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

C Diaz de Heredia, JJ Ortega, MA Diaz… - Bone marrow …, 2008 - nature.com
C Diaz de Heredia, JJ Ortega, MA Diaz, T Olive, I Badell, M Gonzalez-Vicent…
Bone marrow transplantation, 2008nature.com
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 …
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× 10 7/kg infused was 7.9 (range, 2.9–25.0) and median CD34× 10 5/kg 2.9 (range, 1.0–7.9). All patients engrafted. Median days to> 0.5× 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.
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