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)

journal_name

Blood

journal_title

Blood

authors

Ferster A,Tahriri P,Vermylen C,Sturbois G,Corazza F,Fondu P,Devalck C,Dresse MF,Feremans W,Hunninck K,Toppet M,Philippet P,Van Geet C,Sariban E

doi

10.1182/blood.v97.11.3628

subject

Has Abstract

pub_date

2001-06-01 00:00:00

pages

3628-32

issue

11

eissn

0006-4971

issn

1528-0020

journal_volume

97

pub_type

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