title: Unrelated cord blood transplantation in children with idiopathic severe aplastic anemia.
abstract: Early results of unrelated cord blood transplantation (UCBT) for severe aplastic anemia (SAA) were poor with a high rate of engraftment failure. This was attributed to the combination of lower graft cell dose and intact host immune system. We performed UCBT in nine children (median age 9 years) with refractory SAA using increasingly immunosuppressive preparative regimens. The time from diagnosis to UCBT was 3.4-20 months (median age 7.2 years), with all children having failed at least one course of immunosuppression. Donor/recipient HLA matching was six of six (n=1), five of six (n=2) and four of six (n=6). The median nucleated cell dose infused was 5.7 x 10(7) cells/kg (range 3.5-20 x 10(7) cells/kg). Six patients were engrafted after the first UCBT. Two of the three patients without hematopoietic reconstitution were engrafted after a second UCBT. All children receiving >or=120 mg/kg of CY in the preparative regimen were engrafted. The median time to myeloid engraftment was 25 (17-59 days) days. Acute GVHD developed in two, and chronic GVHD in five patients. Five patients developed EBV viremia post transplant (lymphoproliferative disorder in three patients). At a median follow-up of 34 months, seven patients are alive and transfusion-independent. UCBT is a feasible treatment strategy for children with refractory SAA lacking a well-matched adult donor.
authors.affiliation: Division of Pediatric Blood and Marrow Transplantation, Texas Transplant Institute and Methodist Children's Hospital, San Antonio, TX 78229, USA. kawah.chan@mhshealth.com
authors.forename: K W
authors.initials: KW
authors.lastname: Chan
authors.forename: L
authors.initials: L
authors.lastname: McDonald
authors.forename: D
authors.initials: D
authors.lastname: Lim
authors.forename: M S
authors.initials: MS
authors.lastname: Grimley
authors.forename: G
authors.initials: G
authors.lastname: Grayson
authors.forename: D A
authors.initials: DA
authors.lastname: Wall
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D018572:Disease-Free Survival; D006084:Graft Rejection / Q000276:immunology; D006085:Graft Survival / Q000276:immunology; D006086:Graft vs Host Disease / Q000276:immunology; D006801:Humans; D007223:Infant; D018183:Transplantation Chimera; D016896:Treatment Outcome; D055815:Young Adult