title: Hematopoietic stem cell transplantation in thalassemia and sickle cell anemia.
abstract: The globally widespread single-gene disorders β-thalassemia and sickle cell anemia (SCA) can only be cured by allogeneic hematopoietic stem cell transplantation (HSCT). HSCT treatment of thalassemia has substantially improved over the last two decades, with advancements in preventive strategies, control of transplant-related complications, and preparative regimens. A risk class-based transplantation approach results in disease-free survival probabilities of 90%, 84%, and 78% for class 1, 2, and 3 thalassemia patients, respectively. Because of disease advancement, adult thalassemia patients have a higher risk for transplant-related toxicity and a 65% cure rate. Patients without matched donors could benefit from haploidentical mother-to-child transplantation. There is a high cure rate for children with SCA who receive HSCT following myeloablative conditioning protocols. Novel non-myeloablative transplantation protocols could make HSCT available to adult SCA patients who were previously excluded from allogeneic stem cell transplantation.
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia-Mediterranean Institute of Hematology, Policlinic of the University of Rome Tor Vergata, Tor Vergata, Italy. g.lucarelli@fondazioneime.org
authors.forename: Guido
authors.initials: G
authors.lastname: Lucarelli
authors.forename: Antonella
authors.initials: A
authors.lastname: Isgrò
authors.forename: Pietro
authors.initials: P
authors.lastname: Sodani
authors.forename: Javid
authors.initials: J
authors.lastname: Gaziev
mesh_terms: D000328:Adult; D000755:Anemia, Sickle Cell / Q000628:therapy*; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D019520:Living Donors; D019653:Myeloablative Agonists / Q000627:therapeutic use; D013789:Thalassemia / Q000628:therapy*
chemical_list: D019653:Myeloablative Agonists