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Blood, 1 April 2005, Vol. 105, No. 7, pp. 2685-2690.
Prepublished online as a Blood First Edition Paper on December 16, 2004; DOI 10.1182/blood-2004-07-2704.
 Previous Article | Table of Contents | Next Article 
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Hydroxyurea for sickle cell disease in children and for prevention of cerebrovascular events: the Belgian experience
Béatrice Gulbis,
David Haberman,
Dominique Dufour,
Catherine Christophe,
Christiane Vermylen,
Faustin Kagambega,
Francis Corazza,
Christine Devalck,
Marie-Françoise Dresse,
Kathleen Hunninck,
Axel Klein,
Phu Quoc Le,
Michèle Loop,
Philip Maes,
Pierre Philippet,
Eric Sariban,
Chris Van Geet, and
Alina Ferster
From the Departments of Hematology/Oncology and Radiology, Hôpital Universitaire des Enfants, Brussels, Belgium; the Department of Hematology, Hôpital Universitaire Brugmann, Brussels, Belgium; the Department of Clinical Chemistry, Hôpital Universitaire Erasme, Brussels, Belgium; the Department of Pediatric Hematology, Cliniques Universitaire Saint-Luc, Brussels, Belgium; Clinique de l'Espérance, Montegnée, Belgium; Hôpital de la Citadelle, Liège, Belgium; UZ Gent, Gent, Belgium; Paolakinderziekenhuis, Antwerpen, Belgium; and UZ Gasthuisberg, Leuven, Belgium.
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.
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