Long-term hydroxyurea treatment in children with sickle cell disease: tolerance and clinical outcomes.
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 Treatment was also withdrawn in 5 of 6 children who had developed hypersplenism, in 3 because of a pathological transcranial Doppler, and in 2 after a stroke.
de Montalembert
Mariane
M
Service de Pédiatrie Générale, Hôpital Necker, Paris, France. mariane.demontal@nck.ap-hop-paris.fr
Brousse
Valentine
V
Elie
Caroline
C
Bernaudin
Françoise
F
Shi
Junxin
J
Landais
Paul
P
D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000150:complications / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D006918:Hydroxyurea / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D013997:Time Factors; D016896:Treatment Outcome
D006918:Hydroxyurea