Thalassemia Major: Transplantation or Transfusion and Chelation

Publication date: Available online 20 June 2017 Source:Hematology/Oncology and Stem Cell Therapy Author(s): Said Y. Mohamed Thalassemia is the most common monogenic hematologic disease that affects millions in the world and kills thousands of patients every year. Without transfusion or transplantation, patients with thalassemia major are expected to die within months of diagnosis. However, long-term transfusion and chelation therapy is highly challenging for many developing countries where the disease is prevalent, representing a major and unsustainable health burden. Stem cell transplantation is the only cure for thalassemia. It has witnessed major developments that have made it less toxic, more successful, and feasible for a larger number of patients with diverse comorbidities and from a wider range of donors. Advances in human leukocyte antigen typing have greatly refined alternate donor selection with results of matched unrelated donors similar to matched sibling donors. Novel strategies such as haploidentical and cord blood transplantation have increased the possibility of patients with no healthy donor to get a better opportunity to survive and avoid chronic transfusion complications. Cost-effectively, transplantation should be considered the primary treatment of choice in the presence of a suitable related or unrelated donor and at centers with a satisfactory experience in the field of transplantation and particularly, in managing those with thalassemia. Despite som...
Source: Hematology Oncology and Stem Cell Therapy - Category: Cancer & Oncology Source Type: research