. 2023 Apr 12;113420.
doi: 10.1016/j.jpeds.2023.113420. Online ahead of print. https://pubmed.ncbi.nlm.nih.gov/37059388/
Treprostinil in neonates with congenital diaphragmatic hernia-related pulmonary hypertension
Felix R De Bie 1, Catherine M Avitabile 2, Sabrina Flohr 3, Sierra Land 3, Leny Mathew 3, Yan Wang 4, Devon Ash 4, Natalie E Rintoul 5, Holly L Hedrick 3
- PMID: 37059388
- DOI: 10.1016/j.jpeds.2023.113420
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 (BNP) levels and quantitative echocardiographic parameters collected at baseline, one week, two weeks, and one month after treprostinil initiation. Right ventricular (RV) function was assessed by tricuspid annular plane systolic excursion Z-score (TAPSEZ) and speckle tracking echocardiography [global longitudinal (GLS) and free wall strain (FWS)]. Septal position and left ventricular (LV) compression were assessed by eccentricity index (LVEI) 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/min. Median baseline BNP level decreased from 416.9 pg/mL to 120.5 pg/mL after one month. Treprostinil was associated with improved TAPSEZ, RVGLS, RVFWS, LVEI, 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.
Keywords: Bochdalek; Prostacyclin.
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