Stroke Prevention in Sickle Cell Anemia (STOP 1)

This study has been completed.
Sponsor:
Information provided by:
National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier:
NCT00000592
First received: October 27, 1999
Last updated: November 18, 2005
Last verified: November 2005
  Purpose

To reduce episodes of first time stroke by 75 percent in children with sickle cell anemia by the administration of prophylactic transfusion therapy.


Condition Intervention Phase
Anemia, Sickle Cell
Cerebral Embolism and Thrombosis
Cerebrovascular Disorders
Hematologic Diseases
Hemoglobinopathies
Procedure: blood transfusion
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Primary Purpose: Prevention

Resource links provided by NLM:


Further study details as provided by National Heart, Lung, and Blood Institute (NHLBI):

Study Start Date: July 1994
Estimated Study Completion Date: August 2000
Detailed Description:

BACKGROUND:

Stroke, occurring in about 10 percent of pediatric patients with sickle cell disease, is one of the most devastating complications, with a high recurrence rate after the first episode. Several non-randomized studies have shown reduction in stroke recurrence when periodic blood transfusions are administered to maintain hemoglobin S under 30 percent. Periodic blood transfusions are associated with significant risks of iron overload and other complications and must be accompanied by parenteral iron chelation therapy. However, this has become a standard of care for prevention of recurrent stroke in SS children. Thus, a randomized trial of blood transfusion for secondary prevention would not be feasible because it would be considered unethical. Based on various studies, the recurrence rate is reduced from 46 to 67 percent to approximately 7 percent on transfusion therapy. Because most stroke patients are left with some neurological deficit, and face a lifetime of disability, primary prevention would have a significant impact on the management of patients. However, because of complications of blood transfusions, the hypothesis should be proven by a randomized clinical trial.

A primary prevention trial had not been possible because an acceptable means of detecting those children at risk of stroke was not available. The advent of TCD to identify arterial abnormalities for the prediction of stroke has provided a means of detection. TCD abnormalities have a high specificity (100 percent) and high sensitivity (90 percent) for detecting angiographically proven narrowing of arterial diameter. Thus, TCD examination of the basal cerebral arteries is predictive of who will develop a stroke.

DESIGN NARRATIVE:

Randomized, Phase III, multicenter. Approximately 3,000 children from 12 clinics were screened with transcranial Doppler (TCD). A total of 130 were randomized to receive either standard supportive care or periodic blood transfusions if they were found to be at high risk of stroke on the basis of elevated cerebral blood flow as measured by TCD screening tests. Primary endpoints included clinically evident symptoms of cerebral infarction with consistent findings on magnetic resonance imaging (MRI), and/or symptomatic intracranial hemorrhage. Secondary endpoints included asymptomatic brain lesions detected by MRI in brain areas not involved in primary endpoints. Hematologic characteristics of the high risk group were analyzed and serum and DNA samples frozen for future analysis. Recruitment ended in October 1997 with the accrual of 130 subjects. The clinical phase ended in 1999.

  Eligibility

Ages Eligible for Study:   2 Years to 16 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Pediatric patients, ages 24 months to 16 years, with sickle cell anemia or S-beta zero thalassemia.

  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00000592

Sponsors and Collaborators
Investigators
Investigator: Donald Brambilla New England Research Institute Inc.
  More Information

Publications:

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
ClinicalTrials.gov Identifier: NCT00000592     History of Changes
Other Study ID Numbers: 312
Study First Received: October 27, 1999
Last Updated: November 18, 2005
Health Authority: United States: Federal Government

Additional relevant MeSH terms:
Embolism and Thrombosis
Intracranial Embolism and Thrombosis
Anemia
Anemia, Sickle Cell
Cerebrovascular Disorders
Hematologic Diseases
Hemoglobinopathies
Thrombosis
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Brain Diseases
Cardiovascular Diseases
Central Nervous System Diseases
Genetic Diseases, Inborn
Nervous System Diseases
Thromboembolism
Vascular Diseases

ClinicalTrials.gov processed this record on October 20, 2014