Yoshinori Katayama, Katsuya Hirata, Yutaka Nishimura, Atsuko, Nakahari, Mami Takeoka, Masahiko Watanabe, Tetsuya Isayama, Japan Evidence Based Neonatology
Takatsuki General Hospital. Osaka Women’s and Children’s Hospital. Hiroshima City Hiroshima Citizens Hospital. Jichi Medical University Saitama Medical Center. Mie University Graduate School of Medicine. National Center for Child Health and Development.
Japan
Pediatrics International
Pediatr Int 2026; 68:
DOI: 10.1111/ped.70385
Abstract
Background: Although sildenafil is commonly used for bronchopulmonary dysplasia-associated pulmonary hypertension (BPD-PH), high-quality evidence to justify its use remains sparse. Thus, we evaluated the efficacy and safety of sildenafil in preterm infants with BPD or BPD-PH.
Methods: A systematic review of randomized and non-randomized comparative studies identified by searching MEDLINE, Embase, Cochrane Central Register of Controlled trials, Cumulative Index to Nursing and Allied Health Literature, and Ichushi-Web until November 28, 2024. The primary outcome measure was mortality at the time of neonatal intensive care unit (NICU) discharge.
Results: Among the 1944 studies identified, 54 were eligible for full-text screening; however, none met the systematic review criteria. A post hoc narrative synthesis summarized the outcomes of preterm BPD-PH infants receiving sildenafil in nine studies, which were ineligible for the systematic review due to the lack of control groups. These studies included 15-666 infants born in high-income countries from 2001 to 2022. Sildenafil treatment improved the PH symptoms in 47%-77% of patients according to the echocardiographic findings. In patients receiving sildenafil, mortality before NICU discharge was 13%-35% and the rate of severe retinopathy of prematurity was 16%-57%.
Conclusions: Evidence to support the use of sildenafil treatment for preterm infants with BPD-PH to improve mortality and morbidity is lacking. More research, particularly randomized controlled trials, on the efficacy and safety of sildenafil treatment for preterm BPD-PH infants is needed.
Category
Class III. Pulmonary Hypertension Associated with Lung Disease
Medical Therapy. Efficacy or Lack of Efficacy
Medical Therapy. Adverse Effects or Lack of Adverse Effects
Age Focus: Pediatric Pulmonary Vascular Disease
Fresh or Filed Publication: Fresh (PHresh). Less than 1-2 years since publication
Article Access
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