Disseminating and Implementing Research-informed Best Practices

An international panel of more than 100 researchers and clinicians released new scientific evidence and revised recommendations this year on the diagnosis, management, and prevention of concussions. Included in those recommendations is the visio-vestibular exam (VVE) — a clinical diagnostic tool developed and refined over the past decade by researchers and clinicians in Children’s Hospital of Philadelphia Minds Matter Concussion Program.

The newest international recommendations on concussion are based on outcomes from the Sixth International Conference on Concussion in Sport, held in Amsterdam in October 2022, and published in the British Journal of Sports Medicine in June 2023. As part of the consensus statement released at the conference, the international group of experts recommended multiple assessment tools, including the new Child Sport Concussion Office Assessment Tool 6 (Child SCOAT-6). The VVE was incorporated as a key element of the Child SCOAT-6.

"Our ultimate goal is for visio-vestibular testing to be routinely performed on every child who may have a concussion," said Daniel Corwin, MD, MSCE, the Emergency Department lead of the Minds Matter Concussion Program, "regardless of where, when, and by whom their assessment occurs. The fact that the VVE has been recognized internationally as a critical evaluation tool for pediatric concussion is a significant step toward achieving that goal."

Researchers at Children's Hospital of Philadelphia led an international group of experts in developing new guidelines for neonatal resuscitation research. The guidelines were developed by the International Liaison Committee on Resuscitation Neonatal Life Support Task Force and published in the journal Pediatrics. They will standardize data definitions for those engaged in this area of research, allowing for better comparisons across studies and ultimately better outcomes.

Clinical research on neonatal resuscitation has accelerated over recent decades, but there are no standardized definitions or reporting guidelines for neonatal resuscitation clinical studies, making it difficult to compare studies or make clinical recommendations. To address this, the International Liaison Committee on Resuscitation Neonatal Life Support Task Force established a working group to develop reporting guidelines for neonatal resuscitation, based on similar guidelines that were developed for adults in 1990 in Utstein Abbey, Norway.

"We hope the Neonatal Utstein reporting guidelines will assist investigators engaged in neonatal resuscitation research and facilitate data pooling in meta-analyses, enhancing the strength of neonatal resuscitation treatment recommendations and subsequent guidelines," said first author Elizabeth Foglia, MD, MA, MSCE, an attending neonatologist with the Division of Neonatology at CHOP.

An international panel led by a Children's Hospital of Philadelphia expert established new criteria for properly diagnosing myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD). The proposed guidelines are the first clinical guidelines for MOGAD and provide an important framework for distinguishing MOGAD from other neurological disorders.

MOGAD is a neurological disorder that causes inflammation and potential damage of the brain, spinal cord, and optic nerves. It can cause a wide range of symptoms, from vision loss and reduced color vision to weakness in the limbs, confusion, drowsiness, encephalopathy, and seizures. Without international consensus criteria about the disease, many MOGAD patients go undiagnosed.

"We hope that better and earlier diagnosis can lead to more patients receiving appropriate treatments," said Brenda Banwell, MD, co-director of the Neuroscience Center at CHOP and first author of the guidelines. "Consensus diagnostic criteria will allow us to conduct more robust clinical trials to investigate potentially superior therapeutic interventions and ultimately improve long-term outcomes for these patients."

The Patient-Centered Outcomes Research Institute (PCORI) selected Children's Hospital of Philadelphia to participate in a new funding opportunity to promote the uptake of useful research results into practice.

The funding initiative, called the Health Systems Implementation Initiative (HSII), will provide funding to 42 participating healthcare delivery systems. At CHOP, this work will be led by Clinical Futures, a CHOP Research Institute Center of Emphasis, in partnership with the Center for Healthcare Quality and Analytics (CHQA).

"A main focus of Clinical Futures is in translating the best medical evidence into practice. The PCORI Health Systems Implementation Initiative will provide support to realize this goal for the benefit of patients and families," said Alexander Fiks, MD, MSCE, director of Clinical Futures and project lead for CHOP's participation in HSII. "This new initiative builds on prior successes we have achieved with PCORI funding in the area of antimicrobial stewardship, as well as ongoing work in the treatment of kidney stones and rheumatologic conditions in pediatric patients."

Researchers from Children's Hospital of Philadelphia, the Alex's Lemonade Stand Foundation Childhood Cancer Data Lab, the Children's Brain Tumor Network (CBTN), the Pacific Pediatric Neuro-Oncology Consortium (PNOC), and more than 20 additional institutions have partnered to create a first-of-its-kind open-source, reproducible analysis platform for pediatric brain tumors.

With the help of thousands of genomically sequenced samples, researchers used this platform to identify initial findings about genetic variants associated with poorer outcomes that could help guide future diagnostic and therapeutic advances. The details of the platform, called OpenPBTA, were published this year in the journal Cell Genomics.

"While there have been many proponents of an open-source model for scientific research, nothing like this existed for pediatric cancer," said Jo Lynne Rokita, PhD, a supervisory bioinformatics scientist leading OpenPBTA at the Center for Data-Driven Discovery in Biomedicine at CHOP and one of the study's authors. "We designed OpenPBTA so that anyone could access the data, contribute to its analysis, and/or use it in their own research."