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, including the implantation of a vagus nerve stimulator (a pacemaker-like generator implanted in the chest wall and programmed by a physician to stimulate the vagus nerve in the neck) and corpus callosotomy (cutting through the band of fibers that connect the two sides of the brain). While both types of treatment (medications and surgeries) produce some benefit by reducing how often the seizures occur, both also have some risks. All medications can, in some patients, produce moderate to severe side effects. This is particularly true of anti-seizure medications, especially when taken in combination with other medications—most patients with Lennox-Gastaut syndrome take several anti-seizure medications at a time. Similarly, surgeries can, in some patients, also have risks during the procedure, and risks of complications following the surgery. In addition, because of anxiety before the surgery, the need to miss work, the procedure itself, and the recovery period, surgery is stressful for parents and family members. 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.

This study has two components. First, it will engage a network of seven pediatric hospitals in the United States where children with Lennox-Gastaut syndrome are cared for and will determine whether seizure-related emergency department visits and hospitalizations are more likely to be reduced following the use of additional medications or adding palliative surgery to existing medications. We will use emergency healthcare utilization as the main outcome, as it is a reflection of the most severe seizures these children have. We will also determine whether medical versus surgical treatment is more likely to lessen some of the developmental and functional difficulties that affect patients with Lennox-Gastaut syndrome. The study will also determine whether starting therapies at a younger versus older age makes a difference.

Because there is so little evidence about which treatment approach works best, there is also much variability in the use of these treatment approaches. The second component of the study will provide a description of the use of surgical versus medical treatment approaches across 18 pediatric hospitals in the United States (the original seven plus an additional 11). We will describe whether use of these treatments differs across hospitals and over time. The study will also determine whether the use of surgery versus medication is different for children with varied characteristics such as age, medical fragility, race, ethnicity, and insurance type.

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.