Clinical and hematologic effects of hydroxyurea in children with sickle cell anemia☆,☆☆,★,★★
Section snippets
Patients
(Table I). From October 1990 to November 1995, a total of 15 children (male/female ratio, 10:5) were enrolled in an open-label, single-arm hydroxyurea study approved by the institutional review board. The median age of the patients was 15.3 years (range, 4.4 to 18.8). Parental consent was obtained for all patients. Patients with SCA and frequent VOCs (group 1) or severe anemia (group 2) were considered eligible for study. Frequent VOC was defined as three or more hospitalizations for VOCs
Frequency of VOCs
When all 14 patients were considered, 138 VOCs occurred before hydroxyurea therapy during 55.2 patient-years of follow-up, for an average of 2.5 VOCs per patient-year (Table I). The total number of VOCs during hydroxyurea therapy was 31 in a period of 35.7 patient-years, for an average of 0.87 VOC per patient-year. This represents a 65% decrease in the frequency of VOCs and is statistically significant (p <0.00001).
Statistical analysis. On the basis of the pretreatment event rate of 2.5 events
DISCUSSION
In this open-label pilot study the number of VOCs per patient-year was significantly less during hydroxyurea therapy than during the pretherapy period (0.87 vs 2.5 VOCs; p <0.00001), representing a 65% reduction in the frequency of crises. However, because of the open-label design of the study, a placebo effect on decreasing the frequency of crises could not be ruled out.
A double-blind study of adults has recently shown that hydroxyurea decreases the frequency of crises by 44% in comparison
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From the Sections of Pediatric Hematology-Oncology and General Pediatrics, and the Department of Quantitative Health Sciences, New York Medical College, Valhalla
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Supported in part by a grant from Healix Health Care Inc.
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Reprint requests: S. Jayabose, MD, New York Medical College, Munger Pavilion, R.110, Valhalla, NY 10595.
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