title: Hydroxyurea for sickle cell disease in children and for prevention of cerebrovascular events: the Belgian experience.
abstract: 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.
authors.affiliation: Department of Hematology/Oncology, Hôpital Universitaire des Enfants, Av. J. J. Crocq, 15, B-1020 Brussels, Belgium.
authors.forename: Béatrice
authors.initials: B
authors.lastname: Gulbis
authors.forename: David
authors.initials: D
authors.lastname: Haberman
authors.forename: Dominique
authors.initials: D
authors.lastname: Dufour
authors.forename: Catherine
authors.initials: C
authors.lastname: Christophe
authors.forename: Christiane
authors.initials: C
authors.lastname: Vermylen
authors.forename: Faustin
authors.initials: F
authors.lastname: Kagambega
authors.forename: Francis
authors.initials: F
authors.lastname: Corazza
authors.forename: Christine
authors.initials: C
authors.lastname: Devalck
authors.forename: Marie-Françoise
authors.initials: MF
authors.lastname: Dresse
authors.forename: Kathleen
authors.initials: K
authors.lastname: Hunninck
authors.forename: Axel
authors.initials: A
authors.lastname: Klein
authors.forename: Phu Quoc
authors.initials: PQ
authors.lastname: Le
authors.forename: Michèle
authors.initials: M
authors.lastname: Loop
authors.forename: Philip
authors.initials: P
authors.lastname: Maes
authors.forename: Pierre
authors.initials: P
authors.lastname: Philippet
authors.forename: Eric
authors.initials: E
authors.lastname: Sariban
authors.forename: Chris
authors.initials: C
authors.lastname: Van Geet
authors.forename: Alina
authors.initials: A
authors.lastname: Ferster
mesh_terms: 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*
chemical_list: D000986:Antisickling Agents; D006918:Hydroxyurea