Stroke recurrence in children with sickle cell disease treated with hydroxyurea following first clinical stroke.

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.