Beyond What’s Possible
Exploring the Future of Health
Investments in pediatric research hold the opportunity to impact an entire lifespan making it a critical element of Nemours’ strategic vision to redefine the future of children’s health. Nemours’ investments manifest in multiple ways including developing new treatments, personalizing therapies, mining data to inform clinical decisions, and identifying better ways to deliver care where a child or family is — physically, emotionally and socially. A continuous pipeline of innovation requires infrastructure, funding and mentoring — all elements of Nemours’ growing reputation in pediatric research.
Igniting a Sustainable Impact to Improve Children’s Health
Back in 2003, a group of energetic scientists and clinician-scientists at Nemours networked to develop an NIH Centers for Biomedical Research Excellence (COBRE) grant. By brainstorming and exchanging ideas, they produced a proposal that the NIH ranked as one of the best submissions they had received in that round of COBRE funding. The Center for Pediatric Research (CPR) COBRE grant was unique in several ways:
- it was the first COBRE grant awarded to a pediatric research focus;
- Nemours’ center and hospital are primary providers of pediatric care and support for the entire state of Delaware;
- it addressed common barriers to translational research by integrating researchers from a number of major disciplines, enabling and maintaining a focus on mentored research training; and
- its initial narrowed focus (genetics, cardiology and neuroscience during Phase I) helped establish a critical mass of investigators to attract funding and recruit new researchers, ultimately creating a sustainable research center.
The team approach toward solving clinical research questions, exemplified through the CPR, helped further develop the research and clinical programs across all of Nemours.
Today, under the direction of principal investigator Thomas H. Shaffer, MSE, PhD, Nemours CPR remains one of the few (10%) COBRE programs that focus on pediatric disorders. During the past 16 years, the grant (receiving four phases of extension through 2020) has supported nine Target Investigators, two Transitional Investigators, and 12 Pilot Investigators, as well as numerous clinical fellows and grad students.
Each Nemours CPR COBRE investigator has shown remarkable academic progress — being awarded federal, state, corporate and foundation grants; numerous publications and presentations; mentoring students and post-doctoral fellows; and establishing individual investigator laboratories, and enhancing or establishing new core laboratory services at Nemours Children’s Hospital, Delaware.
Of the $485 million NIH distributed nationally through IDeA, $257 million was provided to Delaware researchers through 992 new grant awards; Nemours’ CPR share is estimated at $45.5 million
Supporting Families in Managing Disease
Approximately half of the current NIH-funded CHDS research projects focus on various aspects of pediatric diabetes, including behaviors and barriers related to glycemic control. Two of these NIH grants are R01 type, meaning they seek to solve a specific problem and provide actionable outcomes backed by significant data that, when published, can often be considered a best practice or standard of care. Typical pediatric diabetes management includes quarterly in-person visits between physician and patient; that means, physicians are managing the disease less than 1% of the time — the other 99% of disease management falls on families. Nemours treats families, not just kids; as such providing support and resources for the whole family is imperative. These two NIH R01 grants (see below) are intended to identify and connect families with the resources they need using the broader perspective of what influences a child’s health.
Remedy to Diabetes Distress: A scalable screen-to-treat program for school-age families
Diabetes distress can be similar to depression and include feelings of sadness, anger, despair, burnout and fear — all related to managing diabetes in yourself or someone else living with diabetes. In addition to being indicative of quality of life, diabetes-specific distress can be one of the largest contributors to glycemic control and patient engagement. It is becoming increasingly important to identify patients and family members experiencing diabetes distress so they can receive appropriate support to reduce the distress.
The Nemours team will work to develop, implement, and test the feasibility and acceptability of a novel, practical, and scalable screen-to-treat program for diabetes distress in parents and school-aged children with type I diabetes. This project, the Remedy to Diabetes Distress (R2D2), is funded by a five-year, $3.2 million NIH R01 grant secured by Susana Patton, PhD, ABPP, CDCES, Florida Center Director, Nemours CHDS, and entails the use of screening tools that will be developed and tested in Phase 1 of the study to appropriately identify parents and children for recruitment.
Phase 2 includes an mHealth and telehealth treatment program designed to teach parents and children cognitive-behavioral therapy, mindfulness, and behavioral activation strategies to help them manage feelings of distress.
Home-based Video Telemedicine to Reduce Parental Fear of Hypoglycemia in Youngs Kids with T1D
Managing type 1 diabetes (T1D) in young children introduces greater risk for low blood sugar or
a hypoglycemic event. In part, this is due to increased insulin sensitivity in young kids as well as their unpredictable behavior, eating habits and ability to communicate how they’re feeling. Hypoglycemia can lead to dangerous complications and poor health outcomes.
Through a five-year, $2.9 million NIH R01 grant, also secured by Susana R. Patton, Nemours is seeking ways to help families better manage and cope around managing the fear of hypoglycemia through a unique at-home, video-based telehealth intervention. The 10-week sessions, led by PhD-level interventionists, teach families cognitive behavior therapy strategies (e.g., mindfulness, behavioral activation) to reduce fear and promote better coping for fear that’s unavoidable.
Throughout the intervention, families complete online surveys, submit data from pump or continuous glucose monitor devices to the cloud, and complete at-home test kits that measure glycated hemoglobin. In addition, Actiwatches are provided to study participants to measure physical activity and sleep behaviors. Currently, 50 families have participated in the remote study; the total sample size is expected to grow to 180 in coming years.
Elevating Sickle Cell Disease Research
The Delaware Comprehensive Sickle Cell Research Center (DCSCRC) was established in 2014 through a $10.2 million, five-year COBRE grant awarded by the NIH to Nemours Center for Cancer and Blood Disorders (NCCBD), in collaboration with Delaware State University (one of two Historically Black colleges in the state of Delaware). The Center builds upon strengths in biomedical research, clinical care and faculty to create a sustainable infrastructure supportive of target investigators and cutting-edge research in sickle cell disease (SCD).
Through the COBRE grant, the DCSCRC investigators and clinicians work side-by-side studying the genetic mutation that causes SCD and improving care and outcomes for affected children, to:
- prevent SCD symptoms
- offer families strong psychosocial support
- study the quality of SCD care
- identify genetic pharmacologic approaches to treatment and cure
To date, researchers from the DCSCRC created 60 presentations and published 66 chapters, abstracts and peer-reviewed manuscripts related to associated projects and trials. Most recently, researchers from the DCSCRC published a manuscript for JAMIA Open titled, “Implementation of a Learning Health System for Sickle Cell Disease.” The objective of the study was to create a comprehensive learning healthcare system to support disease management and research using SCD as a model. The multidisciplinary DCSCRC team developed an SCD clinical data dictionary to standardize bedside data entry and inform a scalable environment capable of converting complex electronic health records into knowledge accessible in real time. A custom application, the Sickle Cell Knowledgebase, was developed to improve data analysis and visualization. The system can be used to facilitate patient care and guide medical decision-making, in addition to enhancing the ability to access patient data for research purposes. While the focus was on SCD, the strategy for implementing a learning healthcare system can be applied to many other chronic disease models.
Read the abstract, to learn more about the learning health system for sickle cell disease.
Approximately one in 500 African American babies and one in 11,000 Hispanic infants are born with sickle cell, while 2.5 million Americans carry the sickle cell trait.