Unrelated cord blood transplantation for severe combined immunodeficiency and other primary immunodeficiencies.

ABSTRACT

HCT is currently the treatment of choice for children with severe primary immunodeficiencies (PIDs). Frequently, these patients lack an HLA-identical sibling donor, and umbilical cord blood (UCB) transplantation may be an option; however, experience in this field remains scant. Fifteen children with PID (SCID 11, X-linked lymphoproliferative syndrome 2, Omenn's syndrome 1, Wiskott-Aldrich syndrome 1) received a UCB transplant. The donor was unrelated in 14 cases and related in 1. Median age at transplant was 11.6 months (range, 2.9-68.0) and median weight 7 kg (range, 4-21). Thirteen patients were conditioned with busulphan and cyclophosphamide and 2 with fludarabine and melphalan. Nine patients received antithymocyte globulin. Median NC x 10(7)/kg infused was 7.9 (range, 2.9-25.0) and median CD34 x 10(5)/kg 2.9 (range, 1.0-7.9). All patients engrafted. Median days to >0.5 x 10(9)/l neutrophils was 31. Eight patients developed acute graft-versus-host disease (GvHD) grades II-IV and one chronic GvHD. Viral and fungal infections were frequent. Four patients died three from GvHD grade IV complicated by infection and one from progressive interstitial lung disease. Five-year survival was 0.73+/-0.12. All surviving patients presented complete immunologic reconstitution. No patient is intravenous immunoglobulin (IVIg) replacement therapy-dependent. UCB transplantation is a valid option for children with PID who lack an HLA-identical sibling donor.