Conducting a pilot research study is like test-driving a new car. Pilot studies build preliminary evidence and allow for investigators to ensure everything runs smoothly before committing to an extensive, large-scale study. Three investigators at the Children’s Hospital of Philadelphia are getting the funding they need to get their research ideas on the road, thanks to the CHOP Research Institute’s Center for Pediatric Clinical Effectiveness (CPCE).
The CPCE Pilot Grant Program offers funding opportunities twice a year to CHOP investigators conducting clinical effectiveness studies. The recipient of the Fall 2015 Pilot Grant Award was Nicolas Bamat, MD, and the two recipients of the Spring 2016 award were Sagori Mukhopadhyay, MD, MMSc, and David I. Chu, MD. Read on to learn more about the awardees and their studies.
Nicolas Bamat, MD
Dr. Bamat is a neonatology fellow in the division of Neonatology at CHOP. He received his medical degree at the Perelman School of Medicine at the University of Pennsylvania, where his experiences in the neonatal intensive care unit inspired his research interest in neonatal respiratory failure. Dr. Bamat has conducted studies on positive end expiratory pressure (PEEP), which helps keep premature babies’ lungs open when they’re using a mechanical ventilator to help them breathe.
In his study, Selecting PEEP in ventilated premature infants by reducing ventilation/perfusion mismatch: a feasibility study, Dr. Bamat uses a non-invasive technique to detect measurable differences in ventilation/perfusion mismatch (V/Q), a key contributor to babies’ difficulty with gas exchange, in response to changes in PEEP levels. While the non-invasive measure of V/Q mismatch has been previously used in research, it is especially unique in this study because it has mostly been used in adult populations. If Dr. Bamat and his team are able to establish that changes in V/Q mismatch can be detected in response to changes in PEEP and can apply the technique without major issues, they hope it can be used to decrease lung injury in premature infants.
“It was the first grant that I had been awarded and it really allowed me to move forward with this research that I am very excited about and that I think one day could play an important role in helping babies,” Dr. Bamat said.
Sagori Mukhopadhyay, MD, MMSc
Dr. Mukhopadhyay is an assistant professor of Pediatrics in the division of Neonatology at CHOP. She completed her residency and fellowship in neonatology at Children’s Hospital Boston and received her medical degree from Sawai Man Singh Medical College in India. Her research interests focus on neonatal antibiotic exposure and its long-term impact.
In Dr. Mukhopadhyay’s pilot study, Linking neonatal and pediatric records to form a five-year longitudinal birth cohort, her goal is to link the medical records of babies born at Pennsylvania Hospital or Hospital of the University of Pennsylvania to their pediatric medical records at CHOP. This linkage will provide Dr. Mukhopadhyay with the information she needs for her original research question that asks if there is an association between allergic outcomes in babies who receive antibiotics in the newborn period versus those who don’t.
Record linking has been around for over 60 years, but it has not been popular in the U.S. because of certain regulatory components. This study will link together the mother’s delivery record, the baby’s delivery record, and the pediatric delivery record across different medical systems. Dr. Mukhopadhyay is confident moving forward with the study, and she said that parties from both hospitals are excited and eager to get going. If they are able to link these medical records, there will be a wealth of data available that will open up the field to many more retrospective studies that would not be possible otherwise.
“I was super thrilled,” Dr. Mukhopadhyay said about receiving the CPCE grant. “It feels great to have recognition from people who are more experienced than you and think that your ideas have importance.”
David I. Chu, MD
Dr. Chu is currently completing his fellowship in the division of Urology at CHOP, as well as his Master of Science in Clinical Epidemiology at the University of Pennsylvania. He has clinical and research interests in Wilms tumor, the most common primary kidney cancer in children.
The goal of Dr. Chu’s pilot study, Prospective detection of early chronic kidney disease among Wilms tumor survivors, is to screen for early stage chronic kidney disease among Wilms tumor survivors using two biomarkers, urine microalbumin and masked hypertension as detected with ambulatory blood pressure monitoring.
These biomarkers have been shown to correlate with early stage renal dysfunction in other patient subgroups but have never been formally tested in a prospective fashion in Wilms tumor patients. If the use of these screening tools proves to be efficacious, they could potentially be used to test for early stage chronic kidney disease in other disease processes, such as spina bifida.
A majority of Wilms tumor patients have an entire kidney removed at age 2 or 3, but no one knows the long-term renal risks that this surgery poses. Dr. Chu would like to stratify the study patient population by the type of kidney removal surgery and treatments to explore those questions prospectively. Most of the literature that exists on Wilms tumor and the risk of subsequent chronic kidney disease is retrospective.
Dr. Chu was pleasantly surprised by the CPCE grant award: “We are very fortunate for this opportunity to carry through with this project,” he said. “The grant funds will allow us to proceed forward with our idea, so my team and I are very happy.”
CPCE encourages junior investigators from all CHOP departments and divisions to apply for the next Pilot Grant Award. Applications are due in early April for the Spring cycle and early October for the Fall cycle. For more information on the next award and how to apply, CHOP staff may visit the Research Intranet.