Successful reduced-intensity SCT from unrelated cord blood in three patients with X-linked SCID

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We described three boys with X-linked severe combined immunodeficiency (X-SCID) who were successfully treated by reduced-intensity stem cell transplantation from unrelated cord blood (CB). Mean age at the transplantation was 5.7 months (range, 3-9 months). Pre-transplantation conditioning for all patients consisted of fludarabine (FLU) (30 mg/m2/day) from day -7 to day -2 (total dose 180 mg/m2) and busulfan (BU) 4mg/kg/day from day -3 to day -2 (total dose 8 mg/kg). All of the CB units were serologically matched HLA-A, B, and DR loci. Although two of them had suffered from fungal or bacterial pneumonia before transplantation, there were no other infectious complications during transplantation. All the patients have engrafted and achieved 100% donor chimerism. We also confirmed full donor chimerism of both T and B cells. Only one patients developed acute GVHD grade III, which resolved by increasing the dose of oral corticosteroid. None of the patients have developed chronic GVHD during the follow up for 21 to 77 months. No patients had received intravenous immunoglobulin replacements after the transplantations, or showed delay in psychomotor development. Reduced-intensity conditioning consisting of FLU and BU and transplantation from unrelated CB was effective and safe treatment for patients with X-SCID.
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