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Stroke recurrence in children with sickle cell disease treated with hydroxyurea following first clinical stroke
Susanna Bortolusso Ali1, Michelle Moosang, Lesley King
1Sickle Cell Unit, TMRI, University of the West Indies, Jamaica. susanna.ali@uwimona.edu.jm
Insights
Hydroxyurea (HU) is an effective alternative to chronic transfusions for preventing stroke recurrence in children with sickle cell disease (SCD). This intervention significantly reduces stroke risk and associated complications when blood transfusions are not feasible.
Area of Science:
- Pediatric Hematology
- Neurology
- Public Health
Background:
- Stroke is a major complication of sickle cell disease (SCD) in children.
- Chronic transfusion therapy is the standard for preventing stroke recurrence.
- Access to transfusions is limited in many developing regions.
Purpose of the Study:
- To evaluate hydroxyurea (HU) as an alternative to transfusion for preventing stroke recurrence in children with SCD.
- To assess the impact of HU on stroke recurrence, mortality, and disability in this population.
Main Methods:
- Retrospective cohort study of 43 children with a first clinical stroke between 2000 and 2009.
- Comparison of outcomes between children who received HU and those who did not.
- Analysis of stroke recurrence, mortality, disability, and educational needs.
Main Results:
- Only 1 of 10 children on HU experienced stroke recurrence (2/100 person-years) versus 20 of 33 not on HU (29/100 person-years).
- HU use was associated with a significantly lower risk of stroke recurrence (HR 9.4, P=0.03).
- The non-HU group had higher rates of mortality, moderate-severe physical disability, and special educational needs.
Conclusions:
- Hydroxyurea is a viable and effective intervention for preventing stroke recurrence in children with SCD.
- HU offers a crucial alternative when chronic transfusion programs are impractical or unavailable.
- This approach can mitigate severe neurological deficits and improve long-term outcomes in pediatric SCD patients.
Abstract:
Chronic transfusion therapy is the treatment of choice for preventing stroke recurrence in children with sickle cell disease (SCD). The majority of children affected by this devastating complication live in the developing world where access to regular blood transfusions may be impractical. Since 2000, in the absence of regular blood supplies, all children at the Sickle Cell Unit who had experienced a first clinical stroke were offered hydroxyurea (HU) as the only intervention to prevent stroke recurrence. Forty-four children were identified as having experienced a first clinical stroke between January 1, 2000 and September 30, 2009; one died at that presentation. Forty-three children were therefore followed for 111 person-years, of whom 10 (23.3%) agreed to start HU. Only one child in the HU group, incidence rate 2/100 person-years, had clinical stroke recurrence, compared to 20/33 in the non-HU group, incidence rate 29/100 person-years (Hazard ratio (HR) 9.4 [95% Confidence interval (CI): 1.3-70.6]; P = 0.03). When the groups were compared, in the non-HU group, four died (vs. zero), 13 (53% vs. 10%) had moderate-severe physical disability (P = 0.017), and 12 (44% vs. 20%) required special education or were too disabled to attend school. Our data support the role of HU as a useful intervention for prevention of stroke recurrence in SCD when transfusion programs are not available or practical.
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