title: CD34 stem cell top-ups without conditioning after initial haematopoietic stem cell transplantation for correction of incomplete haematopoietic and immunological recovery in severe congenital immunodeficiencies.
abstract: Haematopoietic stem cell transplantation can be limited by ineffective haematopoiesis and poor immune recovery. A CD34(+) cell infusion without conditioning has the potential to improve stem cell function with limited toxicity. Eighteen patients with congenital immunodeficiencies received CD34(+) boosts for various defects. When given <1 year after the original graft, six of seven cytopenic patients achieved transfusion independence. A second cohort (n = 11) received boosts >1 year after the original graft; only minimal changes in immune function or chimaerism were noted. Unconditioned stem cell boosts have limited toxicity but should be given early after the original graft to be effective.
authors.affiliation: Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UK.
authors.forename: Claire
authors.initials: C
authors.lastname: Booth
authors.forename: Jean-Antoine
authors.initials: JA
authors.lastname: Ribeil
authors.forename: Françoise
authors.initials: F
authors.lastname: Audat
authors.forename: Liliane
authors.initials: L
authors.lastname: Dal-Cortivo
authors.forename: Paul A
authors.initials: PA
authors.lastname: Veys
authors.forename: Adrian J
authors.initials: AJ
authors.lastname: Thrasher
authors.forename: E Graham
authors.initials: EG
authors.lastname: Davies
authors.forename: François
authors.initials: F
authors.lastname: Lefrère
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana-Calvo
authors.forename: H Bobby
authors.initials: HB
authors.lastname: Gaspar
mesh_terms: D000293:Adolescent; D018952:Antigens, CD34 / Q000032:analysis*; D002648:Child; D002675:Child, Preschool; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000151:congenital* / Q000628:therapy*; D007223:Infant; D013997:Time Factors; D019172:Transplantation Conditioning; D016896:Treatment Outcome
chemical_list: D018952:Antigens, CD34