PMID- 24104412 OWN - NLM STAT- MEDLINE DCOM- 20140403 LR - 20220129 IS - 1531-7048 (Electronic) IS - 1065-6251 (Linking) VI - 20 IP - 6 DP - 2013 Nov TI - Haematopoietic stem cell transplantation for acquired aplastic anaemia. PG - 515-20 LID - 10.1097/MOH.0b013e328365af83 [doi] AB - PURPOSE OF REVIEW: Survival outcomes from haematopoietic stem cell transplantation (HSCT) in severe aplastic anaemia (SAA) have improved steadily over the past decades, largely reflecting progress in supportive care and conditioning regimens. Here we review recently published data that highlight the improvements and current issues. RECENT FINDINGS: Human leukocyte antigen (HLA)-matched sibling donor (MSD) HSCT remains the gold standard for SAA patients younger than 40-50 years, with HLA-matched unrelated donor (MUD) HSCT for second line after failure to respond to immunosuppressive therapy (IST). The use of alternative donor sources for aplastic anaemia patients remains limited and problematic, but novel conditioning regimens, particularly in the haploidentical setting, justify further evaluation. In recent studies when comparing alemtuzumab-based conditioning with standard antithymocyte globulin conditioning regimens, lower rates of acute and chronic graft-versus-host disease and better tolerance in older patients are seen. SUMMARY: Improving outcomes may lead to an expanded frontline HSCT role in the future. In children lacking a MSD, increasingly MUD HSCT is being considered as first-line treatment and is also being considered more for young adults. Further research is needed to advance our understanding of the role HSCT has to play in SAA with particular emphasis on alternative donor sources and identifying optimal conditioning regimens. FAU - Young, Moya E AU - Young ME AD - aHaematology Department, Kings College Hospital bKings College London, London, UK. FAU - Potter, Victoria AU - Potter V FAU - Kulasekararaj, Austin G AU - Kulasekararaj AG FAU - Mufti, Ghulam J AU - Mufti GJ FAU - Marsh, Judith C AU - Marsh JC LA - eng GR - MR/J006742/1/MRC_/Medical Research Council/United Kingdom PT - Journal Article PT - Review PL - United States TA - Curr Opin Hematol JT - Current opinion in hematology JID - 9430802 RN - 0 (Antineoplastic Agents) SB - IM MH - Age Factors MH - Anemia, Aplastic/*therapy MH - Antineoplastic Agents/therapeutic use MH - Donor Selection MH - Graft Rejection/prevention & control MH - Hematopoietic Stem Cell Transplantation/*methods MH - Humans MH - Transplantation Conditioning/methods MH - Transplantation Tolerance EDAT- 2013/10/10 06:00 MHDA- 2014/04/04 06:00 CRDT- 2013/10/10 06:00 PHST- 2013/10/10 06:00 [entrez] PHST- 2013/10/10 06:00 [pubmed] PHST- 2014/04/04 06:00 [medline] AID - 00062752-201311000-00006 [pii] AID - 10.1097/MOH.0b013e328365af83 [doi] PST - ppublish SO - Curr Opin Hematol. 2013 Nov;20(6):515-20. doi: 10.1097/MOH.0b013e328365af83.