A recent safety surveillance paper published in Neurology reported that of eleven pediatric patients who were treated with tenecteplase for suspected stroke, there were no instances of intracranial bleeding or other safety concerns. This study is an important first step toward further research to establish the safety and efficacy of tenecteplase for treating pediatric stroke.
The FDA approved the use of tenecteplase for acute ischemic stroke in adults February 2025, after years of unofficial use. The FDA has not yet approved tenecteplase for use in pediatric stroke.
Kristin Guilliams, MD, associate professor of Neurology, of Pediatrics and of Radiology at Washington University School of Medicine in St. Louis, was a co-senior author of the study, which looked at treatment outcomes reported by ten clinicians across eight different clinical sites. St. Louis Children’s Hospital has successfully treated several pediatric strokes with tenecteplase and contributed cases to this study. The paper stated a significant majority of the pediatric patients who were hospitalized for symptoms of stroke and received tenecteplase recovered enough to safely go home following treatment. None of the physicians reported troubling aftereffects in their patients from the treatment .
Acute arterial ischemic stroke is a type of stroke caused when blockage in an artery interrupts blood flow to the brain. The standard treatment for this type of stroke is thrombolysis, which is an intervention designed to break up any blood clots that may cause blood flow issues. After initially being approved by the FDA in 1987 for treating myocardial infarction in adults , alteplase became the most commonly used medication to treat ischemic stroke in both adults and children, though it also is not yet FDA-approved for use in pediatric stroke.
However, tenecteplase has increasingly been replacing alteplase as the standard treatment for ischemic stroke in adults. Unlike alteplase, which is administered as an intravenous (IV) injection followed by an hourlong infusion process, tenecteplase is administered as a single IV injection.
“Thrombolytic medications are high hazard medications,” said Kara Kniska, PharmD, BCPPS, a St. Louis Children’s Hospital PICU Clinical Pharmacy Specialist involved with implementing the tenecteplase protocol at the hospital . “So I’m excited to have an option for the treatment of acute ischemic stroke in pediatrics that is easy to administer like tenecteplase, as compared to alteplase, which is more complicated and therefore creates more opportunities for errors.”
Tenecteplase has also been shown in adults with strokes to have equal or lower risk of bleeding in the brain — a potential post-treatment complication for medications intended to dissolve blood clots — when compared to alteplase. In light of these and other benefits like cost, many stroke and emergency care centers have switched to stocking tenecteplase as their primary treatment option for adults with stroke.
What does this mean for children and adolescents in need of medical care for stroke-like symptoms? Often, the initial medical facility the child is taken to has only tenecteplase on hand as a treatment option. Even when alteplase is available, the emergency room’s stroke protocols or a clinician’s judgement may result in the use of tenecteplase as the first line of treatment. However, unlike alteplase, which has some limited pediatric safety data collected over the years, there was no documented data on the potential safety risks for the use of tenecteplase to treat children with stroke, until this new study was published. And more work remains to be done.
“The very next step is continuing to collect data through this survey as well as organizations like the International Pediatric Stroke Study (IPSS) and the Pediatric Neurocritical Care Research Group (PNCRG),” said Guilliams, who is chief of the Pediatric Neurocritical Care Medicine section at WashU Medicine and is involved with both the IPSS and the PNCRG. “We’ve already doubled the number of children who have received tenecteplase in the database, because we want to make sure the first eleven cases were truly representative and we aren’t missing any safety signals.” After that, further clinical studies are needed to establish a standardized diagnostic criteria and optimal dosage for treating pediatric stroke with tenecteplase. Guilliams said the ultimate goal is “having [tenecteplase] move toward being an FDA-approved treatment for pediatric stroke. That would be huge for kids.”
Tammara Bava, BSN, RN, who was the pediatric neurocritical care nurse coordinator when the tenecteplase protocol was first implemented at St. Louis Children’s Hospital in February 2023, still remembers the first pediatric patient who was treated with tenecteplase. “I was jumping up and down and telling my fiancé about the amazing breakthrough that was occurring in the world of pediatric stroke,” she recalls. “TNK has had a huge positive impact in the patients we see at Children’s. From cost to the administration of the medication, it has improved the way we care for these kiddos. I’m excited to continue to see the amazing outcomes that we’ve seen so far.”
Sun LR, Lee S, Lee-Eng J, Barry M, Galardi MM, Harrar D, Hassanein SM, Rivkin MJ, Torres M, Wilson JL, Amlie-Lefond C, Guilliams K; as the International Pediatric Stroke Study and the Pediatric Neurocritical Care Research Group. Tenecteplase for the treatment of pediatric arterial ischemic stroke: A safety surveillance report. Neurology. Feb. 11, 2025. DOI: 10.1212/WNL.0000000000210310.