Pediatric medicine poses a unique dilemma. Historically, few studies have been conducted on children to determine the proper dose for many commonly prescribed medications. Finding the right drug and the correct amount often seems like trial and error. Steven Leeder, PharmD, PhD, Deputy Director of the Children’s Mercy Research Institute (CMRI), and a team of dedicated researchers are committed to solving this riddle with opportunistic research and big data to provide optimized and improved medicine for kids.
There are opportunities to develop dosage recommendations for children properly. In general, kids are healthy relative to adults, and when children are sick, taking additional blood samples for research may not be in the child’s best interest. This creates a bit of a dilemma – how can data be generated to improve the dosing of medications in children without that process being burdensome to the patients who can benefit? “Solving this dilemma requires creative approaches,” Leeder said.
The improvement Leeder sees is in being opportunistic with research and data collection. The concept is simple; when a pediatric patient is treated at Children’s Mercy, the type and dosage of medication they receive are logged. Blood samples are often drawn for lab tests as part of routine clinical care, and the leftover samples are discarded. Saving the discarded samples and measuring the concentrations of drugs present in the samples allows the research data to be collected without additional patient blood sticks. This approach is called “opportunistic sampling.” It has been applied in several clinical settings, especially the Newborn Intensive Care Nursery, where the number of blood draws needs to be carefully monitored. Opportunistic sampling data can build mathematical models of how children handle medications. The models can then determine the dose of drugs an individual child may require. “We need to be creative in seeking opportunities to collect data; often, the solutions are right in front of us if only we can recognize them,” Leeder said.
Designing studies to investigate variability in response to medications can lead to impactful findings. “I like looking at a dataset and getting it to confess its secrets. The result is an aha moment when you see a pattern you did not expect,” Leeder said. Such an instance happened at CMRI when a researcher in Neonatology led an opportunistic sampling protocol to analyze the pattern of medicine elimination in infants being given a specific drug. The outcome was a deeper understanding of three different patterns in how newborns broke down a medication they were prescribed. A mathematical model has been developed, and it is hoped that in the future, the model will prove helpful in guiding individualized dosing of that medication—resulting in better treatment and outcomes with less medicine for babies.
Children’s Mercy and the Kansas City region have allowed Leeder to succeed with his approach to improving pediatric clinical pharmacology research. “Projects often require other specialists, a statistician, a psychologist, an understanding of patient flow, and more. Children’s Mercy has allowed a culture of curiosity to flourish, and opportunities to partner with great minds from the expanded community at the University of Missouri – Kansas City and the University of Kansas Medical Center have been tremendously rewarding,” Leeder said.
There are many opportunities to improve the treatment of children, which means more positive outcomes. Introducing effective treatment early in the development of childhood diseases may even change the nature of adult diseases. For Leeder, the remedy is to help clinician scientists use creative solutions when prescribing medications to further impact the region’s children.