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.
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.
Booth
Claire
C
Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UK.
Ribeil
Jean-Antoine
JA
Audat
Françoise
F
Dal-Cortivo
Liliane
L
Veys
Paul A
PA
Thrasher
Adrian J
AJ
Davies
E Graham
EG
Lefrère
François
F
Fischer
Alain
A
Cavazzana-Calvo
Marina
M
Gaspar
H Bobby
HB
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
D018952:Antigens, CD34