Pediatric Stroke Awareness: Recent Advances in Diagnosis, Treatment, and Outcomes

Pediatric stroke is an underrecognized topic with a dearth of evidence regarding its incidence, diagnosis, treatment, and outcomes. In recent years, an increasing body of research has shed light on these issues, and treatment advances in the field are ongoing.

Incidence and Impact

In developed countries, available evidence suggests that stroke affects an estimated 1.2 to 5.11 per 100,000 children each year and leads to death in 15% of cases.¹ Stroke incidence and stroke-related mortality are higher in boys and among Black vs White children. Stroke incidence is also higher in Asian and Hispanic vs White children.²

Among children who survive a stroke, 60% are left with permanent deficits.¹ In those with arterial ischemic stroke, for example, studies have found that 74% had a neurologic deficit at the time of discharge, while 63% developed neurologic disability by 6 months, and 46% demonstrated mild, moderate, or severe disability at 2-year follow-up.²

In a 2024 study, researchers who analyzed posts in an online stroke community found long-term impairments affecting survivors of pediatric stroke included dizziness, memory loss, confusion, and chronic pain.³

In a June 2024 published in Stroke, the global age-standardized incidence of pediatric stroke increased between 1990 and 2019, with an average annual percentage change of 0.15% (95% CI, 0.09%-0.21%).⁴ During the same time period, a significant reduction in years of life lost to pediatric stroke was observed (average annual percentage change, -3.33%; 95% CI, -3.38% to -3.28%).

“Recent trends show a higher incidence likely due to improvements in neonatal mortality indexes, as patients with more complex medical conditions are surviving longer,” explained Brandon Lucke-Wolde, MD, PhD, neurosurgery resident at the University of Florida College of Medicine in Gainesville. “Improved access to catheters and CT [computed tomography] angiography has made detection quicker,” added Dr Lucke-Wolde.

Identifying Risk Factors for Pediatric Stroke

In a May 2024 retrospective study published in Heliyon, a team of researchers analyzed data from a large electronic health record database to identify potential risk factors for pediatric stroke in patients aged 18 and younger who were treated at academic and non-academic hospitals in the US.⁵

Among 10,688 children with stroke who were identified in the database, 59% had experienced ischemic stroke and 41% had experienced hemorrhagic stroke. For ischemic stroke, the most common risk factors were hypertension (29%-44%), trauma (19%-33%), and malignancy (11%-24%). For hemorrhagic stroke, the most common risk factors were trauma (32%-64%), malignancy (5%-19%), and arrhythmia (9%-12%). The ischemic stroke cohort demonstrated high odds ratios (ORs) for dyslipidemia, hypertension, and tobacco exposure.⁵

Other risk factors for pediatric stroke include infection, sickle cell disease, and congenital heart defects.⁶

Treatment Advances in Pediatric Stroke

Along with the growing body of research on stroke incidence and risk factors, there have been significant improvements in treatment for pediatric patients with stroke.

“The pediatric stroke community has been working hard to improve care for children with stroke, and there have been many advances for the acute treatment of pediatric ischemic stroke,” according to Lisa Sun, MD, associate professor of neurology and associate program director of the pediatric neurology residency program at Johns Hopkins University School of Medicine in Baltimore, Maryland.

Dr Sun cited substantial progress in pediatric stroke prevention in high-risk groups. For example, due to the routine use of transcranial Doppler screening and regular blood transfusion therapy when indicated, stroke prevalence in children with sickle cell anemia has decreased from approximately 11.0% to 1.0% in recent decades.⁷

Among newer developments, therapies used in the treatment of stroke in adult patients (ie, thrombolytic medication tenecteplase) are being investigated for use in the pediatric population.

Dr Sun co-authored a 2024 survey-based study of pediatric stroke experts designed to glean their experience and preferences regarding the use of tenecteplase in children.⁸

The research team received responses from 33 experts, of whom 70.0% indicated willingness to use tenecteplase in pediatric patients with stroke.⁸ However, the authors pointed to a dearth of data on the use of this therapy in children, as well as limited preparedness of pediatric hospitals to administer the medication. Dr Sun added that another similarly focused study with more data is expected to be published soon.

“The benefits of mechanical thrombectomy have also been demonstrated for children with acute stroke,” Dr Sun continued. In an international, multicenter, prospective registry study published online in October 2024, Dr Sun and her colleagues found improved functional outcomes in pediatric patients with arterial ischemic stroke caused by a large- or medium-vessel occlusion who were treated with endovascular thrombectomy vs best medical treatment.⁹ 

“These advances may improve our ability to care for children presenting with acute strokes,” Dr Sun said. “In addition, new imaging methods, or the new application of older imaging methods, is an area of active investigation to facilitate stroke diagnosis and risk stratification.”²

Clinical Recommendations for Pediatric Stroke

The American Heart Association and American Stroke Association recommend that centers establish “systems and pathways for hyperacute pediatric stroke care,” as noted in a joint scientific statement.¹⁰

Diagnosing stroke in children can be difficult due to a wide array of stroke mimics including migraines, seizures, infection, Bell palsy, demyelinating syndrome, and tumors.^6,11

Findings have shown improved time to diagnosis in centers with standardized pediatric stroke protocols,¹² which typically include “a stroke screening tool in the ED [emergency department] (based on time of onset and symptoms), code stroke activation process, dedicated stroke responder, and triage process for acute MRI [magnetic resonance imaging],” as described in a 2023 paper co-authored by Dr Sun.²

Dr Lucke-Wolde recommended that clinicians maintain a high index of suspicion for stroke in children with diseases such as sickle cell disease who present with neurologic deficits. In cases of suspected stroke, “Clinicians can involve pediatric neurology early to determine the appropriate medication and need for mechanical thrombectomy.”

After a stroke, many children experience fatigue, mental health issues, and cognitive and behavioral challenges, and it is important for clinicians to address these concerns, Dr Sun advised. “Referral to neuropsychology and mental health care should be considered, and referral to local support groups may minimize feelings of isolation for these children.”

Remaining Gaps in Pediatric Stroke Management

Despite the range of developments in pediatric stroke management, numerous remaining gaps warrant attention. “Pediatric devices still need to be optimized in terms of length, size, and appropriate stent retrievers,” Dr Lucke-Wolde elucidated.

There is also a focus on identifying genetic causes or contributors to stroke in children, Dr Sun noted. “Another gap is the role of infection and inflammation in pediatric stroke. This is currently being studied in a large multicenter study.”¹³

To further improve recognition of pediatric stroke, efforts to educate the public are ongoing. “In Maryland, we work with EMS [emergency medical services], schools, and community hospitals to provide education about the signs of stroke,” Dr Sun confirmed. The International Pediatric Stroke Organization (IPSO) has also developed educational resources for health care providers regarding pediatric stroke and cerebrovascular disease.

“Finally, really good outcome measures are still needed in pediatric stroke so we can study the impacts of different types of strokes, as well as their respective treatments,” Dr Sun concluded.