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Acute Vasodilator Response in Pediatric Pulmonary Arterial Hypertension: Current Clinical Practice From the TOPP
Johannes M Douwes1, Tilman Humpl2, Damien Bonnet3
1Centre for Congenital Heart Diseases, Pediatric Cardiology, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands.
Insights
Acute vasodilator response testing (AVT) in pediatric pulmonary arterial hypertension (PAH) shows varied physician assessment compared to established criteria. The Sitbon criteria best identify pediatric idiopathic PAH patients for calcium-channel blocker (CCB) therapy and improved survival.
Area of Science:
- Pediatric Cardiology
- Pulmonary Hypertension Research
- Clinical Practice Guidelines
Background:
- Acute vasodilator response testing (AVT) is crucial for identifying adult pulmonary arterial hypertension (PAH) patients who benefit from calcium-channel blocker (CCB) therapy.
- However, established criteria for AVT response and CCB use in pediatric PAH are not well-defined or studied.
Purpose of the Study:
- To delineate current clinical practices for AVT in pediatric PAH.
- To analyze subsequent treatment decisions, including CCB therapy, based on AVT results in children.
Main Methods:
- Analysis of 529 children with pulmonary hypertension from an international registry (2008-2013).
- Focus on 382 children with evaluable AVT, including idiopathic/familial PAH (IPAH/FPAH) and PAH associated with congenital heart disease (PAH-CHD).
- Comparison of physician-judged AVT response with REVEAL-pediatric and Sitbon criteria.
Main Results:
- Physician assessment of AVT response in IPAH/FPAH varied significantly from REVEAL-pediatric and Sitbon criteria (37% vs. 30% vs. 15%).
- Similar discrepancies were observed in PAH-CHD patients (36% vs. 13% vs. 7%).
- Poor correlation existed between physician judgment and established criteria; only 23% of physician-identified responders received CCB monotherapy. Sitbon criteria identified patients with superior outcomes on CCB therapy.
Conclusions:
- Current AVT interpretation and CCB treatment practices in pediatric PAH deviate from international guidelines.
- The Sitbon criteria are recommended for identifying pediatric IPAH patients who achieve excellent survival with CCB therapy.
Background:
In pulmonary arterial hypertension (PAH), acute vasodilator response testing (AVT) is considered important to identify adult patients with favorable prognosis using calcium-channel blocker (CCB) therapy. However, in pediatric PAH, criteria used to identify acute responders and CCB use are insufficiently studied.
Objectives:
This study sought to describe current clinical practice of AVT and subsequent treatment decisions in pediatric PAH.
Methods:
From January 2008 to May 2013, 529 consecutive children with confirmed pulmonary hypertension were enrolled in an international registry. We analyzed those children with evaluable AVT.
Results:
Of 382 children with evaluable AVT, 212 had idiopathic/familial PAH (IPAH/FPAH) and 105 had PAH associated with congenital heart disease (PAH-CHD). In 70% of the patients, AVT was performed using inhaled nitric oxide; other agents were used in the remaining patients. In IPAH/FPAH patients, 78 (37%) patients were acute responders according to their physician, 62 (30%) according to REVEAL (Registry-to-Evaluate-Early-And-Long-term PAH disease management)-pediatric criteria, and 32 (15%) according to Sitbon criteria. For PAH-CHD patients, the numbers of AVT responders were 38 (36%), 14 (13%), and 7 (7%) respectively. Correlation between AVT responder status as judged by the treating physician and by published response criteria was poor. Moreover, of the IPAH/FPAH patients judged by the treating physician as acute responders, only 23% were treated with CCB without additional PAH-targeted therapy. The Sitbon criteria selected patients with better prognosis who had excellent outcome when treated with CCB.
Conclusions:
The current practice of identifying responders to AVT and subsequent treatment with CCB therapy demonstrated large discrepancies with current international guidelines. Also, in pediatric IPAH, the Sitbon criteria are the criteria of choice to identify patients with excellent survival when treated with CCB therapy.
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