MiscellaneousEffectiveness and Safety of Inhaled Treprostinil for the Treatment of Pulmonary Arterial Hypertension in Children
Section snippets
Methods
To evaluate the safety, efficacy, and tolerability of inhaled treprostinil in children, we retrospectively reviewed the data from all patients with group 1 PAH treated with inhaled treprostinil for ≥6 weeks at 2 large pediatric pulmonary hypertension centers (Columbia University Medical Center and Children's Hospital Colorado). Indications for initiation of inhaled treprostinil therapy included symptomatic PAH on background therapy or as a strategy to transition select patients off parenteral
Results
Twenty-nine consecutive pediatric patients with Group 1 PAH who received inhaled treprostinil for ≥6 weeks were included. Baseline demographic data are listed in Table 2. There were 14 male and 15 female subjects (mean age 11.3 ± 4.5 years, median 12, range 3.2 to 19) with idiopathic PAH (n = 19) or associated PAH (n = 10). Average treatment duration was 15.7 ± 8.2 months (median 17, range 1.5 to 27.5). All patients were on background therapy. Four patients were on calcium channel blockers, 26
Discussion
Inhaled iloprost was initially studied in children with PAH in 22 pediatric patients and was found to have acceptable safety and tolerability.20 Iloprost therapy requires treatments every 2 to 4 hours, with each treatment taking 5 to 20 minutes. Delivery of inhaled iloprost can be difficult for young children because of the need for actuated breaths with the Opti-Neb device. Availability of a longer-acting inhaled prostanoid, inhaled treprostinil, has enabled patients to transition from
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Targeted Therapies for Neonatal Pulmonary Hypertension: Beyond Nitric Oxide
2024, Clinics in PerinatologyEisenmenger Syndrome: JACC State-of-the-Art Review
2022, Journal of the American College of CardiologyCitation Excerpt :Pediatric PAH-CHD occurs in a heterogeneous group of patients with various presentations and phenotypes, with overall worse survival among children with ES when compared with adults.50 There are scarce data on the safety and efficacy of advanced PAH therapies in children with ES, based mainly on adult RCTs including children ≥12 years of age9,10 or RCTs on pediatric patients with PAH.97,98 Large-scale RCTs on pediatric patients with ES are needed, while the heterogeneity of PAH-CHD phenotypes in children and the complexity of ES clearly warrant tertiary diagnosis and treatment, which can only be offered in pediatric PAH centers.
Management of Pulmonary Hypertension in the Pediatric Patient
2022, Cardiology ClinicsCitation Excerpt :Direct airway delivery is also thought to cause less V-Q mismatch. Inhaled iloprost has a short half-life of 20 to 25 minutes and must be given every 1 to 4 hours,43 whereas inhaled treprostinil with its longer half-life can be administered every 6 hours and has been shown to improve exercise capacity and is safe and well tolerated in the pediatric population.63 Similar to cancer therapy experience, combination therapy to target multiple pathways simultaneously has shown benefit in PH therapy.
Pediatric Pulmonary Arterial Hypertension
2020, Pediatric Clinics of North America35 - Childhood Pulmonary Arterial Hypertension
2019, Kendig's Disorders of the Respiratory Tract in ChildrenAn update on the diagnosis and management of bronchopulmonary dysplasia (BPD)-associated pulmonary hypertension
2018, Seminars in PerinatologyCitation Excerpt :Subcutaneous treprostinil has been used to transition some children who were chronically stable on IV epoprostenol,91 and may be effective when added to other vasodilator therapies.92 Preliminary studies of treprostinil imply an acceptable safety profile and clinical benefit in pediatric patients with PH,93 and case series describe dramatic improvement in premature infants who were non-responsive to nitric oxide.90,94 Nitric oxide(NO) is a biological signaling gas molecule synthesized by the enzyme NO synthase (NOS).
This study was funded by Specialized Centers of Clinically Oriented Research Grant HL-084923-02 from the National Institutes of Health, Bethesda, Maryland, Grant UL1RR025780 from the National Institutes of Health/National Center for Research Resources Colorado CTSI, Denver, Colorado, the Jayden DeLuca Foundation, and the Leah Bult PH Research Fund, Denver, Colorado. Columbia University and University of Colorado receive consultant fees from Actelion, Gilead, Pfizer, and United Therapeutics.