E Carpentier1, S Mur2, E Aubry3, L Pognon2, T Rakza4, F Flamein4, D Sharma3, P Tourneux5, L Storme4. 1. 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. 2. Department of Neonatology, University Hospital of Lille, 2 Avenue Oscar Lambret, 59037 Lille, France. 3. 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. 4. 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. 5. 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.
Abstract
BACKGROUND: Prolonged pulmonary hypertension (PH) is highly predictive for pulmonary morbidity and death in infants with congenital diaphragmatic hernia (CDH). OBJECTIVES: 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. CONCLUSION: 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. TYPE OF STUDY: Case series with no comparison group. LEVEL OF EVIDENCE: Level IV.
BACKGROUND: Prolonged pulmonary hypertension (PH) is highly predictive for pulmonary morbidity and death in infants with congenital diaphragmatic hernia (CDH). OBJECTIVES: 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. CONCLUSION: 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. TYPE OF STUDY: Case series with no comparison group. LEVEL OF EVIDENCE: Level IV.
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