Spotlight on Current BPD-Focused Research Studies

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It’s an exciting time for research in the bronchopulmonary dysplasia (BPD) population here at Children’s Hospital of Philadelphia (CHOP). The Newborn and Infant Chronic Lung Disease (CLD) Program within CHOP’s Newborn/ Infant Intensive Care Unit (N/IICU) is one of the world’s largest programs dedicated to the care of infants with severe BPD (sBPD). The CLD Program is dedicated to improving the care of these infants through a flourishing research program. Here, we highlight a handful of our current studies, most of which are actively enrolling infants from our CLD Program.  

NAVA Crossover sBPD Study: Pilot Crossover of Neurally Adjusted Ventilatory Assist (NAVA) and Conventional Flow Triggered Mechanical Ventilation (CMV) in Severe Bronchopulmonary Dysplasia (sBPD)

Due to a lack of current evidence to guide ventilator management to promote lung disease recovery, increase patient-ventilator synchrony, and support neurodevelopment, Erik Jensen, MD, MSCE, and colleagues are investigating whether invasive mechanical ventilation with NAVA as compared to CMV improves respiratory gas exchange in prematurely born infants and young children with sBPD in this prospective, randomized, unblinded, crossover pilot study.

Furosemide in Severe Bronchopulmonary Dysplasia: Comparative Eff ectiveness of a Duration and Dosage Informed Treatment Strategy (FAB-Tol)

This is the second active Furosemide and BPD (FAB) study. The primary outcome is to compare changes in urine volume over time following initiation of sustained furosemide exposure in infants with sBPD. This prospective, observational cohort study with longitudinal urine sampling also compares changes in furosemide urinary excretion rates over time.

EIT During PEEP Grid Study: Eff ects of Positive End Expiratory Pressure on Ventilation and Respiratory Mechanics in Ventilator Dependent Infants/Children with Bronchopulmonary Dysplasia

Lead by Principal Investigator (PI) Howard Panitch, MD, this prospective, nonrandomized, unblinded, interventional trial utilizes electrical impedance tomography (EIT) during a positive-end-expiratory-pressure (PEEP) procedure to compare respiratory mechanic measurements from the ventilator in order to measure the degree of ventilation homogeneity by EIT. The trial will also determine time to ventilation equilibrium by EIT at each PEEP level tested in order to validate and assess which PEEP levels give the optimal respiratory mechanics and gas exchange in an individual infant with sBPD.

Locomotor Learning (LL) in Infants at High Risk for Cerebral Palsy (CP)

In collaboration with the Children’s Hospital of Los Angeles and University of Oklahoma, the Locomotor Learning study, led by PI Laura Prosser, PhD, is enrolling infants at high risk for CP, a well-known comorbidity in infants with BPD, in this longitudinal observational/intervention cohort study. Given the fact that little is known about how infants with brain injury learn and CP risk (or fail to learn) to move and acquire locomotor skills, the objective of this two-part project is to characterize the evolution of locomotor learning over the fi rst 18 months of life in this high-risk population using robust unbiased robotic and sensor technology to longitudinally study infant movement across three consecutive stages during the development of impaired human motor control: early spontaneous movement, prone locomotion (crawling), and upright locomotion (walking).

PANDA Gym: Automated Assessment of Neurodevelopment in Infants at Risk for Motor Disability

Early childhood detection of motor delays or impairments, well-established sequalae of BPD, provides the opportunity for early treatment which improves health outcomes. This multi-part study will use state-of-the-art sensors combined with machine learning algorithms to develop objective, accurate, easy-to-use tools for the early scoring of deficits and lays the foundation for the early prediction of physical disability.

Furosemide Pharmacokinetics in Severe Bronchopulmonary Dysplasia (FAB-PK)

Furosemide is the most commonly used medication in infants with sBPD, but dosage regimens are informed by sparse pharmacokinetic (PK) data from younger preterm infants. In the first of what will likely be many studies examining the use of furosemide in the sBPD population, PI Nicolas Bamat, MD, MSCE, and colleagues are looking to develop furosemide population PK models with covariate analysis for infants with sBPD in this prospective, observational population PK study.

Gastroesophageal ReflUx and the Association with Lung Disease in Preterms (GULP Study)

This study, which has completed enrollment, examines the frequency and severity of gastroesophageal reflux in very preterm infants (< 32 weeks GA). It uses 24-hour esophageal pH/impedance testing with video recording, pulse oximetry, bedside nursing report of clinical signs of reflux, echocardiogram, and one-time sample collection for microbiome analysis in infants receiving primarily enteral or IV nutrition.

Multidimensional Phenotype Classification in Grade 3 Bronchopulmonary Dysplasia

This novel prospective cohort study, led by PI Erik Jensen, MD, MSCE, seeks to develop the first objective phenotype classification system for grade 3 BPD using evidence-based data and define the strength and association between grade 3 BPD phenotypes and neurodevelopmental and pulmonary outcomes through 2 years corrected age. This study will be conducted at CHOP, enrolling 120 infants from the N/IICU, and following them outpatient through the CHOP Neonatal Follow-up Program housed in the CHOP Buerger Center for Advanced Pediatric Care.

Myeloid Derived Suppressor Cell Expression in Newborn Infants: An Observational Cohort Study

Myeloid Derived Suppressor Cells (MDSCs) are white blood cell precursors that are thought to play a role in the development of BPD and other common neonatal diseases and infections. This study aims to quantify the expression of these cells in preterm infants over time and compare this expression to that of term-born infants. Ultimately, the data will allow for improved understanding of these cells and better care for preterm infants.

Phase 1 Trial of Inhaled Tobramycin in BPD

The first phase of this multi-phase trial will identify a safe, well-tolerated dosage of inhaled tobramycin for use in very preterm infants with BPD receiving invasive mechanical ventilation who have evidence of a Gram-negative rod pathogenic bacteria detected by tracheal aspirate culture

ORIM Study: Optical Redox Imaging of Lung Macrophages in Extremely Preterm Infants With or At Risk for Bronchopulmonary Dysplasia (BPD)

This prospective observational study will characterize lung macrophages (LM) in very preterm infants (GA < 30 wks) using optical redox imaging microscopy (ORIM) at three stages of BPD development. Using tracheal aspirates in four cohorts of infants that allows for analysis of lung macrophages, interleukins, RNA sequencing, and microbiome analysis, this study will also compare the LM state and function defined using ORIM in preterm infants with or at risk for BPD to term controls without lung disease and looks to determine the association between lung macrophage ORIM and physiologic and transcriptional measures of lung state, function and inflammation.


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