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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 | Next Article 
Submitted July 16, 2004
Accepted December 3, 2004
Hydroxyurea for sickle cell disease in children and for prevention of cerebrovascular events. The Belgian experience
Beatrice Gulbis, David Haberman, Dominique Dufour, Catherine Christophe, Christiane Vermylen, Faustin Kagambega, Francis Corazza, Christine Devalck, Marie-Francoise Dresse, Kathleen Hunninck, Axel Klein, Phu Quoc Le, Michele Loop, Philip Maes, Pierre Philippet, Eric Sariban, Chris Van Geet, and Alina Ferster*
Department of Clinical Chemistry, Hopital Universitaire Erasme, Brussels, Belgium
Belgian Departments of Hematology/Oncology Unit and Radiology, Hopital Universitaire des Enfants, Brussels, Belgium
Department of Pediatric Hematology, Cliniques Universitaire Saint-Luc, Brussels, Belgium
Department of Hematology, Hopital Universitaire Brugmann, Brussels, Belgium
Department of Pediatric Hematology, Hopital de la Citadelle, Liege, Belgium
Department of Pediatric Hematology, U.Z. Gent, Gent, Belgium
Department of Pediatric Hematology, Paolakinderziekenhuis, Antwerpen, Belgium
Department of Pediatric Hematology, Clinique de l'Esperance, Montegnee, Belgium
Department of Pediatric Hematology, U.Z. Gasthuisberg, Leuven, Belgium
* Corresponding author; email: aferster{at}ulb.ac.be.
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. One hundred twenty seven SCD patients treated with no attempt to reach Maximal Tolerated Doses entered the Belgian Registry: 109 for standard criteria and 18 who were only at risk of stroke. 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 biological 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 one 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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