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Haematologica, Vol 91, Issue 1, 125-128
Copyright © 2006 by Ferrata Storti Foundation

Clinical Trial

Long-term hydroxyurea treatment in children with sickle cell disease: tolerance and clinical outcomes

M de Montalembert, V Brousse, C Elie, F Bernaudin, J Shi, P Landais, and

Service de Pediatrie Generale, Hopital Necker, Paris, France. mariane.demontal@nck.ap-hop-paris.fr

Two hundred twenty-five SCD children have been enrolled in a study assessing the tolerability of hydroxyurea treatment. Mean age at inclusion was 9.2+/-4.4 years, median duration of treatment was 3.8 years. Ten and 75 patients have been treated respectively for more than 10 and 5 years. No severe side effect was related to hydroxyurea treatment, which was discontinued in 81 children mainly for treatment failure (30 cases) or non-compliance (17 cases). Treatment was also withdrawn in 5 of 6 children who had developed hypersplenism, in 3 because of a pathological transcranial Doppler, and in 2 after a stroke.


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