title: Unrelated cord blood transplantation for severe congenital neutropenia: report of two cases with very different transplant courses.
abstract: SCN is characterized by neutropenia, life-threatening infections, and progression to myelodysplastic syndrome/acute myelogenous leukemia. The only curative option is SCT, but few reports using UCB as a stem cell source exist. Here, we report two SCN patients transplanted with UCB. Patient 1 was transplanted at seven yr of age due to increasingly large injections of G-CSF (>100 microg/kg/day) and the risk of developing leukemia. He engrafted promptly and is clinically well and immune reconstituted >2 yr post-transplant. Patient 2 underwent UCB SCT at nine months of age for recurrent severe infections, despite high doses of G-CSF. He rejected his first graft, having 100% host cells on day +35, and immediately underwent a second UCB SCT. He engrafted but experienced late graft rejection six months after the second transplant. He received a third UCB SCT following a more immunosuppressive conditioning regimen. His course was complicated by HHV-6 viremia and gut GVHD, but he is now clinically well and has 99% donor engraftment >20 months post-transplant. We conclude that UCB is an acceptable stem cell source for SCN patients, but conditioning must be adequately immunosuppressive to ensure engraftment in patients without prior chemotherapy.
authors.affiliation: Department of Pediatrics, James Whitcomb Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, IN 46202-5200, USA.
authors.forename: Melissa K
authors.initials: MK
authors.lastname: Markel
authors.forename: Paul R
authors.initials: PR
authors.lastname: Haut
authors.forename: Jamie A
authors.initials: JA
authors.lastname: Renbarger
authors.forename: Kent A
authors.initials: KA
authors.lastname: Robertson
authors.forename: W Scott
authors.initials: WS
authors.lastname: Goebel
mesh_terms: D036101:Cord Blood Stem Cell Transplantation / Q000379:methods*; D005312:Fetal Blood / Q000378:metabolism*; D006084:Graft Rejection; D006085:Graft Survival; D006086:Graft vs Host Disease; D015654:Herpesvirus 6, Human / Q000378:metabolism; D006801:Humans; D007107:Immune System; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007231:Infant, Newborn; D007938:Leukemia / Q000517:prevention & control; D008297:Male; D009503:Neutropenia / Q000097:blood* / Q000151:congenital; D013577:Syndrome; D019172:Transplantation Conditioning / Q000379:methods*
chemical_list: D007166:Immunosuppressive Agents