1. ¹Department of Paediatric Haematology and Oncology, Hospital Vall d'Hebron, Barcelona, Spain
2. ²Department of Paediatric Haematology and Oncology, Hospital Niño Jesús, Madrid, Spain
3. ³Department of Paediatric Haematology and Oncology, Hospital Sant Pau, Barcelona, Spain

Correspondence: Dr C Díaz de Heredia, Department of Paediatric Haematology and Oncology, BMT Unit, Hospital Vall d'Hebron, P° Vall d'Hebron 119–129, Barcelona 08035, Spain. E-mail: crdiaz@vhebron.net

Received 30 November 2006; Revised 24 October 2007; Accepted 1 November 2007; Published online 17 December 2007.

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 10⁷/kg infused was 7.9 (range, 2.9–25.0) and median CD34 10⁵/kg 2.9 (range, 1.0–7.9). All patients engrafted. Median days to >0.5 10⁹/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 0.730.12. 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.