Long-term outcome of children with newly diagnosed pulmonary arterial hypertension: results from the global TOPP registry
Author:
Ploegstra Mark-Jan1ORCID, Ivy David Dunbar2ORCID, Beghetti Maurice3ORCID, Bonnet Damien4, Alehan Dursun5, Ablonczy Laszlo6, Mattos Sandra7, Bowers David8, Humpl Tilman9, Berger Rolf M F1ORCID, Weintraub Robert G, Geiger Ralf, Marx Manfred, Cools Bjorn, Mattos Sandra, Humpl Tilman, Jing Zhi-Cheng, Sondergaard Lars, Bonnet Damien, Apitz Christian, Hager Alfred, Ablonczy Laszlo, Milanesi Ornella, Saji Ben T, Pulido Tomás, Berger Rolf, Douwes Menno, Brun Henrik, Moll Jadwiga, Michalak Krzysztof W, Kawalec Wanda, Małgorzata Żuk, Beghetti MauriceORCID, Boillat Margrit Fasnacht, Olguntürk Rana, Kula Serdar, Alehan Dursun, Schulze-Neick Ingram, Barst Robyn, Atz Andrew M, Ivy D DunbarORCID, Day Ronald W, Austin Eric D, Moore J Donald, Feinstein Jeffrey A, Yung Delphine, Berger John T, Mallory George B,
Affiliation:
1. Center for Congenital Heart Diseases, Department of Pediatric Cardiology, Beatrix Children's Hospital, University of Groningen, University Medical Center Groningen , Hanzeplein 1, PO Box 30.001, 9700 RB Groningen , The Netherlands 2. Department of Pediatrics, Section of Pediatric Cardiology, University of Colorado School of Medicine, Children's Hospital Colorado Heart Institute , Aurora, CO , USA 3. Pediatric Cardiology Unit, Department of the Child and Adolescent, Children's University Hospital Geneva , Geneva , Switzerland 4. M3C-Necker, Hôpital Universitaire Necker-Enfants Malades, Université de Paris Cité , Paris , France 5. Hacettepe University Faculty of Medicine , Ankara , Turkey 6. Gottsegen National Cardiovascular Center , Budapest , Hungary 7. Maternal–Fetal Cardiac Unit, Royal Portuguese Hospital , Recife , Brazil 8. School of Engineering, Arts, Science & Technology , University of Suffolk, Ipswich , UK 9. The Hospital for Sick Children, Critical Care and Cardiology, University of Toronto , Toronto , Canada
Abstract
Abstract
Background and aims
The Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension (TOPP) registry is a global network established to gain insights into the disease course and long-term outcomes of paediatric pulmonary arterial hypertension (PAH). Previously published cohorts in paediatric PAH are obscured by survival bias due to the inclusion of both prevalent (previously diagnosed) and incident (newly diagnosed) patients. The current study aims to describe long-term outcome and its predictors in paediatric PAH, exclusively of newly diagnosed patients.
Methods and results
Five hundred thirty-one children with confirmed pulmonary hypertension, aged ≥3 months and <18 years, were enrolled in the real-world TOPP registry at 33 centres in 20 countries, from 2008 to 2015. Of these, 242 children with newly diagnosed PAH with at least one follow-up visit were included in the current outcome analyses. During long-term follow-up, 42 (17.4%) children died, 9 (3.7%) underwent lung transplantation, 3 (1.2%) atrial septostomy, and 9 (3.7%) Potts shunt palliation (event rates: 6.2, 1.3, 0.4, and 1.4 events per 100 person-years, respectively). One-, three-, and five-year survival free from adverse outcome was 83.9%, 75.2%, and 71.8%, respectively.
Overall, children with open (unrepaired or residual) cardiac shunts had the best survival rates. Younger age, worse World Health Organization functional class, and higher pulmonary vascular resistance index were identified as independent predictors of long-term adverse outcome. Younger age, higher mean right atrial pressure, and lower systemic venous oxygen saturation were specifically identified as independent predictors of early adverse outcome (within 12 months after enrolment).
Conclusion
This comprehensive analysis of survival from time of diagnosis in a large exclusive cohort of children newly diagnosed with PAH describes current-era outcome and its predictors.
Funder
Actelion Pharmaceuticals Ltd
Publisher
Oxford University Press (OUP)
Subject
Cardiology and Cardiovascular Medicine,Health Policy
Cited by
2 articles.
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