Using data to help kids with sickle cell disease

Children's Wisconsin selected as the first site in ASH Research Collaborative Sickle Cell Disease Clinical Trials Network

The announcement that Children's Wisconsin is the first site to be accepted into the American Society of Hematology (ASH) Research Collaborative Sickle Cell Disease Clinical Trials Network came recently, but Julie Panepinto, MD, MSPH, Children's lead researcher of pediatric sickle cell disease (SCD) and its treatment, has been working toward this milestone for years. In 2014 she and Daniel Michalik, MD (a medical student at the time), first began mining electronic health data for information on their SCD population. They developed algorithms — one of which came to be deemed “the Milwaukee Protocol.” This work continues today and is led in partnership with Ashima Singh, PhD, MS. Years earlier, Dr. Panepinto partnered with Children's pediatric emergency physician David Brousseau, MD, MS, to leverage big data from entities like Medicaid and published their findings in top-tier journals such as JAMA.

Now, as part of the ASH Research Collaborative Sickle Cell Disease Clinical Trials Network, Panepinto and her colleagues' experience and research will be contributing real-world evidence in one of the largest hematologic databases. She will be working as part of this collaborative in partnership with Joshua Field, MD, MS, who leads the adult SCD program at the Medical College of Wisconsin.

Historically, sites have small numbers of patients with SCD from which to pull information,” Panepinto says. “For the first time, we'll be able to gather, house and quantify data on SCD patients on a large scale — as in thousands of patients vs hundreds — leaving us with an incredibly rich resource on a rare disease."

Established in 2018, the ASH Research Collaborative aims to improve the lives of people affected by blood diseases by fostering collaborative partnerships to accelerate progress in hematology. The ASH Research Collaborative is a two-pronged initiative.

1. The Data Hub. A technology platform that facilitates the exchange of aggregated information in one place for the purpose of mining research-grade data on hematologic diseases.
2. SCD Clinical Trials Network. This is a patient-centered network that will optimize the conduct of SCD clinical trials, leveraging real-world evidence to improve treatment options for individuals living with SCD. The Network is collaborating with individuals living with SCD, their caregivers, and providers when evaluating which trials to implement.

Children's was selected as the first site for its experienced staff, multidisciplinary clinical program, state-of-the-art trial facilities and industry-leading work in big data — some of which it had already contributed to the ASH Research Collaborative's Data Hub. “We are blessed in the sense that we have two good programs [adult and pediatric] that work collaboratively and serve nearly the whole state population,” Panepinto says.

“Our data work has also allowed us to manage the population of our patients by examining their data on a regular basis,” she says. “Children with SCD, for example, are supposed to have an annual stroke screening. By applying our data to our medical records, we were able to achieve and maintain an annual screening rate at 75% or higher."

The ASH Research Collaborative anticipates announcing additional sites across the country on a rolling basis.