title: Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency.
abstract: Primary immunodeficiencies (PID) are an important cause of childhood mortality. Haematopoietic stem cell transplantation (HSCT) is the best treatment for many PID. Umbilical cord stem cells are an alternative source of HSC. There is little data regarding outcome of umbilical cord stem cell transplantation (UCSCT) for PID. Our single centre experience is reported. A retrospective study of 14 of 148 patients transplanted for PID, who have received 15 UCSCT was performed, with specific regard to graft-versus-host disease (GvHD) and immune reconstitution. Eight patients with severe combined immunodeficiency (SCID), and six with other combined immunodeficiencies were treated. Of the patients, 12 received unrelated cords, and two had sibling transplants. Median age at transplant was 3.5 months, median nucleated cell dose was 0.8 x 10(8)/kg. All engrafted. Median time to neutrophil engraftment was 22 days, median time to platelet engraftment was 51 days. One developed significant grade III GvHD post transplantation. In total, 11 patients had full donor T and six full donor B-cell chimerism, six of nine patients >1 year post-BMT had normal IgG levels and specific antibody responses to tetanus and Hib vaccines; two are being assessed. Two patients died of multi-organ failure related to pre-existing infection and inflammatory complications respectively. UCSCT should be considered for patients requiring stem cell therapy for PID.
authors.affiliation: Paediatric Immunology Department, Newcastle General Hospital, Newcastle upon Tyne, UK.
authors.forename: A
authors.initials: A
authors.lastname: Bhattacharya
authors.forename: M A
authors.initials: MA
authors.lastname: Slatter
authors.forename: C E
authors.initials: CE
authors.lastname: Chapman
authors.forename: D
authors.initials: D
authors.lastname: Barge
authors.forename: A
authors.initials: A
authors.lastname: Jackson
authors.forename: T J
authors.initials: TJ
authors.lastname: Flood
authors.forename: M
authors.initials: M
authors.lastname: Abinun
authors.forename: A J
authors.initials: AJ
authors.lastname: Cant
authors.forename: A R
authors.initials: AR
authors.lastname: Gennery
mesh_terms: D000917:Antibody Formation; D001402:B-Lymphocytes; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease; D006801:Humans; D007107:Immune System / Q000166:cytology / Q000502:physiology; D007074:Immunoglobulin G / Q000097:blood; D007153:Immunologic Deficiency Syndromes / Q000628:therapy*; D007223:Infant; D008297:Male; D012038:Regeneration; D012189:Retrospective Studies; D013601:T-Lymphocytes; D018183:Transplantation Chimera; D016896:Treatment Outcome
chemical_list: D007074:Immunoglobulin G