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Pharmacotherapy for Pulmonary Hypertension in Infants with Bronchopulmonary Dysplasia: Past, Present, and Future
Candice D Fike1, Judy L Aschner2,3
1Department of Pediatrics, University of Utah Health, Salt Lake City, UT 84108, USA.
Insights
Pulmonary hypertension (PH) affects premature infants with bronchopulmonary dysplasia (BPD), leading to high mortality. New PH-targeted therapies are needed, but require pharmacokinetic, safety, and efficacy data before clinical trials.
Area of Science:
- Neonatal Medicine
- Pediatric Cardiology
- Respiratory Medicine
Background:
- Bronchopulmonary dysplasia (BPD) is a common complication in premature infants.
- Pulmonary hypertension (PH) affects 8-42% of infants with BPD, carrying a mortality rate up to 47%.
- Current PH treatments for BPD-PH are off-label and based on expert opinion, lacking robust clinical evidence.
Purpose of the Study:
- To review current and needed treatment strategies for PH in premature infants with BPD.
- To identify knowledge deficits and challenges in developing effective PH-targeted pharmacotherapies.
- To outline approaches for future research, including pharmacokinetic, pharmacodynamic, and safety studies.
Main Methods:
- This is a review article.
- It synthesizes existing literature on BPD-PH treatment.
- It discusses challenges and proposes future research directions.
Main Results:
- There is a critical need for PH-targeted pharmacotherapies in premature infants with BPD-PH.
- Current treatment recommendations lack evidence from randomized controlled trials (RCTs).
- Pre-efficacy studies are essential to gather pharmacokinetic, pharmacodynamic, and safety data.
Conclusions:
- Effective PH-targeted pharmacotherapies are urgently required to improve outcomes for infants with BPD-PH.
- Future research must prioritize RCTs to establish treatment efficacy.
- Developing safe and effective treatments necessitates addressing knowledge gaps and overcoming research challenges.
Abstract:
Approximately 8-42% of premature infants with chronic lung disease of prematurity, bronchopulmonary dysplasia (BPD), develop pulmonary hypertension (PH). Infants with BPD-PH carry alarmingly high mortality rates of up to 47%. Effective PH-targeted pharmacotherapies are desperately needed for these infants. Although many PH-targeted pharmacotherapies are commonly used to treat BPD-PH, all current use is off-label. Moreover, all current recommendations for the use of any PH-targeted therapy in infants with BPD-PH are based on expert opinion and consensus statements. Randomized Control Trials (RCTs) are needed to determine the efficacy of PH-targeted treatments in premature infants with or at risk of BPD-PH. Prior to performing efficacy RCTs, studies need to be conducted to obtain pharmacokinetic, pharmacodynamic, and safety data for any pharmacotherapy used in this understudied and fragile patient population. This review will discuss current and needed treatment strategies, identify knowledge deficits, and delineate both challenges to be overcome and approaches to be taken to develop effective PH-targeted pharmacotherapies that will improve outcomes for premature infants with or at risk of developing BPD-PH.
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