title: Transplantation of highly purified CD34+ progenitor cells from alternative donors in children with refractory severe aplastic anaemia.
abstract: Without transplantation from a human leucocyte antigen-identical family donor, refractory severe aplastic anaemia (SAA) has an unfavourable prognosis. Conventional transplantation from a matched unrelated donor carries a high rate of mortality. We transplanted large numbers of highly purified CD34+ cells from matched unrelated (n = 4), mismatched unrelated (n = 4) and mismatched related (n = 1) donors into nine children with refractory SAA. The grafts consisted of granulocyte colony-stimulating factor-mobilized peripheral positively selected CD34+ cells. A median of 15.1 x 106/kg CD34+ stem cells and 11 x 103/kg CD3+ T-lymphocytes were infused. No additional pharmacological graft versus host disease (GVHD) prophylaxis was given. At a median follow-up of 47 (range 37-72) months, eight patients (89%) were in complete remission with >90% donor chimaerism and no evidence of GVHD. One patient died on day +238 as a consequence of GVHD. The use of highly purified mobilized CD34+ stem cells warrants further clinical exploration in children with refractory SAA.
authors.affiliation: Department of Paediatric Haematology/Oncology, Children's University Hospital, Karl-Franzens-University Graz, Graz, Austria.
authors.forename: Martin
authors.initials: M
authors.lastname: Benesch
authors.forename: Christian
authors.initials: C
authors.lastname: Urban
authors.forename: Karl W
authors.initials: KW
authors.lastname: Sykora
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Schwinger
authors.forename: Felix
authors.initials: F
authors.lastname: Zintl
authors.forename: Herwig
authors.initials: H
authors.lastname: Lackner
authors.forename: Peter
authors.initials: P
authors.lastname: Lang
authors.forename: Rupert
authors.initials: R
authors.lastname: Handgretinger
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000628:therapy*; D000753:Anemia, Refractory / Q000628:therapy*; D018952:Antigens, CD34 / Q000097:blood*; D002469:Cell Separation / Q000379:methods; D002648:Child; D005260:Female; D006084:Graft Rejection / Q000517:prevention & control; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006412:Hematopoietic Stem Cells / Q000276:immunology*; D006648:Histocompatibility; D006801:Humans; D008297:Male; D019172:Transplantation Conditioning / Q000379:methods
chemical_list: D018952:Antigens, CD34