Stroke prevention in sickle cell disease (stop): Final results

R. Adams, D. Brambilla, V. McKie, B. Files, E. Vichinsky, M. Abboud, L. Hsu, C. Pegelow, G. Woods, S. Miller, W. Wang, N. Olivieri, B. Berman, A. Hurlet, S. Piomelli, C. Driscoll

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6 Scopus citations

Abstract

The STOP Trial was a randomized controlled trial to determine if chronic blood transfusion reduced first time stroke in children with Hb SS. Over 2,000 patients were screened with transcranial Doppier (TCD). Sixty-three were randomized to transfusion (goal: to reduce HbS to < 30%) and 67 to standard care between January 1995 and October 1996. The trial was halted in September 1997, after demonstration of a large beneficial treatment effect. Patients were offered transfusion and follow-up through the end of the study in June 2000. The status of 129 were known at close-out. Two died of causes unrelated to stroke. Fourteen patients (excluding one with stroke at randomization) not receiving transfusion had stroke while a total of 3 children had stroke while on transfusion for an average of 28 months. One other had a recurrent stroke despite being on transfusion. Forty-eight of the original 63 randomized to transfusion continued, one crossover restarted chronic transfusion on a clinical basis, and 43 of these 49 were on treatment at study closeout. Of the 67 randomized to standard care, 42 were being transfused; 13 for stroke, one for leg ulcers, and 28 who chose treatment to prevent stroke as of 06/30/00. For the 104 enrolled in the follow-up phase without stroke, TCD and MRI findings at last testing (mid-late 1999) were: 19 (18.27%) abnormal, 34 (32.69%) conditional, 41 (39.42%) normal and 10 (9.62%) inadequate. Close-out MRI showed that 46 (46.46%) had silent infarcts vs. 37% at baseline. There were 10 patients (out of 39 with an évaluable final MRA) with moderate or severe lesions on MRA. A total of 4,536 transfusions were delivered in this study (on and off protocol). No HIV or new Hepatitis C infections were detected. A total of 72 patients received chelation during the study. The STOP Trial demonstrated that: TCD can predict stroke, and the risk is sustained for at least 2-3 years after abnormal exam: transfusion lowers risk of stroke, but is not accepted by all patients, and is associated with complications, especially iron overload. A second trial will examine the duration of transfusion needed to achieve primary stroke prevention in this setting.

Original languageEnglish (US)
Pages (from-to)10a
JournalBlood
Volume96
Issue number11 PART I
StatePublished - Dec 1 2000

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ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

Cite this

Adams, R., Brambilla, D., McKie, V., Files, B., Vichinsky, E., Abboud, M., Hsu, L., Pegelow, C., Woods, G., Miller, S., Wang, W., Olivieri, N., Berman, B., Hurlet, A., Piomelli, S., & Driscoll, C. (2000). Stroke prevention in sickle cell disease (stop): Final results. Blood, 96(11 PART I), 10a.