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Published on: May 11, 2015
Children with pulmonary arterial hypertension and prostanoid therapy: long-term hemodynamics
Stephanie L Siehr1, D Dunbar Ivy, Kathleen Miller-Reed
1Division of Pediatric Cardiology, Department of Pediatrics, Stanford University, Palo Alto, California 94304, USA.
Insights
Pediatric pulmonary arterial hypertension (PAH) patients showed improved hemodynamics on epoprostenol and treprostinil, but gains were not sustained long-term. Five-year transplant-free survival was promising, exceeding adult study outcomes.
Area of Science:
- Pediatric Cardiology
- Pulmonary Hypertension Research
- Vascular Hemodynamics
Background:
- Severe pulmonary arterial hypertension (PAH) in children necessitates advanced treatments like intravenous epoprostenol or treprostinil.
- Limited data exists on the long-term hemodynamic effects and patient outcomes associated with these prostanoid therapies in pediatric PAH.
- Understanding treatment transitions, particularly from epoprostenol to treprostinil, is crucial for optimizing pediatric PAH management.
Purpose of the Study:
- To evaluate the longitudinal hemodynamic changes and clinical outcomes in pediatric patients with severe PAH treated with epoprostenol and treprostinil.
- To compare the effectiveness of epoprostenol and treprostinil in managing pediatric PAH.
- To investigate the outcomes of pediatric patients transitioning from epoprostenol to treprostinil therapy.
Main Methods:
- Retrospective analysis of 77 pediatric patients diagnosed with idiopathic PAH or congenital heart disease-associated PAH between 1992 and 2010.
- Patients received either epoprostenol or treprostinil, with a subgroup transitioning between therapies.
- Hemodynamic parameters, including pulmonary-to-systemic vascular resistance ratio (Rp/Rs), were tracked longitudinally; outcomes were defined as survival or need for transplant.
Main Results:
- Both epoprostenol and treprostinil demonstrated initial improvements in Rp/Rs within 1-2 years, though these benefits were not sustained.
- Patients treated with treprostinil showed a statistically significant reduction in Rp/Rs at 2 years compared to baseline.
- The 5-year transplant-free survival rate for the cohort was 70%, which is notably better than reported outcomes in similar adult studies.
Conclusions:
- While both epoprostenol and treprostinil offer temporary hemodynamic improvements in pediatric PAH, long-term efficacy requires further investigation.
- The observed 5-year transplant-free survival rate suggests that current prostanoid therapies can be effective in improving long-term outcomes for pediatric PAH patients.
- Baseline hemodynamic severity significantly impacts short-term outcomes, highlighting the need for early and aggressive intervention in pediatric PAH.
Background:
Pediatric patients with severe pulmonary arterial hypertension (PAH) are treated with intravenous epoprostenol or intravenous or subcutaneous treprostinil. Little is known about longitudinal hemodynamics and outcomes of epoprostenol, treprostinil, and transitions from epoprostenol to treprostinil.
Methods:
This was retrospective study of 77 pediatric patients (47 idiopathic PAH, 24 congenital heart disease-PAH) receiving epoprostenol or treprostinil from 1992 to 2010 at 2 centers. Outcomes were defined as living vs dead/transplant.
Results:
Mean age at baseline was 7.7 ± 5.2 years, with follow-up of 4.3 ± 3.4 years. Thirty-seven patients were treated with epoprostenol, 20 with treprostinil, and 20 were transitioned from epoprostenol to treprostinil. Mean pulmonary-to-systemic vascular resistance ratio (Rp/Rs) for epoprostenol was 1.0 ± 0.4, 0.8 ± 0.4, 0.8 ± 0.4, 1.0 ± 0.4, and 1.2 ± 0.4, respectively, at baseline, 1, 2, 3, and 4 years. For treprostinil, Rp/Rs was 0.9 ± 0.3, 0.7 ± 0.3, 0.5 ± 0.2, (p < 0.01 vs baseline), and 1.1 ± 0.2, respectively, at baseline, 1, 2, and 3 to 4 years, respectively. There were similar changes in mean pulmonary artery pressure and pulmonary vascular resistance index. The Rp/Rs 1 year after epoprostenol to treprostinil transition increased from 0.6 to 0.8 (n = 7). Changes not statistically significant unless noted. Eight patients died or received a transplant within 2 years of baseline; compared with the rest of the cohort, mean baseline Rp/Rs, right atrial pressure, and pulmonary vascular resistance index were significantly worse in this group. Thirty-nine patients remain on prostanoids, 17 are off, 16 died, and 5 received heart-lung transplant. Kaplan-Meier 5-year transplant-free survival was 70% (95% confidence interval, 56%-80%).
Conclusion:
There was improvement in Rp/Rs on both therapies at 1 to 2 years that was not sustained. The 5-year transplant-free survival was better than in similar adult studies.
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