title: What Unrelated Hematopoietic Stem Cell Transplantation in Thalassemia Taught us about Transplant Immunogenetics.
abstract: Although the past few decades have shown an improvement in the survival and complication-free survival rates in patients with beta-thalassemia major and gene therapy is already at an advanced stage of experimentation, hematopoietic stem cell transplantation (HSCT) continues to be the only effective and realistic approach to the cure of this chronic non-malignant disease. Historically, human leukocyte antigen (HLA)-matched siblings have been the preferred source of donor cells owing to superior outcomes compared with HSCT from other sources. Nowadays, the availability of an international network of voluntary stem cell donor registries and cord blood banks has significantly increased the odds of finding a suitable HLA matched donor. Stringent immunogenetic criteria for donor selection have made it possible to achieve overall survival (OS) and thalassemia-free survival (TFS) rates comparable to those of sibling transplants. However, acute and chronic graft-versus-host disease (GVHD) remains the most important complication in unrelated HSCT in thalassemia, leading to significant rates of morbidity and mortality for a chronic non-malignant disease. A careful immunogenetic assessment of donors and recipients makes it possible to individualize appropriate strategies for its prevention and management. This review provides an overview of recent insights about immunogenetic factors involved in GVHD, which seem to have a potential role in the outcome of transplantation for thalassemia.
authors.affiliation: Bone Marrow Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy; Hematology Unit, Department of Medical Sciences, University of Cagliari, Italy.
authors.forename: Giorgio
authors.initials: G
authors.lastname: La Nasa
authors.affiliation: Bone Marrow Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy.
authors.forename: Adriana
authors.initials: A
authors.lastname: Vacca
authors.affiliation: Regional Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy.
authors.forename: Roberto
authors.initials: R
authors.lastname: Littera
authors.affiliation: Bone Marrow Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy.
authors.forename: Eugenia
authors.initials: E
authors.lastname: Piras
authors.affiliation: Medical Genetics, Department of Medical Sciences, University of Cagliari, Italy.
authors.forename: Sandro
authors.initials: S
authors.lastname: Orru
authors.affiliation: Bone Marrow Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy.
authors.forename: Marianna
authors.initials: M
authors.lastname: Greco
authors.affiliation: Medical Genetics, Department of Medical Sciences, University of Cagliari, Italy.
authors.forename: Carlo
authors.initials: C
authors.lastname: Carcassi
authors.affiliation: Bone Marrow Transplant Center, R. Binaghi Hospital - ASL 8, Cagliari, Italy; Hematology Unit, Department of Medical Sciences, University of Cagliari, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Caocci