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

This study has been completed.
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by:
University of Southern California Identifier:
First received: May 25, 2000
Last updated: March 22, 2017
Last verified: March 2017
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).

Anemia, Sickle Cell
Blood Disease
Cerebrovascular Disorders
Cerebrovascular Accident

Study Type: Observational

Resource links provided by NLM:

Further study details as provided by University of Southern California:

Study Start Date: April 1993
Estimated Study Completion Date: March 1998
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.


Ages Eligible for Study:   up to 100 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
No eligibility criteria
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Please refer to this study by its identifier: NCT00005327

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

Publications: Identifier: NCT00005327     History of Changes
Other Study ID Numbers: 4117
P60HL048484 ( US NIH Grant/Contract Award Number )
Study First Received: May 25, 2000
Last Updated: March 22, 2017

Additional relevant MeSH terms:
Cerebrovascular Disorders
Hematologic Diseases
Anemia, Sickle Cell
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Vascular Diseases
Cardiovascular Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn processed this record on April 28, 2017