title: Successful non-myeloablative stem cell transplantation for a heavily transfused woman with severe aplastic anemia complicated by heart failure.
abstract: A 30-year-old Japanese woman weighing 35 kg with severe hemochromatosis due to multiple transfusions was referred to our clinic for treatment of severe aplastic anemia (SAA). The patient had heart failure with an ejection fraction of 36% requiring diuretics and a severe liver dysfunction with an indocyanine green clearance rate of 18%, as well as other transfusion-related complications such as chronic hepatitis due to hepatitis C virus and diabetes mellitus. She was treated with a non-myeloablative preparative regimen that included fludarabine monophosphate (Flu, 120 mg/m(2)), cyclophosphamide (CY, 1200 mg/m(2)) and antithymocyte globulin (ATG, 15 mg/kg) followed by allogeneic peripheral blood stem cell transplantation (PBSCT) from her HLA-matched sister. The regimen was well tolerated, and engraftment rapidly occurred without any therapy-related complications. Chimerism analysis on day 14 after transplant showed reconstitution with 100% donor cells. She no longer needed transfusion after day 23 and has been well in 90% Karnofsky status at 4 months post transplant. The clinical course of this patient indicates that this preparative regimen enables SAA patients with severe organ failure to safely undergo allogeneic stem cell transplantation.
authors.affiliation: Department of Internal Medicine II, Hokkaido University, School of Medicine, Sapporo, Japan.
authors.forename: M
authors.initials: M
authors.lastname: Nishio
authors.forename: S
authors.initials: S
authors.lastname: Nakao
authors.forename: T
authors.initials: T
authors.lastname: Endo
authors.forename: K
authors.initials: K
authors.lastname: Fujimoto
authors.forename: H
authors.initials: H
authors.lastname: Takashima
authors.forename: T
authors.initials: T
authors.lastname: Sakai
authors.forename: A
authors.initials: A
authors.lastname: Bacigalupo
authors.forename: T
authors.initials: T
authors.lastname: Koike
authors.forename: K
authors.initials: K
authors.lastname: Sawada
mesh_terms: D000328:Adult; D045930:Anabolic Agents / Q000627:therapeutic use; D000741:Anemia, Aplastic / Q000150:complications / Q000188:drug therapy / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D003131:Combined Modality Therapy; D016572:Cyclosporine / Q000627:therapeutic use; D048909:Diabetes Complications; D005260:Female; D006333:Heart Failure / Q000150:complications*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D019190:Iron Overload / Q000209:etiology; D008775:Methylprednisolone / Q000627:therapeutic use; D013601:T-Lymphocytes; D065227:Transfusion Reaction; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives
chemical_list: D045930:Anabolic Agents; D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D014740:Vidarabine; C024352:fludarabine; D008775:Methylprednisolone