A single safety follow-up clinic visit is captured 6 months after study treatment is completed. Eligibility Information from the National Library of Medicine Choosing to participate in a study is an important personal decision. In a placebo-controlled trial, hydroxyurea also had proven clinical efficacy for reducing the frequency of acute symptomatic vaso-occlusion, including painful events and acute chest syndrome [ 12 ].
No more than 2 phlebotomies within the 6 months before screening OR No palpable splenomegaly. The probability that a subject in the alternative therapy treatment group will experience a secondary stroke during the month treatment period is less than or equal to the corresponding probability for the standard treatment group plus 0.
Inability to receive or tolerate chronic red blood cell RBC transfusion therapy, due to any of the following: Almost all children with SCA and stroke receive erythrocyte transfusions indefinitely, and at some clinical sites more subjects have died from complications of iron overload than from the recurrent stroke. Only after subjects were approved for randomization, could they actually be randomized on the study.
When 3 measures of severity stroke, TIA, and death were analyzed together, there were similar numbers of subjects in each treatment arm 10 in the standard treatment arm, 12 in the alternative treatment arm.
Although this management approach represents an important advance, most children with abnormal TCD velocities would not have a stroke even if untreated, and chronic transfusion therapy is burdensome, is indefinite in duration, and quickly necessitates iron chelation therapy.
TIA was more common than stroke with 20 positive adjudications among 15 subjects Figure 3 Band was diagnosed when the clinical presentation and features supported stroke but without radiologic evidence of new infarction. During hydroxyurea dose escalation, overlap transfusions continued for stroke prevention until MTD was reached. Stroke in a cohort of patients with homozygous sickle cell disease. The component stroke and iron hypotheses and the overall composite hypotheses to be tested in this study can be expressed precisely as follows:.
The natural history of stroke in sickle cell disease. Pilot data suggest that hydroxyurea may effectively prevent secondary stroke, while serial phlebotomy can lead to complete resolution of iron overload.
The Relief Study This study has been completed.
Half of the participants will remain on transfusion and chelation. Hydroxyurea has been used clinically for acute complications of SCD and has been thought to be potentially protective against secondary stroke. Department of Health and Human Services. How I use hydroxyurea to treat young patients with sickle cell anemia. The index was used as a record of what the participant did, not as a record of what the participant could do.
Discontinuing prophylactic transfusions used to prevent stroke in sickle cell disease. National Library of Medicine U. During the treatment phase all possible stroke events are adjudicated within approximately 1 month of symptoms; positive decisions mark a primary endpoint and lead to cessation of study treatment for that subject. However, this end point reflected the important clinical linkage between the goals of reducing secondary stroke and removing excess iron.
The single death in the standard treatment arm occurred at week 72, a sudden event at home from pulmonary embolism with right heart failure.