Researchers Launch New Investigations With Clinical Futures Pilot Grants

If scientific research were like building a house, pilot studies would be the foundation. Their purpose is to establish solid evidence that will attract external support for large-scale studies. Clinical Futures a Center of Emphasis at The Children’s Hospital of Philadelphia Research Institute, is giving three investigators the tools that they need to get to start digging.

Through the Clinical Futures Pilot Grant Program, fellows, junior faculty, and other Children’s Hospital investigators interested in clinical effectiveness research have the opportunity twice a year to receive funding to support their pilot studies designed to produce evidence of what works best for treating, diagnosing, and preventing disease. The three recipients of the 2015 Spring Pilot Grant Award are Sandra Amaral, MD, MHS, Sabrina Gmuca, MD, and Laura Figueroa -Phillips, MD.

Find out more about the awardees and highlights of their projects in this Q&A:

Sandra Amaral, MD, MHS

Tell us a little bit about your background:

I am an assistant professor and attending physician in CHOP’s Division of Nephrology and the Kidney Transplant and Dialysis Program. I received my MD from the MCP-Hahnemann University School of Medicine and a Master of Health Science degree in clinical epidemiology from Johns Hopkins University, Rollins School of Public Health.

What is unique about your pilot research study?

My pilot study, “Using a Mobile Game Application to Improve Healthcare Self-management in Adolescents With Solid Organ Transplants” aims to develop a novel educational mobile health (mHealth) game for adolescents with solid organ transplants (SOT) and test its feasibility and acceptability among adolescent SOT recipients and their caregivers. Adolescent SOT recipients have poor long-term graft survival rates, and are at particularly high risk for unnecessary graft loss due to non-adherence in the management of their complex chronic health conditions. This study will employ novel mHealth gaming technology to augment current care practices and to change how adolescents think about healthcare management, with the ultimate goal of reducing graft loss due to non-adherence.

How do you anticipate your study results will lead to future research?

The findings from this study will provide pilot data in support of future research to test the generalizability and efficacy of the mHealth game in a broader adolescent patient population. If successful, this study will transform positive health behavior and medication adherence research, making patient-centered interventions available in an enjoyable and user-friendly platform. This line of research will be broadly relevant to improving outcomes for all pediatric patients managing complex treatment regimens for chronic health conditions.

What was your reaction to receiving the Clinical Futures award?

I was tremendously grateful and excited. The seeds for this project were planted for me about two years ago when I took a class at Penn on Healthcare Innovations. I have been working steadily on refining the project since then but progress has been slow — mostly due to lack of funding. The Clinical Futures pilot funding will provide me with the opportunity to work closely with a game developer and programmer so I can move this idea into a real, testable product for patients and modified by patients and their caregivers.

Sabrina Gmuca, MD

Tell us a little bit about your background:

I am a first-year Rheumatology fellow in CHOP’s Department of Pediatric Rheumatology and the Center for Childhood Arthritis and Rheumatic Diseases. I received my MD from State University of New York Downstate Medical Center College of Medicine and completed my residency in pediatrics at NYU Langone Medical Center. My research interests include the evaluation of treatment and outcomes in autoimmune conditions affecting the central nervous system.

What is unique about your pilot research study?

Pediatric Neuromyelitis Optica (NMO) is a chronic inflammatory disease of the central nervous system that preferentially targets the optic nerves and spinal cord. By analyzing NMO patient outcomes data over a 13-year period, my retrospective cohort pilot study, “Treatment and Outcomes in Neuromyelitis Optica,” will attend to the current lack of standardized care in the therapeutic management of pediatric NMO.

How do you anticipate your study results will lead to future research?

The results of this study will provide an increased understanding of disease outcomes in pediatric and adult NMO, and its findings will be clinically useful for both rheumatologists and neurologists. Further, if successful, the study’s findings will facilitate future comparative effectiveness trials of treatment with biologics, and it will ultimately make possible the establishment of the first standardized therapeutic protocol for pediatric NMO.

What was your reaction to receiving the Clinical Futures award?

Upon receiving word that I had been awarded funding through the Clinical Futures pilot grant, I felt immediately honored and overjoyed. Clinical Futures allows fellow physicians such as myself to jumpstart our research interests and put ideas into action by providing us both valuable mentorship and important financial support. I am excited and eager to start my research and help improve patient outcomes in autoimmune diseases.

Laura Figueroa-Phillips, MD

Tell us a little bit about your background:

I am a first-year Pediatric Hospital Epidemiology and Outcomes Training fellow at CHOP. I received my MD from the University of Maryland School of Medicine and completed my residency in pediatrics at CHOP. My research and clinical interests revolve around improving patient-centered outcomes for children and their families in an inpatient setting, as well as the epidemiology, diagnosis, and treatment of healthcare related infections.

What is unique about your pilot research study?

Long-term, outpatient central venous access is necessary for the treatment of many pediatric conditions, but can result in serious complications, including central line-associated bloodstream infections (CLABSIs). My nested case-control pilot study, “Development of a Clinical Prediction Model for Pediatric Outpatients at Risk for Central Line-Associated Bloodstream Infections” aims to identify risk factors and derive a preliminary clinical prediction model for CLABSI among ambulatory pediatric patients with central venous lines presenting with bacteremia-associated symptoms. This study will be the first to determine the overall rate of CLABSI among the ambulatory pediatric population with central venous lines, to evaluate the entire population of patients at risk for CLABSI, and to derive a clinical prediction model for CLABSI.

How do you anticipate your study results will lead to future research?

The results of this study will serve as a first step in the development of a clinical prediction tool to guide clinical decision-making when patients present to the Emergency Room, and it could ultimately decrease unnecessary hospital admissions, lower the number of adverse effects associated with exposure to broad spectrum antibiotics, improve patient and family quality of life, and decrease healthcare spending.

What was your reaction to receiving the Clinical Futures award?

I was thrilled and honored to be chosen as a Clinical Futures Pilot Grant awardee. The grant will give me a great launching pad to successfully complete my project and will help me set the groundwork to receive funding for related projects in the future.