Felix R. De Bie, Catherine M. Avitabile, Sabrina Flohr, Sierra Land, Leny Mathew, Yan Wang, Devon Ash, Natalie E. Rintoul, Holly L. Hedrick
Children’s Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine. Cluster Woman and Child.
United States and Belgium
Journal of Pediatrics
J Pediatr 2023;
DOI: 10.1016/j.jpeds.2023.113420
Abstract
Objective: To describe our experience with treprostinil, evaluate correlations with cardiac function, and assess for adverse effects in neonates with congenital diaphragmatic hernia-related pulmonary hypertension (CDH-PH).
Study design: A retrospective review of a single-center prospective registry at a quaternary care children’s hospital. Patients included in the study had CDH-PH treated with treprostinil between April 2013 and September 2021. Assessed outcomes were brain-type natriuretic peptide levels and quantitative echocardiographic parameters collected at baseline, 1 week, 2 weeks, and 1 month after treprostinil initiation. Right ventricular (RV) function was assessed by tricuspid annular plane systolic excursion Z-score and speckle tracking echocardiography (global longitudinal and free wall strain). Septal position and left ventricular (LV) compression were assessed by eccentricity index and M-mode Z-scores.
Results: Fifty-one patients were included, with an average expected/observed lung-to-head ratio of 28.4 ± 9.0%. Most patients required extra-corporeal membrane oxygenation (n = 45, 88%). Survival to hospital discharge was 31/49 (63%). Treprostinil was initiated at a median age of 19 days with a median effective dose of 34 ng/kg/minute. Median baseline brain-type natriuretic peptide level decreased from 416.9 pg/mL to 120.5 pg/mL after 1 month. Treprostinil was associated with improved tricuspid annular plane systolic excursion Z-score, RV global longitudinal strain, RV free wall strain, LV eccentricity index, and LV diastolic and systolic dimensions, reflecting less compression by the RV, regardless of ultimate patient survival. No serious adverse effects were recorded.
Conclusions: In neonates with CDH-PH, treprostinil administration is well tolerated and is associated with improved RV size and function.
Category
Class III. Pulmonary Hypertension Associated with Lung Hypoplasia
Diagnostic Testing for Pulmonary Vascular Disease. Non-invasive Testing
Medical Therapy. Efficacy or Lack of Efficacy
Medical Therapy. Adverse Effects or Lack of Efficacy
Age Focus: Pediatric Pulmonary Vascular Disease
Fresh or Filed Publication: Filed (PHiled). Greater than 1-2 years since publication
Article Access
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