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Published on: May 11, 2015
Treprostinil Effectiveness in Higher-Risk Pediatric Patients With Idiopathic and Heritable Pulmonary Arterial
Yuan He1, Qiangqiang Li1, Chen Zhang1
1Department of Pediatric Cardiology, Beijing Anzhen Hospital, Capital Medical University, Beijing, China.
Insights
Treprostinil therapy significantly improved survival for higher-risk pediatric patients with idiopathic or heritable pulmonary arterial hypertension (IPAH/HPAH). This treatment offers better outcomes regardless of the patient's specific genetic mutations.
Area of Science:
- Pediatric Cardiology
- Pulmonary Hypertension Research
- Genetic Basis of Cardiovascular Disease
Background:
- Effectiveness of treprostinil in higher-risk pediatric pulmonary arterial hypertension (PAH) patients with diverse genotypes is not well understood.
- Idiopathic or heritable PAH (IPAH/HPAH) presents unique challenges in pediatric populations.
Purpose of the Study:
- To investigate the prognostic impact of treprostinil therapy in higher-risk pediatric patients diagnosed with IPAH/HPAH.
- To analyze survival rates and clinical improvements in this specific patient group.
Main Methods:
- A cohort of higher-risk pediatric IPAH/HPAH patients treated with treprostinil was compared to a reference cohort on oral medications only.
- Genetic testing for PAH-related mutations was performed on the treprostinil cohort.
- Survival and event-free survival (death, Potts shunt, atrial septostomy) were the primary endpoints.
Main Results:
- The treprostinil cohort (n=49) showed significantly superior survival rates compared to the reference cohort (n=48) (P=0.038).
- All patients in the treprostinil cohort survived with improved clinical characteristics after a median of 5.56 months of therapy.
- One-, 2-, and 3-year survival rates were 91%, 84%, and 69%, respectively, with no significant difference in survival across different genotypes.
Conclusions:
- Treprostinil therapy demonstrates a significant positive impact on the prognosis of higher-risk pediatric IPAH/HPAH patients.
- The benefits of treprostinil are observed irrespective of the underlying genetic mutations in these children.
Background:
Little is known about the effectiveness of treprostinil in higher-risk paediatric patients with various pulmonary arterial hypertension genotypes. This study was designed to investigate the prognosis of higher-risk paediatric patients with idiopathic or heritable pulmonary arterial hypertension (IPAH/HPAH) after treprostinil therapy.
Methods:
Children with IPAH/HPAH who were stratified as higher risk and treated with treprostinil in our centre were included as the study cohort. Those who received only oral medications were included as the reference cohort. All patients in the study cohort received PAH-related genotyping. Survival was defined as no death. Event-free survival was defined as no death, Potts shunt, or atrial septostomy.
Results:
Forty-nine children (median age 7.7 years [interquartile range (IQR) 4.2-11.5 years], 65% female) were included in the study cohort and 48 children were included in the reference cohort; 84% of the study cohort had genetic disorders after genetic testing with a dominance of BMPR2 and ACVRL1 mutations. After a median therapy duration of 5.56 months (IQR 2.66-11.12 months), all patients were alive with significant improvements in clinical characteristics. One-, 2-, and 3-year survival rates were 91%, 84%, and 69%, respectively with a median follow-up duration of 19.17 months (IQR 9.7-29.79 months), which was significantly superior to the reference cohort (P = 0.038). Multivariate Cox regression analysis identified World Health Organisation functional class after therapy as a predictor for survival. There was no significant difference in survival among patients with different genotypes.
Conclusions:
Treprostinil can significantly improve the prognosis in children with IPAH/HPAH who are at higher risk, despite genetic backgrounds.
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