Stroke Prevention Trial in Sickle Cell Anemia (STOP): extended follow-up and final results - PubMed (original) (raw)

Randomized Controlled Trial

Stroke Prevention Trial in Sickle Cell Anemia (STOP): extended follow-up and final results

Margaret T Lee et al. Blood. 2006.

Abstract

The Stroke Prevention Trial in Sickle Cell Anemia (STOP) was a randomized trial to evaluate whether chronic transfusion could prevent initial stroke in children with sickle-cell anemia at high risk as determined by transcranial Doppler (TCD). The trial demonstrated a large benefit of transfusion and was halted early. After termination of the trial, patients participated in a post-trial follow-up study. More patients in the transfusion group (70%) elected transfusion for primary stroke prevention compared with those on standard care (45%). Six patients with persistently abnormal TCD results developed stroke. A minority with initially abnormal TCD results remained stroke-free without transfusion. Except for lower baseline and follow-up TCD velocities compared with those with stroke, no predictive features of this apparent lower-risk subgroup could be determined. TCD results at last testing in 108 patients that did not have stroke were: normal (44.4%), conditional (26.9%), abnormal (22.2%), and inadequate (6.5%). Patients on transfusion were more likely to have normal TCD results. Transfusion resulted in iron overload and alloimmunization, but no infection. The study provides new information on acceptance rates and long-term effects of transfusion. Persistent TCD elevation signals ongoing stroke risk. Reduction in TCD results over time without transfusion is observed in some patients and requires further study.

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Figures

Figure 1.

Figure 1.

Treatment history. (A) Transfusion group. (B) Standard care group. Dates indicate study time points: February 1995 being the start of STOP Trial and September 1997, the end; January 1998 is the start of post-trial followup, and June 2000, its end. Numbers in parentheses represent the number of patients.

Figure 2.

Figure 2.

Kaplan-Meier estimates of the probability of remaining stroke-free among patients receiving transfusion and patients on standard care. The P value was calculated by log-rank test. Tick marks indicate the lengths of observation of patients who did not have a stroke. Four patients were not included in this analysis: 1 patient from the standard care group who experienced intracerebral hematoma as noted on baseline MRI, and 3 patients who developed stroke after change in treatment during the posttrial follow-up (2 from the standard care and 1 from the transfusion group).

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References

    1. Ohene-Frempong K, Weiner S, Sleeper L, et al. Cerebrovascular accidents in sickle cell disease: rates and risk factors. Blood. 1998;91: 288-294. - PubMed
    1. Adams R, McKie V, Nichols F, et al. The use of transcranial ultrasonography to predict stroke in sickle cell disease. N Engl J Med. 1992;326: 605-610. - PubMed
    1. Adams RJ, McKie VC, Carl EM, et al. Long-term stroke risk in children with sickle cell disease screened with transcranial Doppler. Ann Neurol. 1997;42: 699-704. - PubMed
    1. Adams R, McKie V, Hsu L, et al. Prevention of a first stroke by transfusion in children with abnormal results of transcranial Doppler ultrasonography. N Engl J Med. 1998;339: 5-11. - PubMed
    1. NHLBI Clinical Alert: Periodic transfusions lower stroke risk in children with sickle cell anemia (http://www.nlm.nih.gov/databases/alerts/sickle97.html). Bethesda, MD: National Institutes of Health, 1997. Accessed August 6, 2005.

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