Design of the multicenter study of hydroxyurea in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea.

Pubmed ID: 8925656

Journal: Controlled clinical trials

Publication Date: Dec. 1, 1995

Affiliation: Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.

MeSH Terms: Humans, Male, Adult, Female, Anemia, Sickle Cell, Quality of Life, Double-Blind Method, Placebos, Patient Selection, Research Design, Fetal Hemoglobin, Sample Size, Hydroxyurea, Antisickling Agents

Grants: 5-U01-HL45696, 5-U02-HL45692

Authors: McMahon RP, Terrin ML, Waclawiw M, Charache S, Moore RD, Dover GJ, Barton FB, Eckert SV

Cite As: Charache S, Terrin ML, Moore RD, Dover GJ, McMahon RP, Barton FB, Waclawiw M, Eckert SV. Design of the multicenter study of hydroxyurea in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea. Control Clin Trials 1995 Dec;16(6):432-46.

Studies:

Abstract

The Multicenter Study of Hydroxyurea in Sickle Cell Anemia is a randomized double-blind placebo-controlled trial to test whether hydroxyurea can reduce the rate of painful crises in adult patients who have at least three painful crises per year. The sample size of 299 patients yields at least 90% power to detect a 50% or greater reduction in crisis rate. Dosage starts at 15 mg/kg/day and is titrated to the patient's maximum tolerated dose up to 35 mg/kg/day. Placebo dosage is titrated in similar fashion to maintain blinding. Attempts are made to ascertain medical contacts for at least 2 years after study entry. The Core Laboratory, Treatment Distribution Center, and Data Coordinating Center collaborate to provide standardized monitoring for toxicity and dose adjustments. The Core Laboratory also reduces the possibility of inadvertent unmasking of treatment assignment during review of hematologic data in clinical centers. An independent Crisis Review Committee classifies clinical events to assure that outcome evaluations are standardized and unbiased by knowledge of treatment assignments. The Data and Safety Monitoring Board assures scientific integrity of the study, as well as the safety and ethical treatment of study patients. We expect the study to determine whether or not treatment with hydroxyurea can offer significant clinical benefit to patients with the most common hereditary disorder among African-Americans in the United States.