title: Safety and tolerability of subcutaneous treprostinil in newborns with congenital diaphragmatic hernia and life-threatening pulmonary hypertension.
abstract: BACKGROUND
  Prolonged pulmonary hypertension (PH) is highly predictive for pulmonary morbidity and death in infants with congenital diaphragmatic hernia (CDH).
  
  
  OBJECTIVE
  To report the effects and tolerability of subcutaneous treprostinil in newborns with severe CDH and late life-threatening PH.
  
  
  METHODS
  We recorded clinical and echocardiography data before and after starting subcutaneous treprostinil, on patients with severe CDH and late PH, refractory to inhaled nitric oxide and oral sildenafil.
  
  
  RESULTS
  14 patients were treated with treprostinil (gestational age: 39.1±2.0weeks; birth weight: 3200±600g). Prior to treatment, the pre- and post-ductal SpO2 difference (Δ SpO2) was 14±10%. Treprostinil was initiated at a median age of 12days [5-157]. After starting treprostinil, ΔSpO2 decreased to 3% at day 7 (p<0.05), and the mean blood flow velocities in the right pulmonary arteries increased by 110% (p<0.05). 2 of the 14 patients died. At the age of follow up (12months to 3years), the 12 surviving infants were all weaned from respiratory support and discharged home.
  
  
  CONCLUSIONS
  The subcutaneous treprostinil improves pulmonary hemodynamics and outcomes in infants with CDH and life-threatening PH. We suggest that the treatment should be considered in infants with severe CDH and late PH.
  
  
  METHODS
  Case series with no comparison group.
  
  
  METHODS
  Level IV.
authors.affiliation: Department of Neonatology, University Hospital of Lille, 2 Avenue Oscar Lambret, 59037 Lille, France,; Department of Neonatology, University Hospital of Amiens, Place Laennec, 80054 Amiens, Cédex 1, France; PériTox Laboratory (UMI- 01), University of Amiens, UPJV, Place Laennec, 80054 Amiens, Cédex 1, France.
authors.forename: E
authors.initials: E
authors.lastname: Carpentier
authors.affiliation: Department of Neonatology, University Hospital of Lille, 2 Avenue Oscar Lambret, 59037 Lille, France.
authors.forename: S
authors.initials: S
authors.lastname: Mur
authors.affiliation: Department of Pediatric Surgery, University Hospital of Lille, 2 Avenue Oscar Lambret, 59037 Lille, France; EA4489, Perinatal Environment and Health, FHU 1000 days for Health, University Lille - Nord de France, 2, Avenue Oscar Lambret, 59037 Lille, France.
authors.forename: E
authors.initials: E
authors.lastname: Aubry
authors.affiliation: Department of Neonatology, University Hospital of Lille, 2 Avenue Oscar Lambret, 59037 Lille, France.
authors.forename: L
authors.initials: L
authors.lastname: Pognon
authors.affiliation: Department of Neonatology, University Hospital of Lille, 2 Avenue Oscar Lambret, 59037 Lille, France,; EA4489, Perinatal Environment and Health, FHU 1000 days for Health, University Lille - Nord de France, 2, Avenue Oscar Lambret, 59037 Lille, France.
authors.forename: T
authors.initials: T
authors.lastname: Rakza
authors.affiliation: Department of Neonatology, University Hospital of Lille, 2 Avenue Oscar Lambret, 59037 Lille, France,; EA4489, Perinatal Environment and Health, FHU 1000 days for Health, University Lille - Nord de France, 2, Avenue Oscar Lambret, 59037 Lille, France.
authors.forename: F
authors.initials: F
authors.lastname: Flamein
authors.affiliation: Department of Pediatric Surgery, University Hospital of Lille, 2 Avenue Oscar Lambret, 59037 Lille, France; EA4489, Perinatal Environment and Health, FHU 1000 days for Health, University Lille - Nord de France, 2, Avenue Oscar Lambret, 59037 Lille, France.
authors.forename: D
authors.initials: D
authors.lastname: Sharma
authors.affiliation: Department of Neonatology, University Hospital of Amiens, Place Laennec, 80054 Amiens, Cédex 1, France; PériTox Laboratory (UMI- 01), University of Amiens, UPJV, Place Laennec, 80054 Amiens, Cédex 1, France. Electronic address: tourneux.pierre@chu-amiens.fr.
authors.forename: P
authors.initials: P
authors.lastname: Tourneux
authors.affiliation: Department of Neonatology, University Hospital of Lille, 2 Avenue Oscar Lambret, 59037 Lille, France,; EA4489, Perinatal Environment and Health, FHU 1000 days for Health, University Lille - Nord de France, 2, Avenue Oscar Lambret, 59037 Lille, France.
authors.forename: L
authors.initials: L
authors.lastname: Storme
mesh_terms: D000959:Antihypertensive Agents / Q000008:administration & dosage*; D004452:Echocardiography; D011464:Epoprostenol / Q000008:administration & dosage / Q000031:analogs & derivatives*; D005260:Female; D005500:Follow-Up Studies; D005865:Gestational Age; D065630:Hernias, Diaphragmatic, Congenital / Q000150:complications*; D006801:Humans; D006976:Hypertension, Pulmonary / Q000188:drug therapy* / Q000209:etiology*; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D011651:Pulmonary Artery / Q000187:drug effects
chemical_list: D000959:Antihypertensive Agents; D011464:Epoprostenol; C427248:treprostinil
keywords: Congenital diaphragmatic hernia; Infant; Newborn; Prostacyclin; Pulmonary hypertension