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Neurology Research Programs: Stroke Research In Sickle Cell Disease
   
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Stroke Prevention using Transcranial Doppler and Blood Transfusion

How do transfusions work?
Only red blood cells, which carry oxygen, are given to the patient. It is not clear how transfusion prevents stroke, but we know that it increases the ability of the blood to carry oxygen to the brain and other organs and lowers the number of sickle red blood cells in the bloodstream. These effects may stabilize or reverse the conditions in the abnormal brain arteries and prevent the development of stroke.

How safe is transfusion?
The blood supply in the US and Canada is very safe, but risk can never be totally eliminated. Patients in the STOP study were monitored for evidence of blood-borne infections, and at the end of the study, there was no indication of any infection caused by transfusion in over 1500 transfusions.

Transfusion also causes a build-up of iron in the body which can affect vital organs. When red blood cells break down, the iron that is part of each cell is stored in the heart, liver, and other organs. Transfused blood is broken down more rapidly and iron overload becomes a health concern. If a child receives long-term transfusions (3-5 years), chelation is recommended to control the build-up of iron.

Other potential problems are associated with transfusions, including alloimmunization and other reactions. These reactions can be reduced by better matching of blood factors, but cannot yet be eliminated.

How long must children be treated with transfusion after a first-time stroke?
Transfusions used to prevent a second stroke are usually continued at least until the child grows to adulthood. Even then it is not clear how safe it is to stop transfusions. For now, children must remain on treatment indefinitely until further research determines when transfusion can be safely stopped or replaced by another therapy. STOP II is attempting to provide some answers to this question.

Will blood transfusions prevent strokes in all children at risk?
No, STOP data indicate that a small number of children may still have a stroke despite transfusion treatment.

Are there alternatives to transfusion?
Although there is an interest in the use of hydroxyurea, and more standard stroke prevention treatments such as antiplatelet agents and anticoagulation, no clinical trial data support their use for this indication.

Bone marrow transplantation (BMT) can cure SCD, and presumably has the potential to prevent cerebrovascular disease and stroke. Experience with BMT is insufficient to know how well this would work in stroke, although it seems to arrest development of cerebrovascular disease in the small number of patients with stroke and BMT. In France, abnormal TCD is used as an indication for BMT, however opinion is divided in the United States on using abnormal TCD as a sole indication for BMT.

Treatments which are as effective as transfusion but with fewer side effects would represent a great advance in this field and are being considered for investigation. At present, only chronic transfusion is both supported by Class I evidence (a randomized clinical trial of sufficient size and quality) and endorsed by the National Heart, Lung, and Blood Institute.

   
  © 2003 MCG

Questions and Comments to Bill Hamilton 


  February 02, 2004


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