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A simple and inexpensive drug that could make the difference for many children with thalassemia in underserved regions

Children with thalassemia produce deformed red blood cells.  This leaves extra iron lose and unabsorbed in their blood and causes a need for expensive and cumberson blood transfusions to be done every 15 days for their entire lives. Additionally, for children in areas with unsafe blood, frequent transfusions bring the risk of contracting Hepetitis C. If a pill were available that could replace the need for blood transfusions many lives could be saved.
 
Hydroxycarbamide (INN) or Hydroxyurea (HU) has the potential to decrease the need for blood transfusion in a significant proportion of thalassemia patients. The safety of HU in children is well established and its role may be particularly relevant in regions where blood products are not readily available or unsafe. After appropriate therapy with HU a hemoglobin increase maybe observed within the first month of treatment in up to 80% of cases.
 
HU has good effects on increasing hemoglobin levels in thalassemia patients, thus freeing some patients from the need for transfusion and decreasing complications such as osteoporosis and extra medullary hematopoiesis, as well as skeletal deformities and splenomegaly, and increasing energy state.
 
Cure2Children, in collaboration with local centers, has embarked on a project aiming at offering free HU to as many children as possible not having regular access to safe transfusions. HU will help them be prepared for a bone marrow transplant (BMT) that could cure their thalassemia, and that the HLA-typing will help them find a match for the bone marrow donation.  C2C believe that every child deserves access to a cure.

Reference:
1)      http://www.thalassaemia.org.cy/hydroxyurea_management.html
2)      http://en.wikipedia.org/wiki/Hydroxycarbamide

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Posted by: Cure2Children