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Progress in hematopoietic stem cell transplantation as allogeneic cellular gene therapy in thalassemia
Author(s) -
Isgrò Antonella,
Gaziev Javid,
Sodani Pietro,
Lucarelli Guido
Publication year - 2010
Publication title -
annals of the new york academy of sciences
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.712
H-Index - 248
eISSN - 1749-6632
pISSN - 0077-8923
DOI - 10.1111/j.1749-6632.2010.05543.x
Subject(s) - thalassemia , transplantation , medicine , hematopoietic stem cell transplantation , disease , stem cell , haematopoiesis , genetic enhancement , immunology , oncology , pediatrics , gene , biology , genetics
Allogeneic hemopoietic stem cell transplantation (HSCT) represents one of the best cures for thalassemia. Currently, HSCT for thalassemia consists of allogeneic stem cell gene therapy and still awaits autologous genetically modified stem cell transplantation. HSCT for thalassemia has substantially improved over the last two decades, due in large part to improvements in preventive strategies, the effective control of transplant‐related complications, and the development of new preparative regimens. A risk classes‐based approach to transplantation in thalassemia has led to disease‐free survival probability of 87, 85, and 80% in classes 1, 2, and 3 patients, respectively. Adult thalassemia patients, who are higher risk patients for transplant‐related toxicity due to an advanced phase of the disease, have a cure rate of 65% with current treatment protocol. Patients who do not have matched family or unrelated donors could benefit from haploidentical mother‐to‐child transplantation. Overall, the results of this type of transplantation appear encouraging.