title: Feasibility and results of bone marrow transplantation from an HLA-mismatched unrelated donor for children and young adults with acquired severe aplastic anemia.
abstract: Treating patients with severe aplastic anemia (SAA) who fail to respond to immunosuppressive therapy (IST) and do not have an HLA-matched donor is challenging. We report favorable outcomes in 11 patients who underwent bone marrow transplantation (BMT) from an HLA-mismatched unrelated donor. The median age was 11 years (range, 3-20 years). The conditioning regimen consisted of cyclophosphamide (200 mg/kg), antithymocyte globulin (10 mg/kg), and total body irradiation (5 Gy). Patients received tacrolimus and methotrexate for prophylaxis against graft-versus-host disease (GVHD). Donorrecipient pairs were mismatched for the HLA-DR antigen in 8 patients by serologic typing. HLA-A and HLA-B antigens were mismatched in 1 and 2 patients, respectively. Ten patients achieved engraftment. One patient who failed to engraft was rescued by a second transplantation from her mother, who was mismatched at 2 HLA antigens. Acute GVHD of grades II to IV occurred in 2 patients. Three patients developed limited chronic GVHD, and 1 patient developed extensive chronic GVHD of the lung. All patients are alive at 9 to 56 months after transplantation (median, 33 months). Considering our encouraging results, HLA-mismatched unrelated-donor BMT for SAA is feasible as a salvage therapy for nonresponders to IST.
authors.affiliation: Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan.
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Yagasaki
authors.forename: Yoshiyuki
authors.initials: Y
authors.lastname: Takahashi
authors.forename: Kazuko
authors.initials: K
authors.lastname: Kudo
authors.forename: Haruhiko
authors.initials: H
authors.lastname: Ohashi
authors.forename: Asahito
authors.initials: A
authors.lastname: Hama
authors.forename: Tomoko
authors.initials: T
authors.lastname: Yamamoto
authors.forename: Makito
authors.initials: M
authors.lastname: Tanaka
authors.forename: Nao
authors.initials: N
authors.lastname: Yoshida
authors.forename: Hirokazu
authors.initials: H
authors.lastname: Hidaka
authors.forename: Nobuhiro
authors.initials: N
authors.lastname: Nishio
authors.forename: Seiji
authors.initials: S
authors.lastname: Kojima
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000628:therapy*; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D005240:Feasibility Studies; D005260:Female; D006084:Graft Rejection; D006086:Graft vs Host Disease; D006648:Histocompatibility; D015395:Histocompatibility Antigens Class I; D006650:Histocompatibility Testing; D006801:Humans; D008297:Male; D016879:Salvage Therapy; D012720:Severity of Illness Index; D014019:Tissue Donors; D019172:Transplantation Conditioning; D016896:Treatment Outcome
chemical_list: D015395:Histocompatibility Antigens Class I