Research and Publications
Who funded the study?
The National Institutes of Health funded this multi-center study, which is conducted through the PPHNet network of clinicians and researchers dedicated to the improvement of care for children with pulmonary vascular disease.
What is the purpose of the registry?
The goal of this study is to create a registry of patients with pediatric pulmonary hypertension. By gathering a large amount of information over an extended period of time we hope help to patients with childhood onset pulmonary hypertension.
What is a registry?
Patient registries are a way to collect and organize information about a group of people with a specific disease or condition for the purposes of improving understanding of the disease, and developing effective treatments.
Who can participate?
Any patient who has had onset of pulmonary hypertension between birth and 18 years of age may be enrolled. Patients do not have to exhibit signs or symptoms of the disease at the time of enrollment.
Where is the study being conducted?
The study is being conducted at eight hospitals across the country and Canada. See the “Centers page” to find the center nearest you. We anticipate participation of approximately 1,000 patients across all sites.
What would I have to do?
The only information that we gather will already be regularly collected at office visits. No additional visits or tests will be ordered for this research. The information will be pulled directly from the electronic medical record and will immediately be de-identified.
What are the benefits?
This study will help researchers learn more about pediatric onset pulmonary hypertension and how to best care for this growing group of patients. What we learn from this registry may improve the care pediatric pulmonary hypertension patients receive in the future.
How can I get more information about clinical studies?
The National Institutes of Health’s web site, Clinical Trials, provides general background information on clinical trials.
The National Heart, Blood, and Lung Institute, in collaboration with New England Research Institutes, has published a resource for parents, Children and Clinical Studies, at http://www.nhlbi.nih.gov/childrenandclinicalstudies/index.php
How can I get more information about the study and participation?
A summary of the PPHNet study can be found at https://clinicaltrials.gov/ct2/show/NCT02249923
Please contact the PPHNet Center nearest to you for information about eligibility and participation.
Actigraphy Jr. A Prospective Study to Investigate the Combination of Physical Activity and Heart Rate with the Use of Actigraphy as a Novel, Well-defined, Reliable, Sensitive, Easy-to-use, and Non-invasive Study Endpoint to Facilitate Pediatric Pulmonary Arterial Hypertension Trials and Drug Development for Children Ages 0-6 Years,
FDA Project number 75F40199C0090
Site Principal Investigator (Study PI: D. Ivy)
1/2 Kids MoD PAH Trial: Mono- versus Duo-Therapy for Pediatric Pulmonary Arterial Hypertension
NIH NHLB1 1-UG3 HL 151458-01A1
Site Principal Investigators (PIs: L Romer, E Berman Rosenzweig, S Abman)
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.
Principal investigator: Dunbar Ivy https://clinicaltrials.gov/ct2/show/NCT02909608
Pediatric Pulmonary Hypertension Network (PPHNet) Biorepository Program. Sponsor: Cardiovascular Medical Research Network
Principal investigators: Eric D. Austin, M.D., Allen Everett, M.D.
Data Fusion: A Sustainable, Scalable, Open Source Registry Advancing PVD Research. National Heart, Lung, and Blood Institute
(NHLBI) Project #1U01HL121518-01.
Principal Investigators: Abman SH, Mandl KD.
https://clinicaltrials.gov/ct2/show/NCT02249923?term=pediatric+pulmonary+hypertension&rank=2
NHLBI T32 HD101388-01, “Multidisciplinary Research Training in Pediatric Pulmonary Vascular Disease. Principal Investigators: Steven Abman, Eric Austin, Jeffrey Fineman
- The Inception of the Pediatric Trial to Determine Difference Between Mono or Dual Therapy for Pediatric Pulmonary Hypertension
Collaco JM, Abman SH, Austin ED, Avitabile CM, Bates A, Fineman JR, Freire GA, Handler SS, Ivy DD, Krishnan US, Mullen MP, Varghese NP, Yung D, Nies MK, Everett AD, Zimmerman KO, Simmons W, Chakraborty H, Yenokyan G, Newell-Sturdivant A, Christensen E, Eyzaguirre LM, Hanley DF, Rosenzweig EB, Romer LH. Kids Mod PAH trial: A multicenter trial comparing mono- versus duo-therapy for initial treatment of pediatric pulmonary hypertension. Pulm Circ. 2023 Oct 31;13(4):e12305.
