title: Five years of experience with hydroxyurea in children and young adults with sickle cell disease.
abstract: The short-term beneficial effect of hydroxyurea (HU) in sickle cell disease (SCD) has been proven by randomized studies in children and adults. The Belgian registry of HU-treated SCD patients was created to evaluate its long-term efficacy and toxicity. The median follow-up of the 93 patients registered is 3.5 years; clinical and laboratory data have been obtained for 82 patients at 1 year, 61 at 2 years, 44 at 3 years, 33 at 4 years, and 22 after 5 years. On HU, the number of hospitalizations and days hospitalized dropped significantly. Analysis of the 22 patients with a minimum of 5 years of follow-up confirm a significant difference in the number of hospitalizations (P =.0002) and days in the hospital (P <.01), throughout the treatment when compared to prior to HU therapy. The probabilities of not experiencing any event or any vaso-occlusive crisis requiring hospitalization during the 5 years of treatment were, respectively, 47% and 55%. On HU, the rate per 100 patient-years of severe events was estimated to be 3.5% for acute chest syndrome, 1.2% for aplastic crisis, 0.4% for splenic sequestration; it was 0% for the 9 patients with a history of stroke or transient ischemic attack followed for an average of 4 years. No important adverse effect occurred. Long-term chronic treatment with HU for patients with SCD appears feasible, effective, and devoid of any major toxicity; in patients with a history of stroke, HU may be a valid alternative to chronic transfusion support. (Blood. 2001;97:3628-3632)
authors.affiliation: Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, Av J J Crocq, 15-1020 Brussels, Belgium.
authors.forename: A
authors.initials: A
authors.lastname: Ferster
authors.forename: P
authors.initials: P
authors.lastname: Tahriri
authors.forename: C
authors.initials: C
authors.lastname: Vermylen
authors.forename: G
authors.initials: G
authors.lastname: Sturbois
authors.forename: F
authors.initials: F
authors.lastname: Corazza
authors.forename: P
authors.initials: P
authors.lastname: Fondu
authors.forename: C
authors.initials: C
authors.lastname: Devalck
authors.forename: M F
authors.initials: MF
authors.lastname: Dresse
authors.forename: W
authors.initials: W
authors.lastname: Feremans
authors.forename: K
authors.initials: K
authors.lastname: Hunninck
authors.forename: M
authors.initials: M
authors.lastname: Toppet
authors.forename: P
authors.initials: P
authors.lastname: Philippet
authors.forename: C
authors.initials: C
authors.lastname: Van Geet
authors.forename: E
authors.initials: E
authors.lastname: Sariban
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000209:etiology / Q000517:prevention & control; D000755:Anemia, Sickle Cell / Q000150:complications / Q000188:drug therapy*; D000986:Antisickling Agents / Q000627:therapeutic use*; D001157:Arterial Occlusive Diseases / Q000209:etiology / Q000517:prevention & control; D002637:Chest Pain / Q000209:etiology / Q000517:prevention & control; D002648:Child; D002675:Child, Preschool; D005260:Female; D006760:Hospitalization; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use*; D007902:Length of Stay; D008297:Male; D010100:Oxygen / Q000097:blood; D012042:Registries; D013163:Splenomegaly / Q000209:etiology / Q000517:prevention & control; D020521:Stroke / Q000209:etiology / Q000517:prevention & control
chemical_list: D000986:Antisickling Agents; D010100:Oxygen; D006918:Hydroxyurea