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Blood, 1 June 2001, Vol. 97, No. 11, pp. 3628-3632
RED CELLS
Five years of experience with hydroxyurea in children and young
adults with sickle cell disease
Alina Ferster,
Parvine Tahriri,
Christiane Vermylen,
Geneviève Sturbois,
Francis Corazza,
Pierre Fondu,
Christine Devalck,
Marie F. Dresse,
Walter Feremans,
Kathleen Hunninck,
Michele Toppet,
Pierre Philippet,
Chris Van
Geet, and
Eric Sariban
From the Hemato-Oncology Unit, Hôpital
Universitaire des Enfants Reine Fabiola; the Department of Pediatric
Hematology, Cliniques Universitaires St Luc; and the Department of
Hematology, Hôpital Erasme, Brussels, Belgium; Service de
Pédiatrie, Clinique de l'Espérance, Montegnée,
Belgium; Service de Pédiatrie, Hôpital de la
Citadelle, Liège, Belgium; Kliniek voor Kinderziekten,
Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven,
Belgium.
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.
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