Congenital diaphragmatic hernia (CDH) is a complex malformation characterised by a triad of pulmonary hypoplasia, pulmonary hypertension (PH) and cardiac ventricular dysfunction. Much of the mortality and morbidity in CDH is largely accounted for by PH, especially when persistent beyond the neonatal period and refractory to available treatment. Gentle ventilation, haemodynamic optimisation and pulmonary vasodilation constitute the foundations of neonatal treatment of CDH-related PH (CDH-PH). Moreover, early prenatal diagnosis, the ability to assess severity and the developmental nature of the condition generate the perfect rationale for fetal therapy. Shortcomings of currently available clinical therapies in combination with increased understanding of CDH pathophysiology have spurred experimental drug trials, exploring new therapeutic mechanisms to tackle CDH-PH. We herein discuss clinically available neonatal and fetal therapies specifically targeting CDH-PH and review the most promising experimental treatments and future research avenues.
- intensive care units
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Contributors FRDB designed the work. FRDB and LJ drafted the work. CMA, HLH, JD, NER, FMR and DB critically reviewed the work for intellectual content. All authors approved the final version to be published.
Funding FRDB received personal grant support from the Belgian-American Fulbright Commission, Belgian American Education Foundation, Sofina-Boël and Research Foundation Flanders (1S31720N). DB is funded by the Erasmus & Programme of the European Union (Framework Agreement number: 2013-0040). CA is funded by NIH/NHLBI 1K23 HL150337-01A1.
Competing interests None declared.
Provenance and peer review Not commissioned; externally peer reviewed.
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