Five years of experience with hydroxyurea in children and young adults with sickle cell disease.
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)
Ferster
A
A
Hemato-Oncology Unit, Hôpital Universitaire des Enfants Reine Fabiola, Av J J Crocq, 15-1020 Brussels, Belgium.
Tahriri
P
P
Vermylen
C
C
Sturbois
G
G
Corazza
F
F
Fondu
P
P
Devalck
C
C
Dresse
M F
MF
Feremans
W
W
Hunninck
K
K
Toppet
M
M
Philippet
P
P
Van Geet
C
C
Sariban
E
E
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
D000986:Antisickling Agents; D010100:Oxygen; D006918:Hydroxyurea