Medical research is a rewarding field to work in, but young investigators often find that while they have an abundance of bright ideas, scientific funding is not as plentiful. Any opportunity to get a research project off the ground is greatly appreciated, especially by the two Children’s Hospital of Philadelphia physicians who recently received 2014 Pilot Grant Awards from the Center for Pediatric Clinical Effectiveness (CPCE).
The purpose of the CPCE’s Pilot Grant Program is to promote and support fellows, junior faculty, and other CHOP researchers in conducting clinical effectiveness pilot research studies that will attract external support for large-scale studies. The CPCE accepts proposals twice a year, and promising projects undergo at least two rounds of reviews to determine that they fully meet the selection criteria. Candidates must be involved in research designed to produce evidence for what works best for treating, diagnosing, and preventing disease.
The spring 2014 award winners, Andrea Knight, MD, and Lori Kestenbaum, MD, are enthusiastic about launching their investigations.
Dr. Knight is an assistant professor in the Division of Pediatric Rheumatology at CHOP. Her research focus includes patient-centered outcomes and health services research in pediatric systemic lupus erythematosus (SLE), also known as lupus, an autoimmune disease that can affect multiple body systems. Her goal is to facilitate access to mental healthcare and improve health-related quality of life for children with SLE.
The pilot study aims to reveal key patient, caregiver, and provider-level factors that affect mental healthcare for pediatric SLE and mixed connective tissue disease patients (MCTD). The findings from this study will lay the groundwork for developing mental health screening and intervention options for pediatric patients with SLE/MCTD. Practitioners could use this knowledge to create a trusting environment that allows patients with chronic diseases to feel secure in expressing their mental health concerns and to more easily obtain appropriate mental healthcare.
“Ensuring that their mental health is adequately addressed is important because they have a lot of psychological stress dealing with their chronic conditions,” Dr. Knight said. “We want to gain some rich information from patients and their families about their mental health needs and any difficulties that they see to getting them addressed.”
The pilot grant will build on previous findings that Dr. Knight compiled as the principal investigator for a study evaluating the prevalence of depression and anxiety in pediatric SLE patients. She found that depression or anxiety affected about one-third of the patient cohort, but that mental health treatment rates in those with symptoms were low.
Over the next year, Dr. Knight and her study team will conduct patient-centered interviews with 30 participants that will aim to identify possible barriers to the mental health referral process. This experience also will allow Dr. Knight to expand her expertise to include more qualitative research processes.
“Surely, having funding toward a project is hard to capture these days, so it was very exciting to win the grant,” Dr. Knight said. “It also is exciting to know that the CPCE, policy lab, and CHOP as a whole are interested in this issue. I personally think that it is very important to my patients but also to chronic disease patients as a whole.”
The second recipient of the award, Dr. Kestenbaum, is a fellow in Pediatric Infectious Diseases at CHOP. Her research is focused in two areas: understanding vaccine hesitancy to increase vaccine acceptance and modifying antibiotic prescribing practices for common childhood illnesses to reduce antibiotic overuse.
The pilot study will investigate antibiotic prescription practices and efficacy in patients with community-acquired pneumonia (CAP). CAP is the leading cause of mortality for children beyond the neonatal period worldwide, and current guidelines recommend amoxicillin as first line treatment to target the most common causes of CAP. Despite these guidelines, the most commonly prescribed antibiotics are macrolides, a class of antibiotics that does not appropriately treat the most common causes of bacterial pneumonia.
“The [study] design takes advantage of CHOP’s practice-based research network, which is an invaluable resource in the fact that it links together 31 primary care practices, multiple different regions, socioeconomic classes, and is all united under one electronic health record,” Dr. Kestenbaum noted.
The study will entail a thorough analysis of a dataset from the ambulatory EHR system in order to pinpoint patient and provider characteristics that are likely to influence antibiotic prescribing choices in the outpatient setting. Afterward, Dr. Kestenbaum will manually review additional data to compare how treatment regimens with amoxicillin vs. macrolides are associated with patients’ outcomes.
“This study will look at what drives providers to make treatment decisions,” Dr. Kestenbaum said. “Is it something about the patient, is it something about the provider, or is there another factor that drives providers to make treatment decisions that are not in our national guidelines?”
Dr. Kestenbaum expects that results of this comparative effectiveness pilot study will help to support future guideline development and implementation to ensure appropriate antibiotic prescribing practices for the management of CAP.
“The opportunity to have a pilot grant is really exciting,” Dr. Kestenbaum said. “It allows me to collect preliminary data to support future study proposals. The support of the CPCE for those of us who are just starting out is incredibly helpful.”