- Determining the Success of Using Wearable Devices in Infants and Children with Pulmonary Hypertension
Avitabile CM, Yung D, Handler S, Hopper RK, Fineman J, Freire G, Varghese N, Mullen MP, Krishnan US, Austin E, Silveira L. Measurement of Physical Activity by Actigraphy in Infants and Young Children with Pulmonary Arterial Hypertension. The Journal of Pediatrics. 2023 Nov 1;262:113639.
doi.org/10.1016/j.jpeds.2023.113639
- Pulmonary Hypertension in Children with Down Syndrome
Rachel K. Hopper, Steven H. Abman, Eleni G. Elia, Catherine M. Avitabile, Delphine Yung, Mary P. Mullen, Eric D. Austin, Angela Bates, Stephanie S. Handler, Jeffrey A. Feinstein, D. Dunbar Ivy, John P. Kinsella, Kenneth D. Mandl, J. Usha Raj, Lynn A. Sleeper. Pediatric Pulmonary Hypertension Network Investigators. Pulmonary Hypertension in Children with Down Syndrome: Results from the Pediatric Pulmonary Hypertension Network Registry. J Pediatr 2023;252:131-40
doi.org/10.1016/j.jpeds.2022.08.027
- Hemodynamics of Pediatric Pulmonary Hypertension Patients Enrolled in the PPHNet Registry
Rosenzweig EB, Bates A, Mullen MP, Abman SH, Austin ED, Everett A, Fineman J, Feinstein J, Hopper RK, Kinsella JP, Krishnan US, Lu M, Mandl KD, Raj JU, Varghese N, Yung D, Handler SS, Sleeper LA. Pediatric Pulmonary Hypertension Network. Cardiac Catheterization and Hemodynamics in a Multicenter Cohort of Children with Pulmonary Hypertension. Ann Am Thorac Soc. 2022 Jan 20. doi.org/10.1513/AnnalsATS.202108-998OC
- Building a Pediatric Pulmonary Hypertension Program
Handler, SS, Varghese, NP, Rosenzweig, EB, Yung, D, Krishnan, U, Whalen, E, Bates, A, Avitabile, CM, Jackson, EO, Hirsch, R, Fineman, J, Abman, SH, For The Pediatric Pulmonary Hypertension Network (PPHNet). Building a dedicated pediatric pulmonary hypertension program: a consensus statement from the Pediatric Pulmonary Hypertension Network. Pulm Circ. 2022; 12:e12031. doi.org/10.1002/pul2.12031
- Mental Health in Pediatric Pulmonary Hypertension
Parker C, Whalen E, McSweeney J, Brown A, Raj JU, Mullen M. Perspectives on Mental Health Evaluation in Pediatric Pulmonary Hypertension: A Call to Action. Advances in Pulmonary Hypertension. 2022; 21(1): 12-15.
doi.org/10.21693/1933-088X-21.1.12
- The Spectrum of Pulmonary Hypertension in the PPHNet Registry
Abman SH, Mullen MP, Sleeper LA, Austin ED, Rosenzweig EB, Kinsella JP, Ivy D, Hopper RK, Raj JU, Fineman J, Keller RL, Bates A, Krishnan US, Avitabile CM, Davidson A, Natter MD, Mandl KD. Pediatric Pulmonary Hypertension Network. Characterization of paediatric pulmonary hypertensive vascular disease from the PPHNet Registry. Eur Respir J. 2021 Dec 31;59(1):2003337. https://erj.ersjournals.com/content/59/1/2003337.short
- Adverse Drug Events in Pediatric Pulmonary Hypertension
Geva A, Abman SH, Manzi SF, Ivy DD, Mullen MP, Griffin J, Lin C, Savova GK, Mandl KD. Adverse drug event rates in pediatric pulmonary hypertension: a comparison of real-world data sources. J Am Med Inform Assoc. 2020 Feb 1;27(2):294-300.
