Funds awarded by the Patient-Centered Outcomes Research Institute
Anne Berg, PhD, Research professor, and Sandi Lam, MD, MBA, Chief of Neurosurgery at Ann & Robert H. Lurie Children’s Hospital of Chicago have been approved for a $4 million funding award by the Patient-Centered Outcomes Research Institute (PCORI) to conduct a study, Comparative effectiveness of palliative surgery versus additional anti-seizure medications for Lennox-Gastaut Syndrome.
The severe and often dangerous seizures of Lennox-Gastaut syndrome, a serious, ultra-rare form of epilepsy that begins in infancy and early childhood, result in frequent trips to the emergency room and frequent emergency hospitalizations. These seizures also disrupt home life for the patient and for all family members. In part because of the relentless seizures that begin at a very young age, Lennox-Gastaut syndrome is typically accompanied by severe disabilities in gross motor, fine motor, communication, eating, and other important skills needed for daily functioning.
Lennox-Gastaut syndrome has no cure, and although current treatments may help reduce the number of seizures, none are expected to eliminate them entirely; these treatments are merely palliative. The main treatments include anti-seizure medications and some surgical approaches. Currently, there is no strong evidence to support parents and physicians in deciding which type of treatment (more medicines or surgery) will be most successful for a child with Lennox-Gastaut syndrome, and whether one or the other approach may lessen the toll that seizures take on a child’s development and ability to function. The results from this study will help parents and providers make more informed choices about treatment for children with Lennox-Gastaut syndrome and will highlight areas for improvement in providing the best possible health care for this severe, lifelong disorder.
Anne Berg, PhD, Sandi Lam, MD, MBA, and their collaborators teamed up with Tracy Dixon-Salazar, PhD: mother, advocate, and executive director of the Lennox-Gastaut Foundation. "We are excited to work with an all-star team to make this important study a reality." Berg and Lam give credit to their teammates, "The depth of expertise is incredible. Marc Rosenman, MD, in medical informatics and health services research; Joyce Wu, MD, and Anup Patel, MD, in epilepsy; Matt Hall from the Children's Hospital Association as our senior biostatistician; Tracy Dixon-Salazar with essential patient advocacy perspectives - the list goes on. We respect this unique opportunity and responsibility to study treatments for this rare disease and then expand applications of such multicenter research for other patients and diseases in the future." Another unique aspect of the study is that it will draw on the vast health data resources of PCORnet®, the National Patient-Centered Clinical Research Institute, to conduct this study more efficiently. With health records for 66 million patients available for observational studies, the PCORI-funded PCORnet provides vast scale to power research on conditions affecting even small numbers of people.
“This study was selected for PCORI funding not only for its scientific merit and commitment to engaging patients and other stakeholders, but also for its potential to fill an important gap in our health knowledge,” said PCORI Executive Director Nakela L. Cook, MD, MPH. “These results aim to help patients and families of those with a rare condition get information that will help them make a better-informed choice about their care. We look forward to following the study’s progress and working with Lurie Children’s to share the results.”
Berg and Lam’s study was selected for PCORI funding through a highly competitive review process in which patients, clinicians and other stakeholders joined clinical scientists to evaluate the proposals. Applications were assessed for scientific merit, how well they will engage patients and other stakeholders and their methodological rigor among other criteria.
This funding award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.
PCORI is an independent, nonprofit organization initially authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers and clinicians, and other healthcare decision makers with the evidence-based information needed to make better-informed healthcare choices. For more information about PCORI’s funding, visit www.pcori.org.