Hydroxyurea therapy in children severely affected with sickle cell disease☆,☆☆,★
Section snippets
Patient eligibility
Patients between the ages of 10 and 17 years with a diagnosis of hemoglobin SS, hemoglobin SS-α-thalassemia, or hemoglobin S-β0-thalassemia judged to be clinically severe on the basis of at least three or more hospitalizations for acute painful events (pain crises, acute chest syndrome, or priapism) per year for a minimum of 1 year were deemed eligible for enrollment. The protocol for the trial was approved by the institutional review board of each respective institution. The form for informed
RESULTS
Twenty-five individuals severely affected with SCD were eligible for study, 12 from the University of Illinois at Chicago and 13 from the Medical College of Wisconsin-Children's Hospital of Wisconsin. Fifteen patients enrolled in the hydroxyurea trial. Of the patients and families who refused to enter the trial, the reasons given included unwillingness to participate in a clinical trial of a drug that is a chemotherapeutic agent, unwillingness to comply with the protocol because of frequent
DISCUSSION
Frequent (≥3 per year) vaso-occlusive pain crises cause severe morbidity in children with SCD and have been shown to be predictors of early death in adults with SCD.24 The treatment of children has been difficult because bone marrow transplantation poses a significant risk of death and morbidity and because long-term transfusion programs inevitably lead to iron overload, with a resultant need for chelation therapy. Hydroxyurea represents the first oral agent shown to be of benefit in reducing
Acknowledgements
We thank Griffin Rodgers, MD, for the determination of α-genotype.
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From the Department of Pediatrics, Medical College of Wisconsin; the Wisconsin Sickle Cell Disease Comprehensive Care Center, Children's Hospital of Wisconsin; and the Blood Research Institute of the Blood Center of Southeastern Wisconsin, Milwaukee; and the Department of Pediatrics, University of Illinois at Chicago
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Reprint requests: J. Paul Scott, MD, PO Box 2178, Blood Center of Southeastern Wisconsin, Milwaukee, WI 53201-2178.
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0022-3476/96/$5.00 + 0 9/20/73086