title: Treatment initiation in paediatric pulmonary hypertension: insights from a multinational registry.
abstract: Different treatment options for pulmonary hypertension have emerged in recent years, and evidence-based management strategies have improved quality of life and survival in adults. In children with pulmonary vascular disease, therapeutic algorithms are not so clearly defined; this study determined current treatment initiation in children with pulmonary hypertension in participating centres of a registry. Through the multinational Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension registry, patient demographics, diagnosis, and treatment as judged and executed by the local physician were collected. Inclusion criteria were >3 months and <18 years of age and diagnostic cardiac catheterisation consistent with pulmonary hypertension (mean pulmonary arterial pressure ⩾25 mmHg, pulmonary vascular resistance index ⩾3 Wood units×m2, and mean pulmonary capillary wedge pressure ⩽12 mmHg). At diagnostic catheterisation, 217/244 patients (88.9%) were treatment naïve for pulmonary hypertension-targeted therapy. Targeted therapy was initiated after catheterisation in 170 (78.3%) treatment-naïve patients. A total of 19 patients received supportive therapy, 28 patients were not started on therapy, and 26 patients (10.7%) were on targeted treatment before catheterisation. Among treatment-naïve subjects, treatment was initiated with one targeted drug (n=112, 51.6%), dual therapy (n=39, 18%) or triple-therapy (n=5, 2.3%), and calcium channel blockers with one targeted medication in one patient (0.5%). Phosphodiesterase inhibitors type 5 were used frequently; some patients with pulmonary hypertension related to lung disease received targeted therapy. There is a diverse therapeutic approach for children with pulmonary hypertension with a need of better-defined treatment algorithms based on paediatric consensus for different aetiologies including the best possible diagnostic workup.
authors.affiliation: 1Department of Critical Care Medicine,Division of Cardiology,Hospital for Sick Children,Toronto,Canada.
authors.forename: Tilman
authors.initials: T
authors.lastname: Humpl
authors.affiliation: 2Department of Pediatrics,University Medical Center Groningen,Groningen,The Netherlands.
authors.forename: Rolf M F
authors.initials: RMF
authors.lastname: Berger
authors.affiliation: 3Division of Pulmonary Medicine,Vanderbilt Children's Hospital,Nashville,United States of America.
authors.forename: Eric D
authors.initials: ED
authors.lastname: Austin
authors.affiliation: 4Klinik für Kinder- und Jugendmedizin,Kantonsspital Winterthur,Winterthur,Switzerland.
authors.forename: Margrit S
authors.initials: MS
authors.lastname: Fasnacht Boillat
authors.affiliation: 5Paediatric Cardiology,Université Paris Descartes,Necker Enfants Malades,Paris,France.
authors.forename: Damien
authors.initials: D
authors.lastname: Bonnet
authors.affiliation: 6Department of Pediatrics,Section of Cardiology,University of Colorado,Aurora,United States of America.
authors.forename: Dunbar D
authors.initials: DD
authors.lastname: Ivy
authors.affiliation: 7Department of Cardiology,Children's Memorial Health Institute,Warsaw,Poland.
authors.forename: Malgorzata
authors.initials: M
authors.lastname: Zuk
authors.affiliation: 8Children's University Hospital,Geneva,Switzerland.
authors.forename: Maurice
authors.initials: M
authors.lastname: Beghetti
authors.affiliation: 9Great Ormond Street Hospital for Sick Children,London,United Kingdom.
authors.forename: Ingram
authors.initials: I
authors.lastname: Schulze-Neick
mesh_terms: D000293:Adolescent; D000959:Antihypertensive Agents / Q000627:therapeutic use; D002121:Calcium Channel Blockers / Q000627:therapeutic use*; D006328:Cardiac Catheterization / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D006976:Hypertension, Pulmonary / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D007223:Infant; D008297:Male; D058986:Phosphodiesterase 5 Inhibitors / Q000627:therapeutic use*; D011379:Prognosis; D011652:Pulmonary Circulation; D011669:Pulmonary Wedge Pressure / Q000502:physiology*; D012042:Registries; D014665:Vasodilator Agents / Q000627:therapeutic use*
chemical_list: D000959:Antihypertensive Agents; D002121:Calcium Channel Blockers; D058986:Phosphodiesterase 5 Inhibitors; D014665:Vasodilator Agents
keywords: Pulmonary hypertension; children; treatment