- Maintenance and Pain Control for Subcutaneous Treprostinil Sites in Pediatric Patients
Jackson EO, Brown A, McSweeney J, Parker C. Pediatric subcutaneous treprostinil site maintenance and pain control strategies from the Pediatric Pulmonary Hypertension Network. Pulm Circ. 2021 Mar 2;11(1):2045894021994450.
doi.org/10.1177/2045894021994450
- Variations in Therapy for Pulmonary Hypertension Secondary to Left Heart Disease in Children
Nawaytou H, Fineman JR, Moledina S, Ivy D, Abman SH, Cerro MJD. Practice patterns of pulmonary hypertension secondary to left heart disease among pediatric pulmonary hypertension providers. Pulm Circ. 2021 Feb 9;11(1)
doi.org/10.1177/2045894021991446
- Clinical Presentation of TBX4 Gene Mutations and Deletions in Children
Galambos C, Mullen MP, Shieh JT, Schwerk N, Kielt MJ, Ullmann N, Boldrini R, Stucin-Gantar I, Haass C, Bansal M, Agrawal PB, Johnson J, Peca D, Surace C, Cutrera R, Pauciulo MW, Nichols WC, Griese M, Ivy D, Abman SH, Austin ED, Danhaive O. Phenotype characterisation of TBX4 mutation and deletion carriers with neonatal and paediatric pulmonary hypertension. Eur Respir J. 2019 Aug 22;54(2)
https://erj.ersjournals.com/content/54/2/1801965.short
- Racial and Ethnic Diversity in Pediatric Pulmonary Hypertension
Ong MS, Abman S, Austin ED, Feinstein JA, Hopper RK, Krishnan US, Mullen MP, Natter MD, Raj JU, Rosenzweig EB, Mandl KD; Pediatric Pulmonary Hypertension Network and National Heart, Lung, and Blood Institute Pediatric Pulmonary Vascular Disease Outcomes Bioinformatics Clinical Coordinating Center Investigators. Racial and Ethnic Differences in Pediatric Pulmonary Hypertension: An Analysis of the Pediatric Pulmonary Hypertension Network Registry. J Pediatr. 2019 Aug;211:63-71.e6. doi.org/10.1016/j.jpeds.2019.04.046
- The Left Ventricle in Congenital Diaphragmatic Hernia and Implications for Pulmonary Hypertension Management
Kinsella JP, Steinhorn RH, Mullen MP, et al. The Left Ventricle in Congenital Diaphragmatic Hernia: Implications for the Management of Pulmonary Hypertension. J Pediatrics, Vol 197, June 2018 P17-22.
doi.org/10.1016/j.jpeds.2018.02.040
- Comparison Between Registry Data Collection and Electronic Health Record In Pediatric Pulmonary Hypertension
Geva A, Gronsbell JL, Cai T, Cai T, Murphy SN, Lyons JC, Heinz MM, Natter MD, Patibandla N, Bickel J, Mullen MP, Mandl KD; Pediatric Pulmonary Hypertension Network and National Heart, Lung, and Blood Institute Pediatric Pulmonary Vascular Disease Outcomes Bioinformatics Clinical Coordinating Center Investigators. A Computable Phenotype Improves Cohort Ascertainment in a Pediatric Pulmonary Hypertension Registry. J Pediatr. 2017 Sep;188:224-231.e5.
doi.org/10.1016/j.jpeds.2017.05.037
- Pulmonary Hypertension In Bronchopulmonary Dysplasia
Krishnan U, Feinstein JA, Adatia I, et al. Evaluation and Management of Pulmonary Hypertension in Children with Bronchoplumonary Dysplasia. J Pediatrics. Volume 188, 24-34.
doi.org/10.1016/j.jpeds.2017.05.029
- Use of Inhaled Nitric Oxide in Premature Newborns with Pulmonary Hypertension
Kinsella JP, Steinhorn RH, Krishnan US, et al. Recommendations for the Use of Inhaled Nitric Oxide Therapy in Premature Newborns with Severe Pulmonary Hypertension. J Pediatr 2016. 170:312-314.
https://doi.org/10.1016/j.jpeds.2015.11.050
- Pediatric Pulmonary Hypertension Guidelines
Abman SH, Hansmann G, Archer SL, et al. Pediatric Pulmonary Hypertension: Guidelines From the American Heart Association and American Thoracic Society. Circulation 2015; 132(21): 2037-2099.
doi.org/10.1161/CIR.0000000000000329
- Implications of the Warning on the Use of Sildenafil in the Treatment of Pediatric Pulmonary Hypertension
Abman SH, Kinsella JP, Rosenzweig EB, et al. Implications of the U.S. Food and Drug Administration Warning against the Use of Sildenafil for the Treatment of Pediatric Pulmonary Hypertension. American Journal of Respiratory and Critical Care Medicine, Vol. 187, No. 6 (2013), pp. 572-575.
doi.org/10.1164/rccm.201210-1928PP
