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  • A stroke is a general term used to describe an injury to the brain caused by either bleeding (referred to as hemorrhagic) or a lack of oxygen (referred to as ischemic).

    • A stroke usually implies some type of permanent injury to the brain
    • The term infarct or infarction may also refer to a stroke

    Here at Boston Children's Hospital, our Department of Neurosurgery works with many other health care teams throughout the hospital in a multidisciplinary effort dedicated to the treatment of children with strokes.

    How Boston Children's Hospital approaches pediatric stroke

    Our physicians are committed to a rigorous understanding of which practices constitute the very best standard of care for your child. Strokes and related disorders can have broad-reaching effects.  At Children's, we aim to understand and treat all elements of a child's condition.

    • Our Cerebrovascular Disorders and Stroke (CVD and Stroke) Program provides:
      • Fast, comprehensive evaluations to quickly identify if a stroke has occurred and why
      • Combined expertise in child neurology, pediatric neurosurgery, hematology, neurointerventional radiology, pediatric neuroradiology, emergency medicine, child psychiatry, physical and occupational therapy, and speech and language therapy, in one specialized program
      • Access to therapies administered in the earliest stages of stroke that are designed to remove blockages of brain blood vessels
      • Direct referral to physical therapists, occupational therapists, or speech and language therapists to help children address and improve any affected functions
      • Long-term multidisciplinary care to prevent additional strokes 
    • Our Neurosurgical Stroke Program provides exceptional care for children of all ages with strokes or related conditions that call for neurosurgical treatment.
  • What causes stroke?

    Brain cells are incredibly delicate and require a steady supply of oxygen. Even brief interruptions in the delivery of oxygen can cause injury.

    Strokes are caused by three main mechanisms:

    • The blood supply to the brain may be blocked.  This may be caused by tiny clots (emboli or thrombi) plugging the blood vessels of the brain.  Or the vessels themselves may be narrowed, reducing the amount of blood delivered to the brain (as is seen in Moyamoya Syndrome, for example).
    • The blood may not have enough oxygen in it to begin with.  For example, cases when someone can't breathe for long periods of time or in the setting of carbon monoxide poisoning. Or the blood may not be circulating fast enough to bring fresh oxygen to the brain.  For example as might occur if the heart is not beating appropriately.
    • The brain itself might be under pressure.  For example as occurs in brain swelling after trauma or when there is bleeding around the brain. Or there may be bleeding within the substance of the brain itself, which directly injures the brain tissue and also makes it harder for blood with oxygen to be delivered to it. This increased pressure often prevents oxygenated blood from the heart from entering the confines of the skull.

    In all of these cases, the injury to the brain tissue and the lack of oxygen cause some of the brain cells to die, resulting in a stroke.

    What are some conditions that are associated with strokes?

    The following genetic conditions may lead to stroke:

    • Deficiencies of the anticoagulant (blood clot preventing) proteins C and S
    • Deficiency of antithrombin-III, another anticoagulant
    • A mutation in the blood clotting factor prothrombin G20210A
    • Sickle cell disease, in which red blood cells are abnormally shaped
    • Activated protein C resistance, where the body resists the anticoagulant protein C 
    • Dyslipoproteinemias, abnormal concentrations or abnormal lipoproteins (cholesterol molecules) in the blood
    • Homocystinuria, where the body is unable to process amino acids properly
    • Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS), a condition that affects the brain and nervous system and is a result of defect in the mitochondrial genome (inherited exclusively from the mother's side).
    • Fabry disease, which leads to an inability to metabolize fat
    • Menkes disease, which interferes with copper metabolism
    • Tangier disease, a severe reduction in the body's good cholesterol

    Embolic strokes are usually caused by an embolus - a blood clot that forms elsewhere in the body and travels to the brain. Embolic strokes may be associated with the following conditions:

    Thrombotic stroke are caused by a thrombus, or blood clot, that develops in the arteries that supply blood to the brain.  Thrombotic strokes may be associated with the following conditions:

    • Factor V Leiden mutation, which leads to abnormal blood clotting
    • Antiphospholipid antibodies, which leads to blood clots in both arteries and veins
    • Hyperhomocysteinemia, which refers to an abnormally high level of the amino acid homocysteine in the blood
    • Elevated lipoprotein (a), a type of cholesterol

    Or acquired, such as deficiencies in clotting pathways resulting from:

    • Infection
    • Medications
    • Hepatic (liver) or renal (kidney) disease.

    Vasculitis, which refers to blood vessel inflammation, may be caused by infectious or noninfectious conditions. 

    Infections causes include:

    Noninfectious causes encompass a wide variety of autoimmune disorders, including:

    Stokes may also be caused by

    • Illicit drug use
    • Fibromuscular dysplasia, an abnormal cluster of cells growing in the artery wall
    • Arteriovenous Malformations (AVMs), an abnormal cluster of blood vessels
    • Arteriovenous Fistulas (AVFs), an abnormal connection between an artery and vein
    • Vein of Galen Malformations (VOGMs), a blood vessel malformation in the brain
    • Aneurysms, a bulge or ballooning in an artery wall
    • Moyamoya Syndrome, a disease in which the brain arteries are constricted
    • Venous thrombosis, a blood clot that forms within a vein
    • Arterial ischemic stroke, blockage of blood flow in an artery
    • Arterial dissection (vertebral dissection, carotid dissection), a tear in the artery lining

    What are the symptoms of a stroke?

