PMID- 36907612 OWN - NLM STAT- MEDLINE DCOM- 20230314 LR - 20230316 IS - 1558-1977 (Electronic) IS - 0889-8588 (Linking) VI - 37 IP - 2 DP - 2023 Apr TI - Hematopoietic Stem Cell Transplantation in Thalassemia. PG - 413-432 LID - S0889-8588(22)00150-2 [pii] LID - 10.1016/j.hoc.2022.12.009 [doi] AB - 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. CI - Copyright (c) 2022 Elsevier Inc. All rights reserved. FAU - Algeri, Mattia AU - Algeri M AD - Department of Hematology/Oncology, Cell and Gene Therapy - IRCCS, Bambino Gesu Children's Hospital, Rome, Italy. Electronic address: mattia.algeri@opbg.net. FAU - Lodi, Mariachiara AU - Lodi M AD - Department of Hematology/Oncology, Cell and Gene Therapy - IRCCS, Bambino Gesu Children's Hospital, Rome, Italy. FAU - Locatelli, Franco AU - Locatelli F AD - Department of Hematology/Oncology, Cell and Gene Therapy - IRCCS, Bambino Gesu Children's Hospital, Rome, Italy; Department of Life Sciences and Public Health, Catholic University of the Sacred Heart, Rome, Italy. LA - eng PT - Journal Article PT - Review PL - United States TA - Hematol Oncol Clin North Am JT - Hematology/oncology clinics of North America JID - 8709473 SB - IM MH - Humans MH - Quality of Life MH - Transplantation, Homologous/adverse effects MH - *Hematopoietic Stem Cell Transplantation/methods MH - *Thalassemia/therapy MH - *Graft vs Host Disease MH - Transplantation Conditioning/methods MH - Unrelated Donors OTO - NOTNLM OT - Cord blood transplantation OT - Haploidentical transplantation OT - Hematopoietic stem cell transplantation OT - Hemoglobinopathies OT - Sibling donor transplantation OT - Thalassemia OT - Unrelated donor transplantation EDAT- 2023/03/13 06:00 MHDA- 2023/03/15 06:00 CRDT- 2023/03/12 21:55 PHST- 2023/03/12 21:55 [entrez] PHST- 2023/03/13 06:00 [pubmed] PHST- 2023/03/15 06:00 [medline] AID - S0889-8588(22)00150-2 [pii] AID - 10.1016/j.hoc.2022.12.009 [doi] PST - ppublish SO - Hematol Oncol Clin North Am. 2023 Apr;37(2):413-432. doi: 10.1016/j.hoc.2022.12.009.