Vanderbilt Pulmonary Hypertension Research Program.

Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension (TOPP-2)

Sponsor: Association for Pediatric Pulmonary Hypertension
International PIs: Fabrizio Canonaco, M.D., Simone Lerch, M.D., and D. Dunbar Ivy, M.D.
Vanderbilt Site PI: Eric D. Austin, M.D., MSCI

The TOPP-2 registry is an international, non-interventional, prospective registry including children and adolescents newly diagnosed with pulmonary hypertension (PH) to gain further insights in the disease course and long-term outcome of PH in childhood.

Patients will undergo clinical assessments and receive standard medical care, as determined by treating physicians in their daily clinical practice. The TOPP-2 registry is specifically designed to capture the variables proposed as treatment goals in PePH and the reasons for changes in treatment strategy.'

Pulmonary Vein Stenosis Network (PVS Network) Registry Study

Sponsor: Toronto Sick Kids Hospital
International PIs: Christopher Calderone, MD and Rachel Vanderlaan, M.D.
Vanderbilt Site PI: Eric D. Austin, M.D., M.S.C.I.

Our goal is to understand current treatment practices and outcomes, and how best to monitor progression in children with PVS. We will look at retrospective echocardiographic, MRI and CT serial imaging done in this population. We will also look at risk factors that affect outcomes in children with pulmonary vein stenosis/pulmonary vein abnormalities. This will inform clinical decision-making with respect to intervention timing.

Recorded Home Oximetry (RHO) Program Study

Sponsor: PCORI
National PI: Lawrence Rhein, M.D.
Vanderbilt Site PI: Eric D. Austin, M.D., M.S.C.I.

The Recorded Home Oximetry (RHO) Program is an evidence-based, remote patient monitoring program that facilitates safe and effective weaning of home oxygen therapy (HOT) in premature infants with and without PH. RHO provides objective data and is transmitted via Wi-Fi-enabled oximeters, allowing providers to assess oxygenation status and titrate oxygen between clinic visits.