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Treprostinil Improves Persistent Pulmonary Hypertension Associated with Congenital Diaphragmatic Hernia
Kendall M Lawrence1, Holly L Hedrick2, Heather M Monk3
1Department of Pediatric General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA.
Insights
Continuous treprostinil improved pulmonary hypertension markers in infants with congenital diaphragmatic hernia (CDH). This treatment showed no significant side effects, though overall mortality remained high.
Area of Science:
- Neonatal Medicine
- Pediatric Cardiology
- Pulmonary Hypertension Research
Background:
- Congenital diaphragmatic hernia (CDH) is often associated with severe pulmonary hypertension in infants.
- Pulmonary hypertension in CDH poses significant risks and challenges in neonatal care.
- Effective management strategies for CDH-associated pulmonary hypertension are crucial.
Purpose of the Study:
- To evaluate the impact of continuous treprostinil on pulmonary hypertension severity markers in infants with CDH.
- To assess the safety and tolerability of treprostinil in this vulnerable patient population.
- To provide insights into treprostinil's efficacy for severe pulmonary hypertension in neonates with CDH.
Main Methods:
- Retrospective cohort study of infants with CDH-induced pulmonary hypertension treated with treprostinil (2011-2016).
- Pulmonary hypertension severity assessed via echocardiogram and serum B-type natriuretic peptide (BNP) levels.
- Statistical analysis included Fisher exact tests, Wilcoxon-rank sum tests, and mixed-effects models.
Main Results:
- Seventeen infants received treprostinil; 76.5% required extracorporeal support, longer hospital stays, and mechanical ventilation.
- Significant reductions in BNP levels were observed at 1 week and 1 month post-treprostinil initiation.
- Echocardiogram-assessed pulmonary hypertension severity improved significantly by 1 month, with no reported treprostinil-related adverse events.
Conclusions:
- Continuous treprostinil administration is associated with improved pulmonary hypertension severity in infants with CDH.
- Treprostinil demonstrated a favorable safety profile, with no significant adverse events noted.
- Despite improvements in pulmonary hypertension, overall mortality in this high-risk group remains a concern.
Objective:
To evaluate the effect of continuous treprostinil in infants with severe pulmonary hypertension associated with congenital diaphragmatic hernia (CDH) on specific markers of pulmonary hypertension severity and to report the safety and tolerability of treprostinil.
Study Design:
We conducted a retrospective cohort study of infants with CDH-associated pulmonary hypertension treated with treprostinil from January 2011 to September 2016. Severity of pulmonary hypertension was assessed by echocardiogram and serum B-type natriuretic peptide (BNP) by using time points before initiation and 24 hours, 1 week, and 1 month after treprostinil initiation. Fisher exact tests, Wilcoxon-rank sum tests, and mixed-effects models were used for analysis.
Results:
Seventeen patients were treated with treprostinil for a median of 54.5 days (IQR 44.3-110 days). Compared with the concurrent CDH population (n = 147), infants treated with treprostinil were more likely to require extracorporeal support (76.5% vs 25.2%, P < .0001), to have a longer hospital stay (144 vs 60 days, P < .0001), and to need longer mechanical ventilator support (76.5 vs 30.9 days, P < .0001). Following treprostinil initiation, there was a significant reduction in BNP at 1 week (1439 vs 393 pg/mL, P < .01) and 1 month (1439 vs 242 pg/mL, P = .01). Severity of pulmonary hypertension by echocardiogram improved at 1 month (OR 0.14, CI 95% 0.04-0.48, P = .002). Despite these improvements, overall mortality remained high (35%). There were no adverse events related to treprostinil, including no hypotension, hypoxia, or thrombocytopenia.
Conclusions:
In this cohort, treprostinil use was associated with improved severity of pulmonary hypertension assessed by echocardiogram and decreased BNP, with no significant side effects.
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