Quick action could prevent fatal brain bleeds for children in poor nations
In resource-limited areas, waiting 3 days for factor therapy can lead to paralysis
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- Children with hemophilia in resource-poor areas face high risk of fatal brain bleeds.
- Delays in care for symptoms like seizures or confusion worsen outcomes.
- Immediate medical attention for neurological signs is crucial to prevent severe damage.
Children with hemophilia in areas with limited medical resources face a high risk of fatal brain bleeds, a danger that is dramatically worsened by delays in seeking emergency care.
A new study from Thailand reveals that without access to modern preventive therapies, more than a third of pediatric patients experienced bleeding in the central nervous system, often with devastating neurological consequences.
Researchers found that the window for survival depends heavily on speed. Fatal outcomes occurred when families waited several days after the onset of symptoms like seizures or confusion before heading to the hospital. The study serves as an urgent call for parents and caregivers to seek immediate medical attention for any new neurological complaints, especially in regions where standard preventive treatments are unavailable.
The study, “Central Nervous System Bleeding in Children With Haemophilia in Limited Resource,” was published in Haemophilia.
The gap in global hemophilia care
Hemophilia is a group of genetic disorders characterized by a deficiency in certain proteins needed for blood clotting. Specifically, hemophilia A is marked by a deficiency of clotting factor VIII, while hemophilia B is characterized by a deficiency of factor IX. Lacking these key proteins, blood doesn’t clot properly, leading to symptoms such as abnormally easy and prolonged bleeding.
In wealthy parts of the world, standard-of-care treatment for hemophilia involves prophylactic, or preventive, factor replacement therapy, in which a healthy version of the deficient protein is administered regularly to normalize clotting and prevent bleeds. But while replacement therapies are standard in rich nations, in many poorer parts of the world, patients cannot access these prophylactic treatments.
Bleeding into the central nervous system (the brain and spinal cord) can be a fatal complication of hemophilia. In wealthy nations where prophylaxis is standard, this type of bleeding is relatively rare.
In this study, scientists in Thailand reported rates of central nervous system bleeding among 55 children with hemophilia followed at their center. None of these patients had access to prophylactic treatment, and they could only receive on-demand treatment at the center when they experienced bleeds.
Of the 55 children, 19 (34.5%) had a documented history of central nervous system bleeding. Some had more than one documented brain bleed over the years of follow-up.
The researchers noted this rate is significantly higher than in developed nations. They attributed the gap to the widespread use of prophylactic therapy in high-income countries, which helps stop bleeds before they start.
Recognizing symptoms and neurological impacts
In most cases, brain bleeds occurred when patients were younger than two years, though there were also some cases of bleeds documented into adolescence. The most common symptoms associated with brain bleeds were seizures and altered consciousness. In some cases, patients also reported headaches, fever, and/or back pain. In most cases, the bleeds lacked a clear trigger, though a few were associated with injury or medical procedures. Six of the children required surgery to help manage the bleeds, and the rest received on-demand factor treatment.
Ultimately, most of the children fully recovered after their brain bleeds. However, many did not. Some experienced long-lasting neurological problems, such as paralysis or developmental delays, which were attributed to brain bleeds causing damage. Four of the children died of complications related to brain bleeding.
The researchers noted that children with the worst outcomes generally had the longest delays between symptom onset and seeking medical care. Most of the children who died weren’t brought to the hospital until several days after they started displaying signs of neurological problems. As such, the researchers emphasized that prompt recognition and treatment are crucial to optimal management of this life-threatening hemophilia complication.
“Notably, the four patients with the poorest outcomes presented more than [three] days after symptom onset, underscoring the critical importance of early recognition and prompt intervention. Delayed diagnosis may defer the initiation of factor therapy, allowing ongoing bleeding … ultimately leading to brain [damage] and death,” the scientists wrote.
