Allogeneic Transplantation of CD34^+ Peripheral Blood Progenitor Cellsfor Severe Combined Immunodeficiency

概要

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Severe combined immunodeficiency (SCID) is a fatal congenital disorder of the immune system for which the only curative treatment is the reconstitution of the immune system by stem cell transplantation. In the case reported here we attempted the allogeneic transplantation of purified CD34^+ cells derived from granulocyte-colony stimulating factor (G-CSF) mobilized peripheral blood of the HLA-haploidentical father for a three-month-old female SCID patient, in whom both T and B cells were absent, but natural killer (NK) cells were present. Her father was administered with G-CSF (10μg/kg/day) subcutaneously for four days, and a total number of 2.3×10^7 (=4.6×10^6/kg of patient's body weight) CD34^+ cells was harvested. Purified CD34^+ cells were transplanted into the patient without preconditioning. A skin rash consistent with graft-versushost disease (GVHD) was observed on day 10 after transplantation. CD3^+ cells appeared in the peripheral blood on day 14. These findings indicate that allogeneic transplantation of purified CD34^+ cells could be one useful treatment option for SCID patients, although data from a greater number of patients will be required to draw definitive conclusions.
新潟大学の論文