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.