Five years of experience with hydroxyurea in children and young adults with sickle cell disease

Citation
A. Ferster et al., Five years of experience with hydroxyurea in children and young adults with sickle cell disease, BLOOD, 97(11), 2001, pp. 3628-3632
Citations number
22
Categorie Soggetti
Hematology,"Cardiovascular & Hematology Research
Journal title
BLOOD
ISSN journal
00064971 → ACNP
Volume
97
Issue
11
Year of publication
2001
Pages
3628 - 3632
Database
ISI
SICI code
0006-4971(20010601)97:11<3628:FYOEWH>2.0.ZU;2-D
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 Be lgian registry of HU-treated SCD patients was created to evaluate its long- term efficacy and toxicity. The median follow-up of the 93 patients registe red is 3.5 years; clinical and laboratory data have been obtained for 82 pa tients 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 dropp ed significantly. Analysis of the 22 patients with a minimum of 5 years of follow-up confirm a significant difference in the number of hospitalization s (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 attac k 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 o f stroke, HU may be a valid alternative to chronic transfusion support.