    Stroke symptoms vary widely depending on which part of the brain has been injured.  Some parts of the brain can suffer strokes with little or no recognizable symptoms, referred to as "silent" strokes.  Other strokes, even very small ones, can cause devastating symptoms, such as paralysis, blindness or even death, if they occur in sensitive areas of the brain.          

  • Your child's physician will make a diagnosis with physical examination and diagnostic tests. During the examination the physician obtains a complete medical history of your child.

    Diagnostic procedures include:

    • Computerized tomography scan (Also called a CT or CAT scan.) - a diagnostic imaging procedure that uses a combination of x-rays and computer technology to produce cross-sectional images (often called "slices"), both horizontally and vertically, of the body. A CT scan shows detailed images of any part of the body, including the bones, muscles, fat, and organs. CT scans are more detailed thangeneral x-rays and may help the doctor determine what type of fracture your child has.
    • Magnetic resonance imaging (MRI) - a diagnostic procedure that uses a combination of large magnets, radiofrequencies, and a computer to produce detailed images of organs and structures within the body. This test is done to rule out any associated abnormalities.
    • Blood tests
  • The treatment of a stroke has two parts:

    • The underlying cause of the stroke (such as high brain pressure, narrowed blood vessels or an increased tendency of the blood to clot inappropriately) should be diagnosed and corrected, if possible.
    • Regardless of the cause of the stroke, the problems that result from the stroke (weakness, numbness, etc.) can sometimes be improved with therapy over time. Identifying which patients may benefit from specialized therapy might require evaluation by specific health care professionals, such as neurologists, rehabilitation medicine physicians, or physical, occupational or speech therapists.

    At Boston Children's Hospital, the Department of Neurosurgery works with many other health care teams here in a multidisciplinary effort dedicated to the treatment of children with strokes.

  • Otx2

    Researchers have long sought a factor that can switch on the brain's ability to learn. Now, research led by Takao Hensch, PhD, of Boston Children's Hospital's FM Kirby Neurobiology Center and the Department of Neurology, has identified such a trigger. Called Otx2, it signals certain cells in the cortex (parvalbumin cells) to mature and initiate a critical period—a time window when the brain can readily rewire itself.

    Surprisingly, the signal actually comes from the eye—but only after the eye has matured enough to provide good vision. Hensch speculates that other sensory organs may send similar signals to the brain as they mature, triggering critical periods for hearing, smell, etc.

    Controlling the onset of plasticity could help in developmental disorders like autism, in which critical periods are thought to be mis-timed. "If the timing is off, the brain won't set up its circuits properly," says Hensch. Launching a critical period might also help people recover from stroke or brain injury, or learn languages or musical instruments as easily as young children, adds Hensch, who last fall won the highly competitive National Institutes of Health Director's Pioneer Award. He also speculates that Otx2 could be harnessed to carry drugs from the eye to the brain, envisioning eye drops for disorders like amblyopia (lazy eye). Sayaka Sugiyama, PhD, postdoctoral research fellow, was first author of the study, published in Cell on August 8.


    Because injured neurons in the brain or spinal cord can't grow back, damage fromspinal cord injury, stroke or other forms of brain injury can't be repaired. But researchers led by Zhigang He, PhD, a neurologist at Boston Children's Hospital, have found a way to overcome natural inhibitory mechanisms that suppress regeneration, causing nerve fibers to re-grow vigorously.

    Previous studies, including some from He's lab, tried to spur re-growth by removing inhibitory molecules from the neurons' environment. But this approach had only modest effects. He's team, collaborating with Children's neurologist Mustafa Sahin, MD, PhD, now shows that re-growth is primarily regulated from inside the cells. Using genetic techniques, the researchers deleted two of these internal regulators in mice with injured optic nerves. This allowed a growth pathway called mTOR—normally silenced in mature neurons—to become active again. As a result, up to 50 percent of injured neurons survived, versus about 20 percent in mice without the genetic deletions. Up to 10 percent of the mice showed significant, long-distance re-growth of nerve fibers.

    He believes it may be possible to accomplish the same re-growth with drugs. But the next step is to determine whether the regenerating fibers can actually restore function.Kevin Park, PhD, Kai Liu, PhD, Yang Hu, PhD, and Patrice Smith, PhD, all of Neurobiology, were coauthors on the paper, published in Science on November 7.


    Researchers at Children's have discovered an enzyme (known as Mst3b) that's essential for regenerating damaged nerves and may hold promise as a possible treatment for brain and spinal cord injury.

    Inosine for spinal cord injury or stroke

    Neuroscientist Dr Larry Benowitz has found that inosine, a naturally occurring cellular compound improves motor function after spinal cord injury or stroke and is pursuing potential treatments. 

    MoyaMoya Disease

    Neurosurgeon-in-chief, Michael Scott, MD, has pioneered an operation that improves oxygenation of the brain and arrests Moyamoya disease.  He hopes his research will lead to ways of predicting strokes and stimulating blood vessel growth so patients can recover more quickly or avoid stroke altogether.

    Sickle Cell Anemia Risk

    Researchers at Children's have developed a novel approach to predicting the risk of stroke in sickle cell anemia patients by looking at genetic variations.


    Larry Benowitz, PhD, of the Children's Department of Neurosurgery lead a study using molecular therapies to promote the growth of new circuits in nerves in rats. This study could lead to molecular treatment for strokes in humans. Learn more about this exciting research in the Children’s newsroom.

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