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

Completed

• Conditions: Blood Disease; Cerebrovascular Disorders; Anemia, Sickle Cell; Cerebrovascular Accident

• Registration Number: NCT00005327
• Lead Sponsor: 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).

Detailed Description

BACKGROUND:

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

DESIGN NARRATIVE:

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 Timeline

• Study Start: 1993-04
• Primary Completion: 1998-03
• Study First Submitted: 2000-05-25
• Study First Submitted QC: 2000-05-25
• Study First Posted: 2000-05-26
• Study Completion: 2003-03
• Status Verified: 2017-03
• Last Update Submitted: 2019-07-02
• Last Update Posted: 2019-07-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified