Transcript PowerPoint

Sickle Cell Research
Research Topics
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Hydroxyurea
Bone Marrow Transplant
STOP Study
Gene Therapy
Adhesion Blockers
Hydroxyurea
Hydroxyurea or Hydrea has been found to do the
following in adult sickle cell patients:
1. Reduce pain events by one half,
2. Reduce hospital admissions by one half,
3. Reduced the need for transfused blood,
4. Raised the patient's hemoglobin on average by one
gram.
Bone Marrow Transplant
Bone Marrow Transplantation is for those patients with enough
complications from sickle cell disease to take the risk of this
procedure, that replaces the patient's bone marrow with a near
perfect donor marrow, usually from a brother or sister.This
procedure involves destroying the patient's bone marrow with
medications and transfusing the donated marrow as new "seed" .
The patient will start making red cells that the donor makes and not
red cells with the sickle hemoglobin.
STOP Study
The Stroke prevention study uses cranial doppler to record the
sound of blood flow in the blood vessels supplying blood to the
brain. Any blockage will increase the noise of the blood flow and
this may be an early sign that a stroke could occur. Special
studies like MRI can be done to look at the blood flow in the
brain. Strokes may be prevented by a chronic blood transfusion
program
Gene Therapy
Research is now underway
to fix the basic genetic
defect, putting back the
correct amino acid in the
hemoglobin before or
shortly after birth. This
would result in a cure of
the root of the problem.
The method to do this
safely is still undergoing
work in many research
labs around the world
Adhesion Blockers
Medications that keep red cells from
sticking will prevent pain and damage