title: Umbilical cord-blood transplantations from unrelated donors in patients with inherited metabolic diseases: Single-institute experience.
abstract: We evaluated the feasibility of UCBT from unrelated donors and a myeloablative preparative regimen that did not involve anti-thymocyte globulin in five children with lysosomal and peroxisomal diseases. Patients with MPS II (n = 1), adrenoleukodystrophy (n = 1), metachromatic leukodystrophy (n = 2), and Krabbe disease (n = 1) received UCBT between December 2001 and September 2005. All patients received oral Bu (600 mg/m2), CY (200 mg/kg IV), and fludarabine (180 mg/m2 IV). Prophylaxis for GVHD consisted of a combination of tacrolimus and a short methotrexate course. Neutrophil engraftment occurred a median of 24 days (range, 21-25) after transplantation. None had graft rejection. One patient developed grade III acute GVHD and the other four patients had grade I acute GVHD; none had extensive chronic GVHD. One patient developed hemorrhagic cystitis. There were no treatment-related deaths. Although one child with MPS II died of PTLD 10 months after the UCBT, four of the five children are alive 14, 20, 31, and 55 months after transplantation with complete donor chimerism. These results suggest the feasibility of the UCBT with Bu, fludarabine, and CY-preparative regimen for patients with inherited metabolic diseases.
authors.affiliation: Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan. tokimasa@ped.med.osaka-u.ac.jp
authors.forename: Sadao
authors.initials: S
authors.lastname: Tokimasa
authors.forename: Hideaki
authors.initials: H
authors.lastname: Ohta
authors.forename: Sachiko
authors.initials: S
authors.lastname: Takizawa
authors.forename: Shigenori
authors.initials: S
authors.lastname: Kusuki
authors.forename: Yoshiko
authors.initials: Y
authors.lastname: Hashii
authors.forename: Norio
authors.initials: N
authors.lastname: Sakai
authors.forename: Masako
authors.initials: M
authors.lastname: Taniike
authors.forename: Keiichi
authors.initials: K
authors.lastname: Ozono
authors.forename: Junichi
authors.initials: J
authors.lastname: Hara
mesh_terms: D000284:Administration, Oral; D002066:Busulfan / Q000009:adverse effects; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods*; D003520:Cyclophosphamide / Q000009:adverse effects; D005260:Female; D006086:Graft vs Host Disease; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects; D007223:Infant; D016464:Lysosomal Storage Diseases / Q000628:therapy*; D008297:Male; D008727:Methotrexate / Q000009:adverse effects; D018901:Peroxisomal Disorders / Q000628:therapy*; D014740:Vidarabine / Q000009:adverse effects / Q000031:analogs & derivatives
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine; D008727:Methotrexate