Abstract
Treatment strategies in paediatric pulmonary arterial hypertension (PAH) have evolved over the last years, but survival is still poor. Recently, in adults with severe PAH, upfront triple combination therapy (uTCT) from diagnosis has been reported to show significant clinical improvement and excellent long-term outcome. This retrospective, observational study aimed to assess the efficacy of uTCT in paediatric PAH.
Children diagnosed with PAH between 2010 and 2019 and started with uTCT were included. World Health Organization Functional Class (WHO-FC), haemodynamics, echocardiography, 6-min walking distance and serum level of N-terminal pro-brain-natriuretic-peptide were assessed at baseline, after 3 and 6 months and at last available follow-up. Events were defined as death, lung transplantation or Potts shunt.
21 children (median age 4.8 years (2.5–12.8), 57% females) were included. All children except one were in WHO-FC III or IV (28% and 67%, respectively). After 3 months, one child had died and one child had received a Potts shunt. The remaining 19 children showed clinical and echocardiographic improvement, which persisted at 6 months. Children with idiopathic and heritable PAH showed one-, two- and three-year transplant-free survival estimates of 100%, 94% and 87%, albeit 47% of them receiving a Potts shunt during follow-up.
Children with severe PAH, but not pulmonary veno-occlusive disease, improved significantly with uTCT and showed beneficial up to 3-year survival rates, albeit 47% of them receiving a Potts shunt during follow-up. The role of a Potts shunt in conjunction to uTCT in paediatric PAH needs to be further established.
Abstract
Upfront triple combination therapy in severe paediatric PAH resulted in significant clinical, haemodynamic and echocardiographic improvement and favourable 1-, 2- and 3-year survival rates, albeit with 47% receiving a Potts shunt during follow-up https://bit.ly/3iG92PA
Footnotes
This article has an editorial commentary: https://doi.org/10.1183/13993003.04258-2020
Conflict of interest: M.G. Haarman has nothing to disclose.
Conflict of interest: M. Lévy has nothing to disclose.
Conflict of interest: M.T.R. Roofthooft has nothing to disclose.
Conflict of interest: J.M. Douwes has nothing to disclose.
Conflict of interest: T.R. Vissia-Kazemier has nothing to disclose.
Conflict of interest: I. Szezepanski has nothing to disclose.
Conflict of interest: R.M.F. Berger reports that the University Medical Center Groningen contracts with Actelion, Lilly and GSK for advisory board and steering committee activities, outside the submitted work.
Conflict of interest: D. Bonnet reports personal fees for advisory board and steering committee work from Actelion Pharmaceuticals/Janssen, Novartis, Bayer Healthcare and Eli Lilly, personal fees for advisory board work from BMS, during the conduct of the study; non-financial support for meeting attendance from Abbott, outside the submitted work.
Support statement: This study was supported by the Sebald Fund and by Association pour la Recherche en Cardiologie du Foetus à l'Adulte (ARCFA). Funding information for this article has been deposited with the Crossref Funder Registry.
- Received April 10, 2020.
- Accepted August 10, 2020.
- Copyright ©ERS 2021