Challenges, priorities and novel therapies for hypoxemic respiratory failure and pulmonary hypertension in the neonate

J. L. Aschner, J. Gien, N. Ambalavanan, J. P. Kinsella, G. G. Konduri, Satyanarayana Lakshminrusimha, O. D. Saugstad, Robin H Steinhorn

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

Future priorities for the management of hypoxemic respiratory failure (HRF) and pulmonary hypertension include primary prevention of neonatal lung diseases, 'precision medicine' and translating promising clinical and preclinical research into novel therapies. Promising areas of investigation include noninvasive ventilation strategies, emerging pulmonary vasodilators (for example, cinaciguat, intravenous bosentan, rho-kinase inhibitors, peroxisome proliferator-activated receptor-γ agonists) and hemodynamic support (arginine vasopressin). Research challenges include the optimal timing for primary prevention interventions and development of validated biomarkers that predict later disease or serve as surrogates for long-term respiratory outcomes. Differentiating respiratory disease endotypes using biomarkers and experimental therapies tailored to the underlying pathobiology are central to the concept of 'precision medicine' (that is, prevention and treatment strategies that take individual variability into account). The ideal biomarker should be expressed early in the neonatal course to offer an opportunity for effective and targeted interventions to modify outcomes. The feasibility of this approach will depend on the identification and validation of accurate, rapid and affordable point-of-care biomarker tests. Trials targeting patient-specific pathobiology may involve less risk than traditional randomized controlled trials that enroll all at-risk neonates. Such approaches would reduce trial costs, potentially with fewer negative trials and improved health outcomes. Initiatives such as the Prematurity and Respiratory Outcomes Program, supported by the National Heart, Lung, and Blood Institute, provide a framework to develop refined outcome measures and early biomarkers that will enhance our understanding of novel, mechanistic therapeutic targets that can be tested in clinical trials in neonates with HRF.

Original languageEnglish (US)
Pages (from-to)S32-S36
JournalJournal of Perinatology
Volume36
Issue numbers2
DOIs
StatePublished - Jun 1 2016
Externally publishedYes

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Pulmonary Hypertension
Respiratory Insufficiency
Biomarkers
Newborn Infant
Precision Medicine
Primary Prevention
Infant, Newborn, Diseases
Point-of-Care Systems
National Heart, Lung, and Blood Institute (U.S.)
Therapeutics
Noninvasive Ventilation
rho-Associated Kinases
Investigational Therapies
Peroxisome Proliferator-Activated Receptors
Arginine Vasopressin
Vasodilator Agents
Research
Lung Diseases
Randomized Controlled Trials
Hemodynamics

ASJC Scopus subject areas

  • Obstetrics and Gynecology
  • Pediatrics, Perinatology, and Child Health

Cite this

Challenges, priorities and novel therapies for hypoxemic respiratory failure and pulmonary hypertension in the neonate. / Aschner, J. L.; Gien, J.; Ambalavanan, N.; Kinsella, J. P.; Konduri, G. G.; Lakshminrusimha, Satyanarayana; Saugstad, O. D.; Steinhorn, Robin H.

In: Journal of Perinatology, Vol. 36, No. s2, 01.06.2016, p. S32-S36.

Research output: Contribution to journalArticle

Aschner, J. L. ; Gien, J. ; Ambalavanan, N. ; Kinsella, J. P. ; Konduri, G. G. ; Lakshminrusimha, Satyanarayana ; Saugstad, O. D. ; Steinhorn, Robin H. / Challenges, priorities and novel therapies for hypoxemic respiratory failure and pulmonary hypertension in the neonate. In: Journal of Perinatology. 2016 ; Vol. 36, No. s2. pp. S32-S36.
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