Prevention of Cerebral Infarction in Sickle Cell Anemia - Comprehensive Sickle Cell Center

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00005327
Recruitment Status : Completed
First Posted : May 26, 2000
Last Update Posted : March 24, 2017
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by:
University of Southern California

Brief Summary:
To conduct a prospective study aimed at the early detection and treatment of cerebral vascular disease prior to irreversible brain injury in young children with sickle cell anemia (SCA).

Condition or disease
Anemia, Sickle Cell Blood Disease Cerebrovascular Disorders Cerebrovascular Accident

Detailed Description:


Cerebral infarction is a major contributor to childhood morbidity and mortality in sickle cell anemia (SCA)


The investigators tested the hypotheses that young children with SCA experienced a variable period of asymptomatic progressive central nervous system (CNS) vasculopathy prior to cerebral infarction; that pre-infarct CNS vasculopathy could be identified by non-invasive imaging techniques:MRI, magnetic resonance angiography (MRA), and transcranial Doppler (TCD); and that therapeutic intervention at this stage of the disease could significantly reduce the subsequent occurrence of cerebral infarction. MRI, MRA, TCD, and standardized neurologic and psychometric examinations were performed yearly in a cohort of homozygous Hb SS children enrolled at 2-4 years of age. Subjects without MRI evidence of cerebral infarction who had significant cerebral vasculopathy (cerebral arterial stenosis on MRA and/or elevated blood flow velocity on TCD) were randomized to receive either no therapy or chronic transfusion therapy, in order to determine the risk of subsequent cerebral infarction in untreated subjects with these abnormalities, and the extent to which transfusion therapy could significantly reduce the risk. Subjects with evidence of prior cerebral infarction on MRI, whether symptomatic or asymptomatic, were randomized to receive either chronic transfusion therapy alone ('standard therapy') or chronic transfusion therapy plus ticlopidine, in order to determine whether ticlopidine could significantly increase the efficacy of standard therapy in preventing recurrent cerebral infarction in SCA. Subjects with prior cerebral infarction were also offered the option of bone marrow transplantation if an HLA-identical non-SS sibling donor was available.

Study Type : Observational
Study Start Date : April 1993
Study Completion Date : March 1998

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 100 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
No eligibility criteria

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00005327

Sponsors and Collaborators
University of Southern California
National Heart, Lung, and Blood Institute (NHLBI)
OverallOfficial: Darleen Powars University of Southern California

Publications: Identifier: NCT00005327     History of Changes
Other Study ID Numbers: 4117
P60HL048484 ( U.S. NIH Grant/Contract )
First Posted: May 26, 2000    Key Record Dates
Last Update Posted: March 24, 2017
Last Verified: March 2017

Additional relevant MeSH terms:
Cerebrovascular Disorders
Anemia, Sickle Cell
Hematologic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Vascular Diseases
Cardiovascular Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn