Hydroxyurea for sickle cell disease in children and for prevention of cerebrovascular events: the Belgian experience.
Hydroxyurea (HU) is considered to be the most successful drug therapy for severe sickle cell disease (SCD). Nevertheless, questions remain regarding its benefits in very young children and its role in the prevention of cerebrovascular events. There were 127 SCD patients treated with no attempt to reach maximal tolerated doses who entered the Belgian Registry: 109 for standard criteria and 18 who were at risk of stroke only. During 426 patient-years of follow-up for patients with standard criteria, 3.3 acute chest syndromes, 1.3 cerebrovascular events, and 1.1 osteonecrosis per 100 patient-years were observed. A subgroup of 32 patients followed for 6 years experienced significant benefit over this period. In each subgroup of children (younger than 2 years, 2-5, 6-9, and 10-19 years) followed for 2 years, clinical and biologic changes were similar, except for children younger than 2 years who had no total hemoglobin increase and remained at risk of severe anemia. In 72 patients evaluated by transcranial Doppler studies (TCD), 34 patients were at risk of primary stroke and only 1 had a cerebrovascular event after a follow-up of 96 patient-years. These results confirm the benefit of HU, even in very young children, and its possible role in primary stroke prevention.
Gulbis
Béatrice
B
Department of Hematology/Oncology, Hôpital Universitaire des Enfants, Av. J. J. Crocq, 15, B-1020 Brussels, Belgium.
Haberman
David
D
Dufour
Dominique
D
Christophe
Catherine
C
Vermylen
Christiane
C
Kagambega
Faustin
F
Corazza
Francis
F
Devalck
Christine
C
Dresse
Marie-Françoise
MF
Hunninck
Kathleen
K
Klein
Axel
A
Le
Phu Quoc
PQ
Loop
Michèle
M
Maes
Philip
P
Philippet
Pierre
P
Sariban
Eric
E
Van Geet
Chris
C
Ferster
Alina
A
D000293:Adolescent; D000328:Adult; D000755:Anemia, Sickle Cell / Q000188:drug therapy* / Q000453:epidemiology; D000986:Antisickling Agents / Q000008:administration & dosage* / Q000009:adverse effects; D001530:Belgium / Q000453:epidemiology; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage* / Q000009:adverse effects; D007223:Infant; D008297:Male; D010349:Patient Compliance; D012042:Registries; D012307:Risk Factors; D055502:Secondary Prevention; D020521:Stroke / Q000453:epidemiology / Q000517:prevention & control*
D000986:Antisickling Agents; D006918:Hydroxyurea