thalassemia

• Projects

  

  More than 50,000 children have thalassemia major in Pakistan (photos), for many bone marrow transplantation offers the only chance of survival. Cure2Children has supported the cure of an initial group of children and gave a critical contribution to the start up of two new transplant units managed by Pakistani professionals.

  Cure2Children, with SOS Infanzia nel Mondo, Policlinico Gemelli in Rome, and the Italian peace-keeping force, has supported the treament of the first children with leukemia in Kosovo (photos).

   

• A stem cell transplant network in Pakistan

  Thalassemia major is the most common life-threatening genetic disease. Even though long-term supportive care with regular red cell transfusion and intensive chelation therapy for iron overload may prolong life-expectancy to the fourth decade of life, bone marrow transplantation remains the only curative option. Appropriate supportive care is quite expensive and may not be regularly available in most low-income areas. Without transfusion support children rarely survive beyond 5 years and with transfusions but no chelation therapy life expectancy rarely extends beyond adolescence. The overall costs of long term supportive care are well above those of transplantation, this trend will most likely increase over time as chelation therapy becomes more expensive while transplant procedures tend to become simpler and more tolerable.

• Workshop on thalassemia treatment in Pakistan

  Cure2Children has organised a 2-days workshop in Siena to discuss about the Thalassemia Project.

• Collaborative projects for the cure of Thalassemia Major

  Casa Marchini Carrozza, Via Portigiani 3, Fiesole (Florence, Italy).

   

• Thalassemia major

  Thalassemia major is the most common life-threatening genetic disease. Even though long-term supportive care with regular red cell transfusion and intensive chelation therapy for iron overload may prolong life-expectancy to the fourth decade of life, bone marrow transplantation remains the only curative option. Appropriate supportive care is quite expensive and may not be regularly available in most low-income areas. Without transfusion support children rarely survive beyond 5 years and with transfusions but no chelation therapy life expectancy rarely extends beyond adolescence. The overall costs of long term supportive care are well above those of transplantation, this trend will most likely increasing over time as chelation therapy becomes more expensive while transplant procedures tend to become simpler and more tolerable.

• A thalassemia major prevention program in PakistanThis project will proceed in parallel with the transplantation program and will aim at the screening of first-degree relative of thalassemia patients enrolled in the transplantation program. In fact the carrier frequency in this selected population will probably be in the range of 30% as opposed to the 5-6% of the general population in Pakistan. Considering that the average number of children per family is 4, the total number of first degree relatives will be 30 (6 uncle/aountie plus 24 cousins). Brothers and sisters of the transplant candidate will be evaluated as part of transplant preparation.