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

Cerebrovascular Accident Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00005327
• Other study ID #: 4117
• Secondary ID: P60HL048484
• Status: Completed
• Start date: April 1993
• Est. completion date: March 2003

Study information

• Verified date: March 2017
• Source: University of Southern California
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Est. completion date: March 2003
• Est. primary completion date: March 1998
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 100 Years
• Eligibility: No eligibility criteria

Related Conditions & MeSH terms

• Anemia
• Anemia, Sickle Cell
• Blood Disease
• Cerebrovascular Accident
• Cerebrovascular Disorders
• Hematologic Diseases
• Stroke

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
University of Southern California	National Heart, Lung, and Blood Institute (NHLBI)

References & Publications (2)

Powars DR, Conti PS, Wong WY, Groncy P, Hyman C, Smith E, Ewing N, Keenan RN, Zee CS, Harold Y, Hiti AL, Teng EL, Chan LS. Cerebral vasculopathy in sickle cell anemia: diagnostic contribution of positron emission tomography. Blood. 1999 Jan 1;93(1):71-9. — View Citation

Powars DR. Management of cerebral vasculopathy in children with sickle cell anaemia. Br J Haematol. 2000 Mar;108(4):666-78. Review. — View Citation