Stroke Prevention Trial in Sickle Cell Anemia

Robert J. Adams, Virgil C. McKie, Don Brambilla, Elizabeth Carl, Dianne Gallagher, Fenwick T. Nichols, Steve Roach, Miguel Abboud, Brian Berman, Catherine Driscoll, Beatrice Files, Lewis Hsu, Anne Hurlet, Scott Miller, Nancy Olivieri, Charles Pegelow, Charles Scher, Elliott Vichinsky, Winfred Wang, Gerald WoodsAbdullah Kutlar, Elizabeth Wright, Susan Hagner, Foss Tighe, Jonathan Lewin, Joel Cure, Robert A. Zimmerman, Myron A. Waclawiw

Research output: Contribution to journalArticlepeer-review

188 Scopus citations


Stroke occurs in 7-8% of children with Sickle Cell Disease (Hb SS) and is a major cause of morbidity. Rates of recurrence have been reduced from 46-90% to less than 10% through chronic blood transfusions. Prevention of first stroke, however, would be preferable because even one stroke can cause irreversible brain injury. Transcranial Doppler (TCD) ultrasound can detect arterial blood flow rates associated with subsequent stroke risk. By combining TCD screening and a potentially effective treatment, first stroke may be prevented. The Stroke Prevention Trial in Sickle Cell Anemia (STOP) is the first stroke prevention trial in Hb SS and the first randomized, controlled use of transfusion in Hb SS. This multi-center trial is designed to test whether reducing sickle hemoglobin to 30% or less with periodic blood transfusions will reduce first-time stroke by at least 70% compared to standard care. Primary endpoints will be clinically evident symptoms of cerebral infarction with consistent findings on Magnetic Resonance Imaging and Angiography (MRI/MRA) or symptomatic intracranial hemorrhage. Secondary endpoints will be asymptomatic brain lesions detected by MRI in brain areas not involved in primary endpoints. The design calls for a 6-month start-up interval, 18 months of TCD screening and randomization, and observation for stroke from entry through month 54. Key features of the trial are standardized TCD and MRI/MRA protocols interpreted blindly, and blinded adjudication of endpoints. The sample size (60 per treatment group) is based on prospective data relating TCD velocity to risk of stroke. A time-averaged mean velocity of ≤ 200 cm/sec is associated with a 46% risk of cerebral infarction over 39 months. The sample size is sufficient to detect 70% reduction in the primary endpoint at 90% power. This trial will determine if transfusion is effective in the primary prevention of stroke. Secondary aims may further the understanding of the effects of transfusion on the brain and guide future research into cerebrovascular disease in Hb SS.

Original languageEnglish (US)
Pages (from-to)110-129
Number of pages20
JournalControlled clinical trials
Issue number1
StatePublished - Feb 1998
Externally publishedYes


  • Cerebral hemorrhage
  • Cerebral infarction
  • Children/pediatric
  • Magnetic Resonance Angiography
  • Magnetic Resonance Imaging
  • Randomized controlled trial
  • Sickle Cell Disease
  • Stroke
  • Transcranial Doppler ultrasound
  • Transfusion

ASJC Scopus subject areas

  • Pharmacology


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