BOF: General (Haemoglobinopathy)

Which ONE of the following complications of paediatric sickle cell disease has been shown to be reduced by the use of hydroxyurea from the age of 12 months?

a) Dactylitis

b) Gall bladder disease

c) Streptococcal bacteremia

d) Renal impairment

e) Transient ischemic attacks

Answer:

Dactylitis

Explanation:

The BABY HUG trial randomised around 200 infants between the age of 9 and 18 months with HbSS or HBSβ-thalassemia⁰ to receive hydroxyurea or placebo. There were significant reduction in rates of acute chest syndrome, pain crisis, dactylitis and hospitalisation overall, but not gall bladder, splenic sequestration, transient ischemic attacks or bacteremia/sepsis complications. There was no excess of bacterial infections, severe haematological toxicity or secondary malignancy in the hydroxyurea group. Gastroenteritis rates were also reduced in the hydroxyurea group but this finding was interpreted with caution due to its not having been observed before. No children died on this study but the long term impact has not been assessed.

Reference:

Wong T, Brandow WL and Lottenberg R. Update on the use of hydroxyurea therapy in sickle cell disease. Blood Dec 2014, 124 (26) 3850-3857