title: Unrelated donor cord blood transplantation for childhood severe aplastic anemia after a modified conditioning.
abstract: Treatment of severe aplastic anemia (SAA) patients who lack human leukocyte antigen (HLA)-matched donors and failed immunosuppressive therapy (IST) is challenging. Recently, umbilical cord blood transplantation (CBT) after non-myeloablative therapy has been reported in adult but not in childhood SAA. However, most cases resulted in mixed donor chimerism and incomplete hematological recovery. We reported an 11-yr-old girl with recurred SAA 5 yr after IST who underwent unrelated donor CBT after a modified regimen. This patient had renal and cardiac dysfunction, and lacked suitable bone marrow donors. The 3.9 x 10(7)/kg CB cells from an HLA one-locus mismatched unrelated donor were infused after conditioning with total body irradiation (5 Gy), melphalan (120 mg/m(2)), and fludarabin (120 mg/m(2)). Hematological recovery was favorable in complete chimerism. A major complication was only skin graft-versus-host disease (grade I). CB could be an alternate stem cell source for childhood SAA after modified preparative regimen.
authors.affiliation: Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan. ohgas@pediatr.med.kyushu-u.ac.jp
authors.forename: Shouichi
authors.initials: S
authors.lastname: Ohga
authors.forename: Kiyomi
authors.initials: K
authors.lastname: Ichino
authors.forename: Kazuna
authors.initials: K
authors.lastname: Goto
authors.forename: Sagano
authors.initials: S
authors.lastname: Hattori
authors.forename: Akihiko
authors.initials: A
authors.lastname: Nomura
authors.forename: Hidetoshi
authors.initials: H
authors.lastname: Takada
authors.forename: Katsumasa
authors.initials: K
authors.lastname: Nakamura
authors.forename: Toshiro
authors.initials: T
authors.lastname: Hara
mesh_terms: D000741:Anemia, Aplastic / Q000628:therapy*; D002648:Child; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods; D005260:Female; D005500:Follow-Up Studies; D005938:Glucocorticoids / Q000008:administration & dosage; D006086:Graft vs Host Disease / Q000188:drug therapy; D006648:Histocompatibility; D006650:Histocompatibility Testing; D006801:Humans; D008558:Melphalan / Q000008:administration & dosage; D019653:Myeloablative Agonists / Q000008:administration & dosage; D011239:Prednisolone / Q000008:administration & dosage; D012720:Severity of Illness Index; D013997:Time Factors; D018183:Transplantation Chimera; D019172:Transplantation Conditioning; D016896:Treatment Outcome; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives; D014916:Whole-Body Irradiation / Q000379:methods
chemical_list: D005938:Glucocorticoids; D019653:Myeloablative Agonists; D011239:Prednisolone; D014740:Vidarabine; D008558:Melphalan