PMID- 15934838 OWN - NLM STAT- MEDLINE DCOM- 20060406 LR - 20191026 IS - 1744-7682 (Electronic) IS - 1471-2598 (Linking) VI - 5 IP - 5 DP - 2005 May TI - Haematopoietic stem cell transplantation to treat aplastic anaemia. PG - 617-26 AB - Aplastic anaemia (AA) consists of pancytopenia and empty bone marrow. Its incidence varies worldwide but predominates in developing countries. Diverse aetiologies are involved, with autoimmunity at the centre of the picture. For the 70% of patients with the severe and very severe forms of AA and who lack a human leukocyte antigen (HLA)-matched sibling, immunosuppressive therapy (IST) is key in treating the disease, with a remission rate close to 70%, an 80-90% 5-year survival rate in responding patients and a relapse rate close to 10%. For the 30% with a sibling donor available, haematopoietic stem cell transplant (HSCT) from bone marrow or peripheral blood has up to a 90% chance of cure, with a 5-10% graft rejection/failure rate. Patients who fail IST (25-30%) and lack a sibling donor can benefit from CD34(+)-enriched, partially T cell-depleted unrelated stem cell transplants, with a general survival rate up to 37%, the newest source of stem cells for this modality being cord blood. Non-myeloablative, irradiation-free conditioning regimens offer appreciable benefits, and new immunosuppressive agents, such as fludarabine and alemtuzumab, have been incorporated with promising preliminary results. Graft-versus-host disease, graft failure and infections remain significant challenges in HSCT for which innovative treatment strategies are being developed at present. FAU - Jaime-Perez, Jose C AU - Jaime-Perez JC AD - Universidad Autonoma de Nuevo Leon Servicio de Hematologia, Hospital Universitario, Dr Jose E. Gonzalez, Edificio Dr Rodrigo Barragan, 2 piso., Avenida Madero y Gonzalitos, Monterrey, Nuevo Leon, C.P. 64460, Mexico. carjaime@hotmail.com FAU - Ruiz-Arguelles, Guillermo J AU - Ruiz-Arguelles GJ FAU - Gomez-Almaguer, David AU - Gomez-Almaguer D LA - eng PT - Journal Article PT - Review PL - England TA - Expert Opin Biol Ther JT - Expert opinion on biological therapy JID - 101125414 SB - IM MH - Anemia, Aplastic/blood/*surgery MH - Animals MH - Clinical Trials as Topic/statistics & numerical data MH - Graft Rejection/blood/prevention & control MH - Hematopoietic Stem Cell Transplantation/*methods MH - Humans MH - Transplantation Conditioning/*methods RF - 78 EDAT- 2005/06/07 09:00 MHDA- 2006/04/07 09:00 CRDT- 2005/06/07 09:00 PHST- 2005/06/07 09:00 [pubmed] PHST- 2006/04/07 09:00 [medline] PHST- 2005/06/07 09:00 [entrez] AID - 10.1517/14712598.5.5.617 [doi] PST - ppublish SO - Expert Opin Biol Ther. 2005 May;5(5):617-26. doi: 10.1517/14712598.5.5.617.