Children’s Hospital is a national leader for research networks that accelerate and broaden research.
For McKenzie, 3, research that indicates which treatment is the best to keep her kidney disease from progressing can’t come soon enough. McKenzie has steroid-resistant nephrotic syndrome, so her kidneys don’t properly filter protein, which could lead to loss of function or even kidney failure over time. A new medication is keeping it in check — for now — and she’s home in East Windsor, N.J., playing with dolls and coloring like before her diagnosis.
It’s for children like McKenzie that Children’s Hospital of Philadelphia (CHOP) has taken a national leadership role in research that focuses on identifying the medications, therapies and procedures that work best — especially from the patients’ and families’ perspective — and that finds answers much more quickly than in the past.
Christopher Forrest, MD, PhD, an academic investigator at CHOP, has led the creation of the national research network of pediatric hospitals, known as PEDSnet, that was established to provide information that will help families make the best decisions possible about their children’s care.
From Years to Months
In traditional multi-institution research, it can take several years to get a study off the ground. Studies under the umbrella of PEDSnet, however, have all participating hospitals agree ahead of time to the precise details of data collection, protocols and institutional review board approval, so they can move from idea to enrolling the first patient in just a few months. It’s also a much less expensive way to do research.
Because PEDSnet includes 5.3 million children in 23 states from eight pediatric hospitals, its studies can enroll many more patients than any single institution could on its own — which is critical for studying rare diseases like McKenzie’s.
Last year, Forrest was also appointed chair of the research committee for PCORnet, a national patient-centered clinical research network. In this role, he will help shape research studies that include a participant population of up to 80 million Americans.
“Big data is changing the face of clinical research,” says Forrest. “This approach includes millions of people — the scale is unprecedented — and it fully honors the patient perspective. Research will be faster, cheaper, better.”
PCORnet is the flagship program of the Patient-Centered Outcomes Research Institute (PCORI), an independent nonprofit that was congressionally authorized by the Affordable Care Act. PEDSnet is one of 13 clinical data research networks under PCORnet.
PCORI has been a major, national sponsor for electronic health record-fueled research that also involves patient and family input. PCORnet covers dozens of research networks that study specific populations and diseases. PEDSnet, which also receives funding from the National Institutes of Health and other entities, focuses on childhood diseases. GLEAN will study pediatric kidney disease, using data from PEDSnet and NephCure.
Mining Electronic Health Records
The networks rely on hospitals’ electronic health records (EHRs) to gather data. Each time patients visit the doctor, their diagnosis and any procedures they receive are recorded in their EHR under a special code. Researchers can sort and analyze the data by patient demographics such as age, gender, code and outcome — without names attached. That information is combined with answers patients and families report to get the full picture of which treatment is most effective.
Within PEDSnet is a CHOP-led research network dedicated to pediatric kidney disease, headed by two of McKenzie’s nephrologists, Michelle Denburg, MD, MSCE, and Susan Furth, MD, PhD, chief of the Division of Nephrology, and in collaboration with doctors from the seven other PEDSnet institutions.
“By establishing the Glomerular Disease Learning Network, which we call GLEAN, our goal is to discover what treatments have been the most successful in real-world settings to prevent the progression of kidney disease,” says Denburg. “Because we can capture data on millions of children, the potential is powerful.”
For GLEAN research, only medical information on children with certain diagnoses, as identified by diagnosis codes, would be included — no names or personal information. GLEAN is also partnering with another kidney-disease network, the NephCure Kidney Network, which includes patients of all ages, to further broaden its reach.
One proposed study is to compare fractures and other musculoskeletal outcomes in children with kidney disease against the experience of healthy children. Kidney disease is associated with multiple disturbances in calcium, phosphate and vitamin D metabolism that can affect growth and lifelong bone health. Before, a typical single-center study of children with glomerular disease might enroll 20 patients; GLEAN hopes to represent more than 2,000 patients and, as a result, the findings will have a higher degree of validity and impact.
More CHOP-led Efforts
This type of scale is being replicated in other disease areas. Kathleen Sullivan, MD, PhD, Chief of the Division of Allergy and Immunology, leads a network focused on immunodeficiency.
Gastroenterologist Andrew Grossman, MD, is the site leader for a study on Crohn’s disease. And oncologist Richard Aplenc, MD, PhD, MSCE, heads a study looking at whether it’s better to recover from treatment for acute myeloid leukemia at home or in a hospital.
Because we can capture data on millions of children, the potential is powerful.
Michelle Denburg, MD, MSCE
A key component of PEDSnet, and other networks under PCORnet, is patient and family involvement. Amy Kratchman, a family consultant at Children’s Hospital, was selected to represent caregivers and parents of pediatric patients on PCORI’s Advisory Panel on Patient Engagement. The panel ensures PCORI engages patients and other healthcare stakeholders in research and maintains a patient-centered culture in all of its work.
That means patients — and, for children, their family members — are involved in deciding research priorities. While a physician might be keenly interested in how a particular medication affects a patient’s cholesterol levels or kidney function, for example, families may be more concerned about quality-of-life issues: Does a treatment allow my child to go to school, play with friends, sleep through the night?
“It will be a trusted resource for families, ours included, to learn about what our kids are facing,” says McKenzie’s mother, Kelly Carella. “I appreciate the feeling of not being alone. We’d be happy to contribute to this kind of research.”