Stroke Prevention in Sickle Cell Anemia (STOP 2)

Completed

Phase 3 Results

Trial Description

To determine how long blood transfusions are needed for primary stroke prevention. Also, to determine the duration of risk associated with abnormal transcranial Doppler ultrasound (TCD) and to determine the specificity of the stroke risk model developed in STOP 1 in patients with abnormal TCD measurements.

Detailed Description

BACKGROUND:
Without intervention, 11 percent of children with sickle cell anemia will sustain a stroke before the age of 20. The STOP 2 follows on the recently completed NHLBI trial, Stroke Prevention Trial in Sickle Cell Anemia (STOP 1), which found that patients at high risk for stroke could be identified using transcranial Doppler (TCD) ultrasound, and that the incidence of stroke could be reduced by 90 percent in those children by periodic blood transfusion for at least 36 months. However, chronic intermittent blood transfusions are cumbersome, expensive and associated with morbidity from iron overload. Thus, this study to determine if transfusion can be safely halted after 30 months of treatment is critically important to the continued clinical care of patients with sickle cell disease at risk for stroke.
DESIGN NARRATIVE:
In this multicenter, randomized clinical trial, 100 children will be randomized to continue to receive periodic transfusion therapy and 50 to discontinue receiving periodic transfusion therapy. The primary endpoint will be the reversion of the transcranial Doppler velocity levels to greater than 200 cm/second, indicating return of increased risk for overt stroke. Recruitment will be in two phases. Phase I will include those patients who began transfusions before April 1, 1999. These will come mainly from the STOP 1 cohort. Those who begin transfusions after April 1, 1999 but before April 1, 2001 will be eligible for Phase II of recruitment. All patients will receive quarterly TCD examinations. Patients who revert to high risk will be offered return to transfusion. The overall design includes a three month start-up, the two phases of recruitment (established STOP patients and new patients) for a total of 36 months, 18 months of observations after recruitment ends and then 3 months of wrap-up.
The study completion date listed in this record was obtained from the "End Date" entered in the Protocol Registration and Results System (PRS) record.

Conditions

Interventions

Trial Design

  • Allocation: Randomized
  • Purpose: Prevention

Patient Involvement

Patients who have been receiving regular transfusions for 30 months will be randomized to continue or to cease transfusion treatment. Patients will be examined quarterly for a period of 18 months. Those who revert to a high risk of stroke will be reassigned to the alternate group.

Outcomes

Type Measure Time Frame Safety Issue
Primary Incidence of stroke or reversion to high risk of stroke, defined as transcranial Doppler (TCD) measured flow rate >= 200 cm/s.

Sponsors