Hematopoietic Stem Cell Transplantation in Thalassemia. 2023

Mattia Algeri, and Mariachiara Lodi, and Franco Locatelli
Department of Hematology/Oncology, Cell and Gene Therapy - IRCCS, Bambino Gesù Children's Hospital, Rome, Italy. Electronic address: mattia.algeri@opbg.net.

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only consolidated, potentially curative treatment for patients with transfusion-dependent thalassemia major. In the past few decades, several new approaches have reduced the toxicity of conditioning regimens and decreased the incidence of graft-versus-host disease, improving patients' outcomes and quality of life. In addition, the progressive availability of alternative stem cell sources from unrelated or haploidentical donors or umbilical cord blood has made HSCT a feasible option for an increasing number of subjects lacking an human leukocyte antigen (HLA)-identical sibling. This review provides an overview of allogeneic hematopoietic stem cell transplantation in thalassemia, reassesses current clinical results, and discusses future perspectives.

UI MeSH Term Description Entries
D011788 Quality of Life A generic concept reflecting concern with the modification and enhancement of life attributes, e.g., physical, political, moral, social environment as well as health and disease. HRQOL,Health-Related Quality Of Life,Life Quality,Health Related Quality Of Life
D006086 Graft vs Host Disease The clinical entity characterized by anorexia, diarrhea, loss of hair, leukopenia, thrombocytopenia, growth retardation, and eventual death brought about by the GRAFT VS HOST REACTION. Graft-Versus-Host Disease,Homologous Wasting Disease,Runt Disease,Graft-vs-Host Disease,Disease, Graft-Versus-Host,Disease, Graft-vs-Host,Disease, Homologous Wasting,Disease, Runt,Diseases, Graft-Versus-Host,Diseases, Graft-vs-Host,Graft Versus Host Disease,Graft-Versus-Host Diseases,Graft-vs-Host Diseases
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D013789 Thalassemia A group of hereditary hemolytic anemias in which there is decreased synthesis of one or more hemoglobin polypeptide chains. There are several genetic types with clinical pictures ranging from barely detectable hematologic abnormality to severe and fatal anemia. Thalassemias
D014184 Transplantation, Homologous Transplantation between individuals of the same species. Usually refers to genetically disparate individuals in contradistinction to isogeneic transplantation for genetically identical individuals. Transplantation, Allogeneic,Allogeneic Grafting,Allogeneic Transplantation,Allografting,Homografting,Homologous Transplantation,Grafting, Allogeneic
D061349 Unrelated Donors Providers of tissues for transplant to non-related individuals. Donors, Unrelated,Donor, Unrelated,Unrelated Donor
D018380 Hematopoietic Stem Cell Transplantation Transfer of HEMATOPOIETIC STEM CELLS from BONE MARROW or BLOOD between individuals within the same species (TRANSPLANTATION, HOMOLOGOUS) or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). Hematopoietic stem cell transplantation has been used as an alternative to BONE MARROW TRANSPLANTATION in the treatment of a variety of neoplasms. Stem Cell Transplantation, Hematopoietic,Transplantation, Hematopoietic Stem Cell
D019172 Transplantation Conditioning Preparative treatment of transplant recipient with various conditioning regimens including radiation, immune sera, chemotherapy, and/or immunosuppressive agents, prior to transplantation. Transplantation conditioning is very common before bone marrow transplantation. Conditioning, Transplantation,Conditionings, Transplantation,Transplantation Conditionings

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